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Are AI-powered skin-check tools on the horizon for dermatologists, PCPs?
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Given that about 6.3 billion smartphones would soon be in use, this AI approach could provide a gateway for “low-cost universal access to vital diagnostic care,” wrote Justin M. Ko, MD, MBA, a dermatologist, and colleagues from Stanford (Calif.) University that included other dermatologists and engineers.
Dr. Ko and his coauthors described how they trained a computer system to identify both benign and cancerous skin lesions. They used an approach known as a convolutional neural network, often deployed for projects seeking to train computers to “see” through image analysis. They said that their test of this system found it to be on par with the performance of 21 board-certified dermatologists.
“This fast, scalable method is deployable on mobile devices and holds the potential for substantial clinical impact, including broadening the scope of primary care practice and augmenting clinical decision-making for dermatology specialists,” they wrote in their paper.
More than 6 years later, there are signs that companies are making progress toward moving skin checks using this technology into U.S. primary care settings – but only with devices that employ special tools.
It may prove tougher for companies to eventually secure the sign-off of the U.S. Food and Drug Administration for mobile apps intended to let consumers handle this task with smartphones.
Such tools would need to be proven highly accurate before release, because too many false positives mean that people would be needlessly exposed to biopsies, said Sancy A. Leachman, MD, PhD, director of the melanoma research program and chair of the department of dermatology at Oregon Health & Science University, Portland.
And false-negative readings would allow melanoma to advance and even be fatal, Dr. Leachman told this news organization.
Roxana Daneshjou, MD, PhD, a dermatologist at Stanford who has studied the promise and the pitfalls of AI in medicine, said that developers of a consumer skin-check app would need to know how people would react to their readings. That includes a good sense of how often they would appropriately seek medical care for a concerning reading. (She was not an author of the previously cited Nature paper but has published widely on AI.)
“The direct-to-consumer diagnostic space makes me nervous,” Dr. Daneshjou said in an interview. “In order to do it, you really need to have good studies in consumer populations prior to release. You need to show how effective it is with follow up.”
FDA shows interest – and reservations
As of July, the FDA had not yet given its okay for marketing of any consumer apps intended to help people detect signs of skin cancer, an agency spokesperson told this news organization.
To date, the agency has only cleared two AI-based products for this task, both meant to be used by dermatologists. And only one of these two products, Scibase’s Nevisense, remains in use in the United States. The other, MelaFind, has been discontinued. In 2017, Strata Skin Sciences said that the product did not win “a significant enough level of acceptance by dermatologists to justify the continued investment” in it. And the company said it notified the 90 owners of MelaFind devices in the United States that it would no longer support the device.
But another company, DermaSensor, said in a 2021 press release that it expects its AI-powered tool, also named DermaSensor, to be the “first ever FDA cleared or approved skin cancer detection device for primary care providers.”
The Miami-based firm said that the FDA had granted its product a “breakthrough” device designation. A breakthrough designation means that agency staff will offer extra help and guidance to companies in developing a product, because of its expected benefit for patients.
In a 2020 press release, 3Derm Systems, now owned by Digital Diagnostics, made a similar announcement about winning FDA breakthrough designation for an AI-powered tool intended to allow skin checks in primary care settings.
(The FDA generally does not comment on its reviews of experimental drugs and devices, but companies can do so. Several other companies have announced FDA breakthrough designations for AI-driven products intended to check for skin lesions, but these might be used in settings other than primary care.)
Both DermaSensor and Digital Diagnostics have chairs with notable track records for winning FDA approvals of other devices. DermaSensor’s Maurice Ferre, MD, also is the chairman of Insightec, which in 2016 won the first FDA approval for a device with a breakthrough designation device that uses ultrasound to treat tremors.
In 2018, the FDA allowed Digital Diagnostics, then called IDx, to introduce in the United States the first medical device using AI in primary care offices to check for signs of diabetic retinopathy. This product also had an FDA breakthrough designation. The executive chairman and founder of Digital Diagnostics is Michael Abramoff, MD, PhD, professor of engineering and ophthalmology at the University of Iowa, Iowa City. Dr. Abramoff and the team behind the AI tool for retinopathy, now called the LumineticsCore system, also scored a notable win with Medicare, which agreed to cover use of the product through a dedicated CPT code.
FDA draft guidance
The FDA has acknowledged the interest in broadening access to skin checks via AI.
This was a topic of discussion at a 2-day advisory committee meeting the FDA held last year. In April 2023, the FDA outlined some of its expectations for future regulation of skin-analyzing tools as part of a wide-ranging draft guidance document intended to aid companies in their efforts to develop products using a form of AI known as machine learning.
In the document, the FDA described how it might approach applications for “hypothetical” devices using this kind of AI, such as a special tool to help primary care clinicians identify lesions in need of further investigation. Such a product would use a specific camera for gathering data for its initial clearance, in the FDA’s hypothetical scenario.
The FDA staff offered technical suggestions about what the developer of this hypothetical device would have to do to extend its use to smartphones and tablets while keeping clinicians as the intended users.
Some of these expanded uses could fall within the bounds of the FDA’s initial clearance and thus not trigger a need for a new marketing submission, the agency said. But seeking to shift this hypothetical product to “patient-facing” use would require a new marketing submission to the FDA, the agency said.
In this scenario, a company would expect people to follow up with a dermatologist after receiving a report suggesting cancer. Thus, this kind of a change could expose patients to “many new, unconsidered risks,” the FDA said.
Reality check?
The state of current efforts to develop consumer apps for checking for skin cancer seems to be summarized well on the website for the MoleMapper. The app was developed by researchers at OHSU to help people track how their moles change over time.
“Mole Mapper is NOT designed to provide medical advice, professional diagnosis, opinion, or treatment. Currently, there is not enough data to develop an app that can diagnose melanoma, but if enough data is collected through Mole Mapper and shared with researchers, it may be possible in the future,” the app’s website says.
OHSU released MoleMapper as an iPhone app in 2015. The aim of this project was to help people track the moles on their skin while also fostering an experiment in “citizen science,” OHSU’s Dr. Leachman told this news organization.
OHSU researchers hoped that the digital images taken by members of the public on cell phones could one day be used to develop diagnostic algorithms for melanoma.
But around 2017, the MoleMapper team realized that they would not be able to create a diagnostic app at this time, Dr. Leachman explained. They could not collect enough data of adequate quality.
And by 2021, it was clear that they could not even develop a successful app to triage patients to assess who needs to be seen quickly. The amount of data required was, at this point, beyond what the team could collect, Dr. Leachman said in an interview.
That was a disappointment because the team had successfully completed the difficult task of creating a confidential pathway for collecting these images via both iPhones and smartphones run on Android.
“We thought if we built it, people would come, but that’s not what happened,” Dr. Leachman said. Many patients didn’t want their images used for research or would fail to follow up with details of biopsy reports. Sometimes images were not captured well enough to be of use.
“You need at least hundreds of thousands, if not millions, of data points that have been verified with pathologies, and nobody was giving us back that data. That was the reality,” Dr. Leachman said.
There were valuable lessons in that setback. The OHSU team now has a better grasp of the challenges of trying to build a data-collection system that could prove helpful in assessing skin lesions.
“If you don’t build it, you don’t know” what can go wrong, she said.
Dr. Leachman said other scientists who have worked on similar projects to build skin-analyzing apps have probably encountered the same difficulties, although they may not reveal these issues. “I think that a lot of people build these things and then they try to make it into something that it’s not,” she said.
In addition to the challenges with gathering images, dermatologists frequently need to rely on touch and other clues from in-person visits when diagnosing a suspicious lesion. “There’s something about seeing and feeling the skin in person that can’t be captured completely with an image,” Dr. Leachman said.
Public demand
Still, regulators must face the strong and immediate interest consumers have in using AI to check on moles and skin conditions, despite continuing questions about how well this approach might work.
In June, Google announced in a blog post that its Google Lens tool can help people research skin conditions.
“Just take a picture or upload a photo through Lens, and you’ll find visual matches to inform your search,” Google said in a blog post. “This feature also works if you’re not sure how to describe something else on your body, like a bump on your lip, a line on your nails or hair loss on your head. This feature is currently available in the U.S.”
Google also continues work on DermAssist, an app that’s intended to help people get personalized information about skin concerns using three photos. It is not currently publicly available, a Google spokesperson told this news organization.
Several skin-analyzing apps are already available in the Apple and Google Play stores. The British Association of Dermatologists last year issued a press release warning consumers that these apps may not be safe or effective and thus may put patients at risk for misdiagnosis.
“Unfortunately, AI-based apps which do not appear to meet regulatory requirements crop up more often than we would like,” the association said. “Additionally, the evidence to support the use of AI to diagnose skin conditions is weak which means that when it is used, it may not be safe or effective and it is possible that AI is putting patients at risk of misdiagnosis.”
Delicate and difficult balancing act
At this time, regulators, entrepreneurs, and the medical community face a delicate balancing act in considering how best to deploy AI in skin care, Dr. Ko said in an interview. (In addition to being one of the authors on the widely cited 2017 Nature paper mentioned above, Dr. Ko served until March as the initial chair of the American Academy of Dermatology’s Augmented Intelligence Committee.)
There are many solid reasons why there hasn’t been speedy progress to deploy AI in dermatology, as many envisioned a few years ago, Dr. Ko said.
Some of those reasons are specific to dermatology; this field doesn’t have a ready set of robust data from which to build AI-driven tools. In this aspect, dermatology is decades behind specialties like radiology, pathology, and ophthalmology, where clinicians have long been accumulating and storing images and other data in more standardized ways, Dr. Ko said.
“If you went to most dermatology practices and said, ‘Hey, let me learn from the data accumulated over the course of your 30-year practice to help us develop new tools,’” there may not be a whole lot there,” Dr. Ko said.
Beyond the start-up hurdles is the larger concern Dr. Ko shares with other dermatologists who work in this field, such as Dr. Daneshjou and Dr. Leachman. What would clinicians without much dermatology training and patients do with the readings from AI-driven tools and apps?
There would need to be significant research to show that such products actually help get people treated for skin diseases, including skin cancer.
Dr. Ko praised Google for being open about the stumbles with its efforts to use its AI tool for identifying diabetic retinopathy in a test in Thailand. Real-world hitches included poor Internet connections and poor image quality.
Developing reliable systems, processes, and workflows will be paramount for eventual widespread use of AI-driven tools, Dr. Ko said.
“It’s all those hidden things that are not sexy,” as are announcements about algorithms working about as well as clinicians in diagnosis, Dr. Ko said. “They don’t get the media attention, but they’re going to be make or break for AI, not just in our field but [for] AI in general.”
But he added that there also needs to be a recognition that AI-driven tools and products, even if somewhat imperfect, can help people get access to care.
In many cases, shortages of specialists prevent people from getting screened for treatable conditions such as skin cancer and retinopathy. The challenge is setting an appropriate standard to make sure that AI-driven products would help most patients in practice, without raising it so high that no such products emerge.
“There’s a risk of holding too high of a bar,” Dr. Ko said. “There is harm in not moving forward as well.”
A version of this article first appeared on Medscape.com.
.
Given that about 6.3 billion smartphones would soon be in use, this AI approach could provide a gateway for “low-cost universal access to vital diagnostic care,” wrote Justin M. Ko, MD, MBA, a dermatologist, and colleagues from Stanford (Calif.) University that included other dermatologists and engineers.
Dr. Ko and his coauthors described how they trained a computer system to identify both benign and cancerous skin lesions. They used an approach known as a convolutional neural network, often deployed for projects seeking to train computers to “see” through image analysis. They said that their test of this system found it to be on par with the performance of 21 board-certified dermatologists.
“This fast, scalable method is deployable on mobile devices and holds the potential for substantial clinical impact, including broadening the scope of primary care practice and augmenting clinical decision-making for dermatology specialists,” they wrote in their paper.
More than 6 years later, there are signs that companies are making progress toward moving skin checks using this technology into U.S. primary care settings – but only with devices that employ special tools.
It may prove tougher for companies to eventually secure the sign-off of the U.S. Food and Drug Administration for mobile apps intended to let consumers handle this task with smartphones.
Such tools would need to be proven highly accurate before release, because too many false positives mean that people would be needlessly exposed to biopsies, said Sancy A. Leachman, MD, PhD, director of the melanoma research program and chair of the department of dermatology at Oregon Health & Science University, Portland.
And false-negative readings would allow melanoma to advance and even be fatal, Dr. Leachman told this news organization.
Roxana Daneshjou, MD, PhD, a dermatologist at Stanford who has studied the promise and the pitfalls of AI in medicine, said that developers of a consumer skin-check app would need to know how people would react to their readings. That includes a good sense of how often they would appropriately seek medical care for a concerning reading. (She was not an author of the previously cited Nature paper but has published widely on AI.)
“The direct-to-consumer diagnostic space makes me nervous,” Dr. Daneshjou said in an interview. “In order to do it, you really need to have good studies in consumer populations prior to release. You need to show how effective it is with follow up.”
FDA shows interest – and reservations
As of July, the FDA had not yet given its okay for marketing of any consumer apps intended to help people detect signs of skin cancer, an agency spokesperson told this news organization.
To date, the agency has only cleared two AI-based products for this task, both meant to be used by dermatologists. And only one of these two products, Scibase’s Nevisense, remains in use in the United States. The other, MelaFind, has been discontinued. In 2017, Strata Skin Sciences said that the product did not win “a significant enough level of acceptance by dermatologists to justify the continued investment” in it. And the company said it notified the 90 owners of MelaFind devices in the United States that it would no longer support the device.
But another company, DermaSensor, said in a 2021 press release that it expects its AI-powered tool, also named DermaSensor, to be the “first ever FDA cleared or approved skin cancer detection device for primary care providers.”
The Miami-based firm said that the FDA had granted its product a “breakthrough” device designation. A breakthrough designation means that agency staff will offer extra help and guidance to companies in developing a product, because of its expected benefit for patients.
In a 2020 press release, 3Derm Systems, now owned by Digital Diagnostics, made a similar announcement about winning FDA breakthrough designation for an AI-powered tool intended to allow skin checks in primary care settings.
(The FDA generally does not comment on its reviews of experimental drugs and devices, but companies can do so. Several other companies have announced FDA breakthrough designations for AI-driven products intended to check for skin lesions, but these might be used in settings other than primary care.)
Both DermaSensor and Digital Diagnostics have chairs with notable track records for winning FDA approvals of other devices. DermaSensor’s Maurice Ferre, MD, also is the chairman of Insightec, which in 2016 won the first FDA approval for a device with a breakthrough designation device that uses ultrasound to treat tremors.
In 2018, the FDA allowed Digital Diagnostics, then called IDx, to introduce in the United States the first medical device using AI in primary care offices to check for signs of diabetic retinopathy. This product also had an FDA breakthrough designation. The executive chairman and founder of Digital Diagnostics is Michael Abramoff, MD, PhD, professor of engineering and ophthalmology at the University of Iowa, Iowa City. Dr. Abramoff and the team behind the AI tool for retinopathy, now called the LumineticsCore system, also scored a notable win with Medicare, which agreed to cover use of the product through a dedicated CPT code.
FDA draft guidance
The FDA has acknowledged the interest in broadening access to skin checks via AI.
This was a topic of discussion at a 2-day advisory committee meeting the FDA held last year. In April 2023, the FDA outlined some of its expectations for future regulation of skin-analyzing tools as part of a wide-ranging draft guidance document intended to aid companies in their efforts to develop products using a form of AI known as machine learning.
In the document, the FDA described how it might approach applications for “hypothetical” devices using this kind of AI, such as a special tool to help primary care clinicians identify lesions in need of further investigation. Such a product would use a specific camera for gathering data for its initial clearance, in the FDA’s hypothetical scenario.
The FDA staff offered technical suggestions about what the developer of this hypothetical device would have to do to extend its use to smartphones and tablets while keeping clinicians as the intended users.
Some of these expanded uses could fall within the bounds of the FDA’s initial clearance and thus not trigger a need for a new marketing submission, the agency said. But seeking to shift this hypothetical product to “patient-facing” use would require a new marketing submission to the FDA, the agency said.
In this scenario, a company would expect people to follow up with a dermatologist after receiving a report suggesting cancer. Thus, this kind of a change could expose patients to “many new, unconsidered risks,” the FDA said.
Reality check?
The state of current efforts to develop consumer apps for checking for skin cancer seems to be summarized well on the website for the MoleMapper. The app was developed by researchers at OHSU to help people track how their moles change over time.
“Mole Mapper is NOT designed to provide medical advice, professional diagnosis, opinion, or treatment. Currently, there is not enough data to develop an app that can diagnose melanoma, but if enough data is collected through Mole Mapper and shared with researchers, it may be possible in the future,” the app’s website says.
OHSU released MoleMapper as an iPhone app in 2015. The aim of this project was to help people track the moles on their skin while also fostering an experiment in “citizen science,” OHSU’s Dr. Leachman told this news organization.
OHSU researchers hoped that the digital images taken by members of the public on cell phones could one day be used to develop diagnostic algorithms for melanoma.
But around 2017, the MoleMapper team realized that they would not be able to create a diagnostic app at this time, Dr. Leachman explained. They could not collect enough data of adequate quality.
And by 2021, it was clear that they could not even develop a successful app to triage patients to assess who needs to be seen quickly. The amount of data required was, at this point, beyond what the team could collect, Dr. Leachman said in an interview.
That was a disappointment because the team had successfully completed the difficult task of creating a confidential pathway for collecting these images via both iPhones and smartphones run on Android.
“We thought if we built it, people would come, but that’s not what happened,” Dr. Leachman said. Many patients didn’t want their images used for research or would fail to follow up with details of biopsy reports. Sometimes images were not captured well enough to be of use.
“You need at least hundreds of thousands, if not millions, of data points that have been verified with pathologies, and nobody was giving us back that data. That was the reality,” Dr. Leachman said.
There were valuable lessons in that setback. The OHSU team now has a better grasp of the challenges of trying to build a data-collection system that could prove helpful in assessing skin lesions.
“If you don’t build it, you don’t know” what can go wrong, she said.
Dr. Leachman said other scientists who have worked on similar projects to build skin-analyzing apps have probably encountered the same difficulties, although they may not reveal these issues. “I think that a lot of people build these things and then they try to make it into something that it’s not,” she said.
In addition to the challenges with gathering images, dermatologists frequently need to rely on touch and other clues from in-person visits when diagnosing a suspicious lesion. “There’s something about seeing and feeling the skin in person that can’t be captured completely with an image,” Dr. Leachman said.
Public demand
Still, regulators must face the strong and immediate interest consumers have in using AI to check on moles and skin conditions, despite continuing questions about how well this approach might work.
In June, Google announced in a blog post that its Google Lens tool can help people research skin conditions.
“Just take a picture or upload a photo through Lens, and you’ll find visual matches to inform your search,” Google said in a blog post. “This feature also works if you’re not sure how to describe something else on your body, like a bump on your lip, a line on your nails or hair loss on your head. This feature is currently available in the U.S.”
Google also continues work on DermAssist, an app that’s intended to help people get personalized information about skin concerns using three photos. It is not currently publicly available, a Google spokesperson told this news organization.
Several skin-analyzing apps are already available in the Apple and Google Play stores. The British Association of Dermatologists last year issued a press release warning consumers that these apps may not be safe or effective and thus may put patients at risk for misdiagnosis.
“Unfortunately, AI-based apps which do not appear to meet regulatory requirements crop up more often than we would like,” the association said. “Additionally, the evidence to support the use of AI to diagnose skin conditions is weak which means that when it is used, it may not be safe or effective and it is possible that AI is putting patients at risk of misdiagnosis.”
Delicate and difficult balancing act
At this time, regulators, entrepreneurs, and the medical community face a delicate balancing act in considering how best to deploy AI in skin care, Dr. Ko said in an interview. (In addition to being one of the authors on the widely cited 2017 Nature paper mentioned above, Dr. Ko served until March as the initial chair of the American Academy of Dermatology’s Augmented Intelligence Committee.)
There are many solid reasons why there hasn’t been speedy progress to deploy AI in dermatology, as many envisioned a few years ago, Dr. Ko said.
Some of those reasons are specific to dermatology; this field doesn’t have a ready set of robust data from which to build AI-driven tools. In this aspect, dermatology is decades behind specialties like radiology, pathology, and ophthalmology, where clinicians have long been accumulating and storing images and other data in more standardized ways, Dr. Ko said.
“If you went to most dermatology practices and said, ‘Hey, let me learn from the data accumulated over the course of your 30-year practice to help us develop new tools,’” there may not be a whole lot there,” Dr. Ko said.
Beyond the start-up hurdles is the larger concern Dr. Ko shares with other dermatologists who work in this field, such as Dr. Daneshjou and Dr. Leachman. What would clinicians without much dermatology training and patients do with the readings from AI-driven tools and apps?
There would need to be significant research to show that such products actually help get people treated for skin diseases, including skin cancer.
Dr. Ko praised Google for being open about the stumbles with its efforts to use its AI tool for identifying diabetic retinopathy in a test in Thailand. Real-world hitches included poor Internet connections and poor image quality.
Developing reliable systems, processes, and workflows will be paramount for eventual widespread use of AI-driven tools, Dr. Ko said.
“It’s all those hidden things that are not sexy,” as are announcements about algorithms working about as well as clinicians in diagnosis, Dr. Ko said. “They don’t get the media attention, but they’re going to be make or break for AI, not just in our field but [for] AI in general.”
But he added that there also needs to be a recognition that AI-driven tools and products, even if somewhat imperfect, can help people get access to care.
In many cases, shortages of specialists prevent people from getting screened for treatable conditions such as skin cancer and retinopathy. The challenge is setting an appropriate standard to make sure that AI-driven products would help most patients in practice, without raising it so high that no such products emerge.
“There’s a risk of holding too high of a bar,” Dr. Ko said. “There is harm in not moving forward as well.”
A version of this article first appeared on Medscape.com.
.
Given that about 6.3 billion smartphones would soon be in use, this AI approach could provide a gateway for “low-cost universal access to vital diagnostic care,” wrote Justin M. Ko, MD, MBA, a dermatologist, and colleagues from Stanford (Calif.) University that included other dermatologists and engineers.
Dr. Ko and his coauthors described how they trained a computer system to identify both benign and cancerous skin lesions. They used an approach known as a convolutional neural network, often deployed for projects seeking to train computers to “see” through image analysis. They said that their test of this system found it to be on par with the performance of 21 board-certified dermatologists.
“This fast, scalable method is deployable on mobile devices and holds the potential for substantial clinical impact, including broadening the scope of primary care practice and augmenting clinical decision-making for dermatology specialists,” they wrote in their paper.
More than 6 years later, there are signs that companies are making progress toward moving skin checks using this technology into U.S. primary care settings – but only with devices that employ special tools.
It may prove tougher for companies to eventually secure the sign-off of the U.S. Food and Drug Administration for mobile apps intended to let consumers handle this task with smartphones.
Such tools would need to be proven highly accurate before release, because too many false positives mean that people would be needlessly exposed to biopsies, said Sancy A. Leachman, MD, PhD, director of the melanoma research program and chair of the department of dermatology at Oregon Health & Science University, Portland.
And false-negative readings would allow melanoma to advance and even be fatal, Dr. Leachman told this news organization.
Roxana Daneshjou, MD, PhD, a dermatologist at Stanford who has studied the promise and the pitfalls of AI in medicine, said that developers of a consumer skin-check app would need to know how people would react to their readings. That includes a good sense of how often they would appropriately seek medical care for a concerning reading. (She was not an author of the previously cited Nature paper but has published widely on AI.)
“The direct-to-consumer diagnostic space makes me nervous,” Dr. Daneshjou said in an interview. “In order to do it, you really need to have good studies in consumer populations prior to release. You need to show how effective it is with follow up.”
FDA shows interest – and reservations
As of July, the FDA had not yet given its okay for marketing of any consumer apps intended to help people detect signs of skin cancer, an agency spokesperson told this news organization.
To date, the agency has only cleared two AI-based products for this task, both meant to be used by dermatologists. And only one of these two products, Scibase’s Nevisense, remains in use in the United States. The other, MelaFind, has been discontinued. In 2017, Strata Skin Sciences said that the product did not win “a significant enough level of acceptance by dermatologists to justify the continued investment” in it. And the company said it notified the 90 owners of MelaFind devices in the United States that it would no longer support the device.
But another company, DermaSensor, said in a 2021 press release that it expects its AI-powered tool, also named DermaSensor, to be the “first ever FDA cleared or approved skin cancer detection device for primary care providers.”
The Miami-based firm said that the FDA had granted its product a “breakthrough” device designation. A breakthrough designation means that agency staff will offer extra help and guidance to companies in developing a product, because of its expected benefit for patients.
In a 2020 press release, 3Derm Systems, now owned by Digital Diagnostics, made a similar announcement about winning FDA breakthrough designation for an AI-powered tool intended to allow skin checks in primary care settings.
(The FDA generally does not comment on its reviews of experimental drugs and devices, but companies can do so. Several other companies have announced FDA breakthrough designations for AI-driven products intended to check for skin lesions, but these might be used in settings other than primary care.)
Both DermaSensor and Digital Diagnostics have chairs with notable track records for winning FDA approvals of other devices. DermaSensor’s Maurice Ferre, MD, also is the chairman of Insightec, which in 2016 won the first FDA approval for a device with a breakthrough designation device that uses ultrasound to treat tremors.
In 2018, the FDA allowed Digital Diagnostics, then called IDx, to introduce in the United States the first medical device using AI in primary care offices to check for signs of diabetic retinopathy. This product also had an FDA breakthrough designation. The executive chairman and founder of Digital Diagnostics is Michael Abramoff, MD, PhD, professor of engineering and ophthalmology at the University of Iowa, Iowa City. Dr. Abramoff and the team behind the AI tool for retinopathy, now called the LumineticsCore system, also scored a notable win with Medicare, which agreed to cover use of the product through a dedicated CPT code.
FDA draft guidance
The FDA has acknowledged the interest in broadening access to skin checks via AI.
This was a topic of discussion at a 2-day advisory committee meeting the FDA held last year. In April 2023, the FDA outlined some of its expectations for future regulation of skin-analyzing tools as part of a wide-ranging draft guidance document intended to aid companies in their efforts to develop products using a form of AI known as machine learning.
In the document, the FDA described how it might approach applications for “hypothetical” devices using this kind of AI, such as a special tool to help primary care clinicians identify lesions in need of further investigation. Such a product would use a specific camera for gathering data for its initial clearance, in the FDA’s hypothetical scenario.
The FDA staff offered technical suggestions about what the developer of this hypothetical device would have to do to extend its use to smartphones and tablets while keeping clinicians as the intended users.
Some of these expanded uses could fall within the bounds of the FDA’s initial clearance and thus not trigger a need for a new marketing submission, the agency said. But seeking to shift this hypothetical product to “patient-facing” use would require a new marketing submission to the FDA, the agency said.
In this scenario, a company would expect people to follow up with a dermatologist after receiving a report suggesting cancer. Thus, this kind of a change could expose patients to “many new, unconsidered risks,” the FDA said.
Reality check?
The state of current efforts to develop consumer apps for checking for skin cancer seems to be summarized well on the website for the MoleMapper. The app was developed by researchers at OHSU to help people track how their moles change over time.
“Mole Mapper is NOT designed to provide medical advice, professional diagnosis, opinion, or treatment. Currently, there is not enough data to develop an app that can diagnose melanoma, but if enough data is collected through Mole Mapper and shared with researchers, it may be possible in the future,” the app’s website says.
OHSU released MoleMapper as an iPhone app in 2015. The aim of this project was to help people track the moles on their skin while also fostering an experiment in “citizen science,” OHSU’s Dr. Leachman told this news organization.
OHSU researchers hoped that the digital images taken by members of the public on cell phones could one day be used to develop diagnostic algorithms for melanoma.
But around 2017, the MoleMapper team realized that they would not be able to create a diagnostic app at this time, Dr. Leachman explained. They could not collect enough data of adequate quality.
And by 2021, it was clear that they could not even develop a successful app to triage patients to assess who needs to be seen quickly. The amount of data required was, at this point, beyond what the team could collect, Dr. Leachman said in an interview.
That was a disappointment because the team had successfully completed the difficult task of creating a confidential pathway for collecting these images via both iPhones and smartphones run on Android.
“We thought if we built it, people would come, but that’s not what happened,” Dr. Leachman said. Many patients didn’t want their images used for research or would fail to follow up with details of biopsy reports. Sometimes images were not captured well enough to be of use.
“You need at least hundreds of thousands, if not millions, of data points that have been verified with pathologies, and nobody was giving us back that data. That was the reality,” Dr. Leachman said.
There were valuable lessons in that setback. The OHSU team now has a better grasp of the challenges of trying to build a data-collection system that could prove helpful in assessing skin lesions.
“If you don’t build it, you don’t know” what can go wrong, she said.
Dr. Leachman said other scientists who have worked on similar projects to build skin-analyzing apps have probably encountered the same difficulties, although they may not reveal these issues. “I think that a lot of people build these things and then they try to make it into something that it’s not,” she said.
In addition to the challenges with gathering images, dermatologists frequently need to rely on touch and other clues from in-person visits when diagnosing a suspicious lesion. “There’s something about seeing and feeling the skin in person that can’t be captured completely with an image,” Dr. Leachman said.
Public demand
Still, regulators must face the strong and immediate interest consumers have in using AI to check on moles and skin conditions, despite continuing questions about how well this approach might work.
In June, Google announced in a blog post that its Google Lens tool can help people research skin conditions.
“Just take a picture or upload a photo through Lens, and you’ll find visual matches to inform your search,” Google said in a blog post. “This feature also works if you’re not sure how to describe something else on your body, like a bump on your lip, a line on your nails or hair loss on your head. This feature is currently available in the U.S.”
Google also continues work on DermAssist, an app that’s intended to help people get personalized information about skin concerns using three photos. It is not currently publicly available, a Google spokesperson told this news organization.
Several skin-analyzing apps are already available in the Apple and Google Play stores. The British Association of Dermatologists last year issued a press release warning consumers that these apps may not be safe or effective and thus may put patients at risk for misdiagnosis.
“Unfortunately, AI-based apps which do not appear to meet regulatory requirements crop up more often than we would like,” the association said. “Additionally, the evidence to support the use of AI to diagnose skin conditions is weak which means that when it is used, it may not be safe or effective and it is possible that AI is putting patients at risk of misdiagnosis.”
Delicate and difficult balancing act
At this time, regulators, entrepreneurs, and the medical community face a delicate balancing act in considering how best to deploy AI in skin care, Dr. Ko said in an interview. (In addition to being one of the authors on the widely cited 2017 Nature paper mentioned above, Dr. Ko served until March as the initial chair of the American Academy of Dermatology’s Augmented Intelligence Committee.)
There are many solid reasons why there hasn’t been speedy progress to deploy AI in dermatology, as many envisioned a few years ago, Dr. Ko said.
Some of those reasons are specific to dermatology; this field doesn’t have a ready set of robust data from which to build AI-driven tools. In this aspect, dermatology is decades behind specialties like radiology, pathology, and ophthalmology, where clinicians have long been accumulating and storing images and other data in more standardized ways, Dr. Ko said.
“If you went to most dermatology practices and said, ‘Hey, let me learn from the data accumulated over the course of your 30-year practice to help us develop new tools,’” there may not be a whole lot there,” Dr. Ko said.
Beyond the start-up hurdles is the larger concern Dr. Ko shares with other dermatologists who work in this field, such as Dr. Daneshjou and Dr. Leachman. What would clinicians without much dermatology training and patients do with the readings from AI-driven tools and apps?
There would need to be significant research to show that such products actually help get people treated for skin diseases, including skin cancer.
Dr. Ko praised Google for being open about the stumbles with its efforts to use its AI tool for identifying diabetic retinopathy in a test in Thailand. Real-world hitches included poor Internet connections and poor image quality.
Developing reliable systems, processes, and workflows will be paramount for eventual widespread use of AI-driven tools, Dr. Ko said.
“It’s all those hidden things that are not sexy,” as are announcements about algorithms working about as well as clinicians in diagnosis, Dr. Ko said. “They don’t get the media attention, but they’re going to be make or break for AI, not just in our field but [for] AI in general.”
But he added that there also needs to be a recognition that AI-driven tools and products, even if somewhat imperfect, can help people get access to care.
In many cases, shortages of specialists prevent people from getting screened for treatable conditions such as skin cancer and retinopathy. The challenge is setting an appropriate standard to make sure that AI-driven products would help most patients in practice, without raising it so high that no such products emerge.
“There’s a risk of holding too high of a bar,” Dr. Ko said. “There is harm in not moving forward as well.”
A version of this article first appeared on Medscape.com.
Rising patient costs tied to private equity ownership
The report was a collaboration of University of California, Berkeley, staff and researchers from two nonprofits, the American Antitrust Institute and the Washington Center for Equitable Growth. It provides “convincing evidence that incentives to put profits before patients have grown stronger with an increase in private equity ownership of physician practices,” lead author Richard Scheffler, PhD, of UC Berkeley said in a statement.
The report also noted that private equity acquisitions of physician groups have risen sixfold in just a decade, increasing from 75 deals in 2012 to 484 deals in 2021.
Separately, the American Medical Association earlier released a separate report on trends in physician practice arrangements, finding that the percentage of physicians working in private equity–owned groups was 4.5% in 2022, the same as in its previous 2020 report. The share of physicians working in private practices fell by 13 percentage points from 60.1% to 46.7% between 2012 and 2022, the AMA reported.
The Berkeley report and the AMA update come amid rising concerns about the effects of the decline of independent physician practices. The U.S. Senate Finance Committee, which oversees most federal health spending, held a June hearing examining the causes and consequences of increased corporate ownership in health care, including a look at physician practices.
“It’s increasingly clear that consolidation in health care is not lowering costs or increasing the quality of Americans’ health care,” Senate Finance Chairman Ron Wyden (D-Ore.) said in an email. “For private equity in health care in particular, there needs to be more transparency around ownership so the effect on these business relationships can be better understood.”
Federal and state agencies do not generally track acquisitions of physician practices.
The UC Berkeley report impressively documents the rising influence of private equity in health care, for which it’s tough to find good data, said Karen Joynt Maddox, MD, MPH, of Washington University in St. Louis. Dr. Maddox, a cardiologist and policy researcher who also has studied the effects of consolidation in health care, examined the new report at the request of this news organization.
“They did a great job with the data,” Dr. Maddox said. “One of the big issues around private equity, and in general, ‘corporatization’ and consolidation of health care, is that there’s not a great way to track ownership changes. It’s really difficult to study.”
Dr. Scheffler and colleagues used data from the commercial firm PitchBook to identify acquisitions of physician practices by private equity firms. They consulted IQVIA’s physician databases – OneKey and SK&A Office-Based Physicians Database – to learn about the location, size, and specialties of acquired practices. They also used data from the nonprofit Health Care Cost Institute, which tracks commercial health plan claims, to assess how private equity acquisitions affected prices.
The researchers then matched the findings for practices acquired by private equity firms from 2015 to 2021 against those for comparable physician practices that remained independent from 2012 to 2021.
The authors then tied private-equity ownership to the following price increases:
- Gastroenterology (14%; 95% confidence interval, 7.9%-20.4%
- Oncology (16.4%; 95% CI, 5.5%-28.4%)
- Dermatology (4.0%; 95% CI, 1%-7.1%)
- Ob.gyn. (8.8%; 95% CI, 3.8%-14%)
- Ophthalmology (8.7%; 95% CI, 5.1%-12.3%)
- Radiology (8.2%; 95% CI, 0.8%-16.1%)
- Orthopedics (7.1%; 95% CI, 2.2%-12.3%)
- Primary care (4.1%; 95% CI, 1.3%-7%)
The analysis also found higher prices for cardiology (8.7%; 95% CI, –6.4% to 26.1%) and urology (4.2%; 95% CI, –2.3% to 11.1%), but neither of these findings was statistically significant, one of the authors, Daniel R. Arnold, PhD, of UC Berkeley, said in an email. This was most likely caused by smaller sample sizes for these fields.
Factors driving consolidation
The two reports and the Senate Finance consolidation hearing raised similar issues, including calls to look at the factors driving more physicians out of independent practice, including Medicare reimbursement that may not keep up with rising inflation.
The Berkeley report authors called for Congress to add a broad inflation component to the Medicare physician fee schedule. It also called on Congress to add cases where Medicare, the biggest U.S. purchaser of health care, pays less for services when performed in independent practices than in hospital-affiliated ones.
Shawn Martin, executive vice president and CEO of the American Academy of Family Physicians, said his group appreciates how the report from UC Berkeley and nonprofit groups echoed recommendations many clinicians have made, including the call for a broad inflation adjustment for the fee schedule.
“To move the needle forward, Congress must advance site-neutral payment policies while also addressing the administrative requirements that take physicians away from the important work of caring for patients,” Mr. Martin said in an email.
Arnold Ventures provided funding for the report, which was a joint project of the American Antitrust Institute, the Nicholas C. Petris Center on Health Care Markets and Consumer Welfare, UC Berkeley, and the Washington Center for Equitable Growth.
A version of this article appeared on Medscape.com.
The report was a collaboration of University of California, Berkeley, staff and researchers from two nonprofits, the American Antitrust Institute and the Washington Center for Equitable Growth. It provides “convincing evidence that incentives to put profits before patients have grown stronger with an increase in private equity ownership of physician practices,” lead author Richard Scheffler, PhD, of UC Berkeley said in a statement.
The report also noted that private equity acquisitions of physician groups have risen sixfold in just a decade, increasing from 75 deals in 2012 to 484 deals in 2021.
Separately, the American Medical Association earlier released a separate report on trends in physician practice arrangements, finding that the percentage of physicians working in private equity–owned groups was 4.5% in 2022, the same as in its previous 2020 report. The share of physicians working in private practices fell by 13 percentage points from 60.1% to 46.7% between 2012 and 2022, the AMA reported.
The Berkeley report and the AMA update come amid rising concerns about the effects of the decline of independent physician practices. The U.S. Senate Finance Committee, which oversees most federal health spending, held a June hearing examining the causes and consequences of increased corporate ownership in health care, including a look at physician practices.
“It’s increasingly clear that consolidation in health care is not lowering costs or increasing the quality of Americans’ health care,” Senate Finance Chairman Ron Wyden (D-Ore.) said in an email. “For private equity in health care in particular, there needs to be more transparency around ownership so the effect on these business relationships can be better understood.”
Federal and state agencies do not generally track acquisitions of physician practices.
The UC Berkeley report impressively documents the rising influence of private equity in health care, for which it’s tough to find good data, said Karen Joynt Maddox, MD, MPH, of Washington University in St. Louis. Dr. Maddox, a cardiologist and policy researcher who also has studied the effects of consolidation in health care, examined the new report at the request of this news organization.
“They did a great job with the data,” Dr. Maddox said. “One of the big issues around private equity, and in general, ‘corporatization’ and consolidation of health care, is that there’s not a great way to track ownership changes. It’s really difficult to study.”
Dr. Scheffler and colleagues used data from the commercial firm PitchBook to identify acquisitions of physician practices by private equity firms. They consulted IQVIA’s physician databases – OneKey and SK&A Office-Based Physicians Database – to learn about the location, size, and specialties of acquired practices. They also used data from the nonprofit Health Care Cost Institute, which tracks commercial health plan claims, to assess how private equity acquisitions affected prices.
The researchers then matched the findings for practices acquired by private equity firms from 2015 to 2021 against those for comparable physician practices that remained independent from 2012 to 2021.
The authors then tied private-equity ownership to the following price increases:
- Gastroenterology (14%; 95% confidence interval, 7.9%-20.4%
- Oncology (16.4%; 95% CI, 5.5%-28.4%)
- Dermatology (4.0%; 95% CI, 1%-7.1%)
- Ob.gyn. (8.8%; 95% CI, 3.8%-14%)
- Ophthalmology (8.7%; 95% CI, 5.1%-12.3%)
- Radiology (8.2%; 95% CI, 0.8%-16.1%)
- Orthopedics (7.1%; 95% CI, 2.2%-12.3%)
- Primary care (4.1%; 95% CI, 1.3%-7%)
The analysis also found higher prices for cardiology (8.7%; 95% CI, –6.4% to 26.1%) and urology (4.2%; 95% CI, –2.3% to 11.1%), but neither of these findings was statistically significant, one of the authors, Daniel R. Arnold, PhD, of UC Berkeley, said in an email. This was most likely caused by smaller sample sizes for these fields.
Factors driving consolidation
The two reports and the Senate Finance consolidation hearing raised similar issues, including calls to look at the factors driving more physicians out of independent practice, including Medicare reimbursement that may not keep up with rising inflation.
The Berkeley report authors called for Congress to add a broad inflation component to the Medicare physician fee schedule. It also called on Congress to add cases where Medicare, the biggest U.S. purchaser of health care, pays less for services when performed in independent practices than in hospital-affiliated ones.
Shawn Martin, executive vice president and CEO of the American Academy of Family Physicians, said his group appreciates how the report from UC Berkeley and nonprofit groups echoed recommendations many clinicians have made, including the call for a broad inflation adjustment for the fee schedule.
“To move the needle forward, Congress must advance site-neutral payment policies while also addressing the administrative requirements that take physicians away from the important work of caring for patients,” Mr. Martin said in an email.
Arnold Ventures provided funding for the report, which was a joint project of the American Antitrust Institute, the Nicholas C. Petris Center on Health Care Markets and Consumer Welfare, UC Berkeley, and the Washington Center for Equitable Growth.
A version of this article appeared on Medscape.com.
The report was a collaboration of University of California, Berkeley, staff and researchers from two nonprofits, the American Antitrust Institute and the Washington Center for Equitable Growth. It provides “convincing evidence that incentives to put profits before patients have grown stronger with an increase in private equity ownership of physician practices,” lead author Richard Scheffler, PhD, of UC Berkeley said in a statement.
The report also noted that private equity acquisitions of physician groups have risen sixfold in just a decade, increasing from 75 deals in 2012 to 484 deals in 2021.
Separately, the American Medical Association earlier released a separate report on trends in physician practice arrangements, finding that the percentage of physicians working in private equity–owned groups was 4.5% in 2022, the same as in its previous 2020 report. The share of physicians working in private practices fell by 13 percentage points from 60.1% to 46.7% between 2012 and 2022, the AMA reported.
The Berkeley report and the AMA update come amid rising concerns about the effects of the decline of independent physician practices. The U.S. Senate Finance Committee, which oversees most federal health spending, held a June hearing examining the causes and consequences of increased corporate ownership in health care, including a look at physician practices.
“It’s increasingly clear that consolidation in health care is not lowering costs or increasing the quality of Americans’ health care,” Senate Finance Chairman Ron Wyden (D-Ore.) said in an email. “For private equity in health care in particular, there needs to be more transparency around ownership so the effect on these business relationships can be better understood.”
Federal and state agencies do not generally track acquisitions of physician practices.
The UC Berkeley report impressively documents the rising influence of private equity in health care, for which it’s tough to find good data, said Karen Joynt Maddox, MD, MPH, of Washington University in St. Louis. Dr. Maddox, a cardiologist and policy researcher who also has studied the effects of consolidation in health care, examined the new report at the request of this news organization.
“They did a great job with the data,” Dr. Maddox said. “One of the big issues around private equity, and in general, ‘corporatization’ and consolidation of health care, is that there’s not a great way to track ownership changes. It’s really difficult to study.”
Dr. Scheffler and colleagues used data from the commercial firm PitchBook to identify acquisitions of physician practices by private equity firms. They consulted IQVIA’s physician databases – OneKey and SK&A Office-Based Physicians Database – to learn about the location, size, and specialties of acquired practices. They also used data from the nonprofit Health Care Cost Institute, which tracks commercial health plan claims, to assess how private equity acquisitions affected prices.
The researchers then matched the findings for practices acquired by private equity firms from 2015 to 2021 against those for comparable physician practices that remained independent from 2012 to 2021.
The authors then tied private-equity ownership to the following price increases:
- Gastroenterology (14%; 95% confidence interval, 7.9%-20.4%
- Oncology (16.4%; 95% CI, 5.5%-28.4%)
- Dermatology (4.0%; 95% CI, 1%-7.1%)
- Ob.gyn. (8.8%; 95% CI, 3.8%-14%)
- Ophthalmology (8.7%; 95% CI, 5.1%-12.3%)
- Radiology (8.2%; 95% CI, 0.8%-16.1%)
- Orthopedics (7.1%; 95% CI, 2.2%-12.3%)
- Primary care (4.1%; 95% CI, 1.3%-7%)
The analysis also found higher prices for cardiology (8.7%; 95% CI, –6.4% to 26.1%) and urology (4.2%; 95% CI, –2.3% to 11.1%), but neither of these findings was statistically significant, one of the authors, Daniel R. Arnold, PhD, of UC Berkeley, said in an email. This was most likely caused by smaller sample sizes for these fields.
Factors driving consolidation
The two reports and the Senate Finance consolidation hearing raised similar issues, including calls to look at the factors driving more physicians out of independent practice, including Medicare reimbursement that may not keep up with rising inflation.
The Berkeley report authors called for Congress to add a broad inflation component to the Medicare physician fee schedule. It also called on Congress to add cases where Medicare, the biggest U.S. purchaser of health care, pays less for services when performed in independent practices than in hospital-affiliated ones.
Shawn Martin, executive vice president and CEO of the American Academy of Family Physicians, said his group appreciates how the report from UC Berkeley and nonprofit groups echoed recommendations many clinicians have made, including the call for a broad inflation adjustment for the fee schedule.
“To move the needle forward, Congress must advance site-neutral payment policies while also addressing the administrative requirements that take physicians away from the important work of caring for patients,” Mr. Martin said in an email.
Arnold Ventures provided funding for the report, which was a joint project of the American Antitrust Institute, the Nicholas C. Petris Center on Health Care Markets and Consumer Welfare, UC Berkeley, and the Washington Center for Equitable Growth.
A version of this article appeared on Medscape.com.
U.S. mammogram update sparks concern, reignites debates
, while also renewing debates about the timing of these tests and the screening approaches used.
The U.S. Preventive Services Task Force is currently finalizing an update to its recommendations on breast cancer screening. In May, the task force released a proposed update that dropped the initial age for routine mammogram screening from 50 to 40.
The task force intends to give a “B” rating to this recommendation, which covers screening every other year up to age 74 for women deemed average risk for breast cancer.
The task force’s rating carries clout, A. Mark Fendrick, MD, director of the Value-Based Insurance Design at the University of Michigan, Ann Arbor, said in an interview.
For one, the Affordable Care Act requires that private insurers cover services that get top A or B marks from USPSTF without charging copays.
However, Dr. Fendrick noted, such coverage does not necessarily apply to follow-up testing when a routine mammogram comes back with a positive finding. The expense of follow-up testing may deter some women from seeking follow-up diagnostic imaging or biopsies after an abnormal result on a screening mammogram.
A recent analysis in JAMA Network Open found that women facing higher anticipated out-of-pocket costs for breast cancer diagnostic tests, based on their health insurance plan, were less likely to get that follow-up screening. For instance, the use of breast MRI decreased by nearly 24% between patients undergoing subsequent diagnostic testing in plans with the lowest out-of-pocket costs vs. those with the highest.
“The study’s central finding that some women who have an abnormal result on a mammogram may not get appropriate follow-up because of cost is worrisome,” said Dr. Fendrick and Ilana B. Richman, MD, MHS, in an accompanying commentary to the JAMA analysis. “On an individual level, high out-of-pocket costs may directly contribute to worse health outcomes or require individuals to use scarce financial resources that may otherwise be used for critical items such as food or rent.”
For patients to fully benefit from early detection, the USPSTF would also need to make clear that follow-up diagnostic mammograms are covered, Dr. Fendrick said.
The ongoing debates
Concerns over the costs of potential follow-up tests are not the only issues experts have highlighted since USPSTF released its updated draft guidance on screening mammography.
The task force’s proposed update has also reignited questions and uncertainties surrounding when to screen, how often, and what types are best.
When it comes to frequency, the major organizations that provide screening guidance don’t see eye to eye. The USPSTF recommends breast cancer screening every other year, while the American College of Radiology recommends screening every year because that approach leads to saves “the most lives.”
At this time, the American College of Obstetricians and Gynecologists guidance currently teeters in the middle, suggesting either annual or biennial screening and highlighting the pros and cons of either approach. According to ACOG, “annual screening intervals appear to result in the least number of breast cancer deaths, particularly in younger women, but at the cost of additional callbacks and biopsies.”
When to begin screening represents another point of contention. While some experts, such as ACOG, agree with the task force’s decision to lower the screening start age to 40, others point to the need for greater nuance on setting the appropriate screening age. The main issue: the task force’s draft sets a uniform age to begin screening, but the risk for breast cancer and breast cancer mortality is not uniform across different racial and ethnic groups.
A recent study published in JAMA Network Open found that, among women aged 40-49, breast cancer mortality was highest among Black women (27 deaths per 100,000 person-years) followed by White women (15 deaths per 100,000 person-years). Based on a recommended screening age of 50, the authors suggested that Black women should start screening at age 42, whereas White women could start at 51.
“These findings suggest that health policy makers and clinicians could consider an alternative, race and ethnicity–adapted approach in which Black female patients start screening earlier,” writes Tianhui Chen, PhD, of China’s Zhejiang Cancer Hospital and coauthor of the study.
Weighing in on the guidance, the nonprofit National Center for Health Research urged the task force to consider suggesting different screening schedules based on race and ethnicity data. That would mean the recommendation to start at age 40 should only apply to Black women and other groups with higher-than-average risk for breast cancer at a younger age.
“Women are capable of understanding why the age to start mammography screening may be different for women with different risk factors,” the National Center for Health Research wrote in a comment to USPSTF, provided to this news organization by request. “What is confusing is when some physician groups recommend annual mammograms for all women starting at age 40, even though the data do not support that recommendation.”
While the ACR agreed with the task force’s recommendation to lower the screening age, the organization suggested starting risk assessments based on racial variations in breast cancer incidence and death even earlier. Specifically, the ACR recommended that high-risk groups, such as Black women, get risk assessments by age 25 to determine whether mammography before age 40 is needed.
Screening options for women with dense breasts may be some of the most challenging to weigh. Having dense breasts increases an individual’s risk for breast cancer, and mammography alone is not as effective at identifying breast cancer among these women. However, the evidence on the benefits vs. harms of additional screening beyond mammography remains mixed.
As a result, the task force decided to maintain its “I” grade on additional screening beyond mammography for these women – a grade that indicates insufficient evidence to determine the benefits and harms for a service.
The task force largely based its decision on the findings of two key reports. One report from the Cancer Intervention and Surveillance Modeling Network, which modeled potential outcomes of different screening strategies, indicated that extra screening might reduce breast cancer mortality in those with dense breasts, but at a cost of more false-positive reports.
The second report, a review from the Kaiser Permanente Evidence-based Practice Center, reaffirmed the benefits of routine mammography for reducing deaths from breast cancer, but found no solid evidence that different strategies – including supplemental screening in women with denser breasts – lowered breast cancer mortality or the risk of progression to advanced cancer. Further studies may show which approaches work best to reduce breast cancer deaths, the report said.
In this instance, ACOG agreed with USPSTF: “Based on the lack of data, ACOG does not recommend routine use of alternative or adjunctive tests to screening mammography in women with dense breasts who are asymptomatic and have no additional risk factors.”
Women with dense breasts should still be encouraged to receive regular screening mammography, even if the results they get may not be as accurate as those for women with less dense breasts, said Diana L. Miglioretti, PhD, of the University of California, Davis, who worked on a report for the USPSTF guidelines.
What’s next?
Despite ongoing debate and uncertainties surrounding some breast screening guidance, support for ending copay requirements for follow-up tests after a positive mammogram finding is widespread.
According to Dr. Fendrick, the USPSTF should expand coverage of follow-up testing after a positive mammogram to ensure people receive routine screening and any necessary diagnostic tests, as it did with colon cancer.
Before 2021, patients could face high costs for a colonoscopy following a positive stool-based Cologuard test. But in 2021, the USPSTF said that positive results on stool-based tests would require follow-up with colonoscopy, defining this follow-up as part of the screening benefit. In 2022, Medicare followed by setting a policy that ended the copay for these follow-up colonoscopies.
For breast screening, there are efforts underway in Congress to end copays for breast screening. In May, Rep. Rosa DeLauro (D-Conn.) introduced a bill, the Find It Early Act, that would require both private and government insurers to cover the out-of-pocket costs for many women receiving screening with ultrasound and MRI.
When the USPSTF finalizes its breast screening guidelines, the recommendations will be woven into discussions between primary care physicians and patients about breast cancer screening.
As guidelines and evidence evolve, “we’re learning to adjust” and communicate these changes to patients, said Tochi Iroku-Malize, MD, president of the American Academy of Family Physicians.
However, gaps in the guidance will leave some open-ended questions about optimal screening practices and how much screening may cost.
Given that, Dr. Iroku-Malize takes many factors into account when discussing screening options with her patients. Based on the new information and the patient’s information, she said she will tell her patients, “We’re going to adjust our guidance as to what you need.”
A version of this article first appeared on Medscape.com.
, while also renewing debates about the timing of these tests and the screening approaches used.
The U.S. Preventive Services Task Force is currently finalizing an update to its recommendations on breast cancer screening. In May, the task force released a proposed update that dropped the initial age for routine mammogram screening from 50 to 40.
The task force intends to give a “B” rating to this recommendation, which covers screening every other year up to age 74 for women deemed average risk for breast cancer.
The task force’s rating carries clout, A. Mark Fendrick, MD, director of the Value-Based Insurance Design at the University of Michigan, Ann Arbor, said in an interview.
For one, the Affordable Care Act requires that private insurers cover services that get top A or B marks from USPSTF without charging copays.
However, Dr. Fendrick noted, such coverage does not necessarily apply to follow-up testing when a routine mammogram comes back with a positive finding. The expense of follow-up testing may deter some women from seeking follow-up diagnostic imaging or biopsies after an abnormal result on a screening mammogram.
A recent analysis in JAMA Network Open found that women facing higher anticipated out-of-pocket costs for breast cancer diagnostic tests, based on their health insurance plan, were less likely to get that follow-up screening. For instance, the use of breast MRI decreased by nearly 24% between patients undergoing subsequent diagnostic testing in plans with the lowest out-of-pocket costs vs. those with the highest.
“The study’s central finding that some women who have an abnormal result on a mammogram may not get appropriate follow-up because of cost is worrisome,” said Dr. Fendrick and Ilana B. Richman, MD, MHS, in an accompanying commentary to the JAMA analysis. “On an individual level, high out-of-pocket costs may directly contribute to worse health outcomes or require individuals to use scarce financial resources that may otherwise be used for critical items such as food or rent.”
For patients to fully benefit from early detection, the USPSTF would also need to make clear that follow-up diagnostic mammograms are covered, Dr. Fendrick said.
The ongoing debates
Concerns over the costs of potential follow-up tests are not the only issues experts have highlighted since USPSTF released its updated draft guidance on screening mammography.
The task force’s proposed update has also reignited questions and uncertainties surrounding when to screen, how often, and what types are best.
When it comes to frequency, the major organizations that provide screening guidance don’t see eye to eye. The USPSTF recommends breast cancer screening every other year, while the American College of Radiology recommends screening every year because that approach leads to saves “the most lives.”
At this time, the American College of Obstetricians and Gynecologists guidance currently teeters in the middle, suggesting either annual or biennial screening and highlighting the pros and cons of either approach. According to ACOG, “annual screening intervals appear to result in the least number of breast cancer deaths, particularly in younger women, but at the cost of additional callbacks and biopsies.”
When to begin screening represents another point of contention. While some experts, such as ACOG, agree with the task force’s decision to lower the screening start age to 40, others point to the need for greater nuance on setting the appropriate screening age. The main issue: the task force’s draft sets a uniform age to begin screening, but the risk for breast cancer and breast cancer mortality is not uniform across different racial and ethnic groups.
A recent study published in JAMA Network Open found that, among women aged 40-49, breast cancer mortality was highest among Black women (27 deaths per 100,000 person-years) followed by White women (15 deaths per 100,000 person-years). Based on a recommended screening age of 50, the authors suggested that Black women should start screening at age 42, whereas White women could start at 51.
“These findings suggest that health policy makers and clinicians could consider an alternative, race and ethnicity–adapted approach in which Black female patients start screening earlier,” writes Tianhui Chen, PhD, of China’s Zhejiang Cancer Hospital and coauthor of the study.
Weighing in on the guidance, the nonprofit National Center for Health Research urged the task force to consider suggesting different screening schedules based on race and ethnicity data. That would mean the recommendation to start at age 40 should only apply to Black women and other groups with higher-than-average risk for breast cancer at a younger age.
“Women are capable of understanding why the age to start mammography screening may be different for women with different risk factors,” the National Center for Health Research wrote in a comment to USPSTF, provided to this news organization by request. “What is confusing is when some physician groups recommend annual mammograms for all women starting at age 40, even though the data do not support that recommendation.”
While the ACR agreed with the task force’s recommendation to lower the screening age, the organization suggested starting risk assessments based on racial variations in breast cancer incidence and death even earlier. Specifically, the ACR recommended that high-risk groups, such as Black women, get risk assessments by age 25 to determine whether mammography before age 40 is needed.
Screening options for women with dense breasts may be some of the most challenging to weigh. Having dense breasts increases an individual’s risk for breast cancer, and mammography alone is not as effective at identifying breast cancer among these women. However, the evidence on the benefits vs. harms of additional screening beyond mammography remains mixed.
As a result, the task force decided to maintain its “I” grade on additional screening beyond mammography for these women – a grade that indicates insufficient evidence to determine the benefits and harms for a service.
The task force largely based its decision on the findings of two key reports. One report from the Cancer Intervention and Surveillance Modeling Network, which modeled potential outcomes of different screening strategies, indicated that extra screening might reduce breast cancer mortality in those with dense breasts, but at a cost of more false-positive reports.
The second report, a review from the Kaiser Permanente Evidence-based Practice Center, reaffirmed the benefits of routine mammography for reducing deaths from breast cancer, but found no solid evidence that different strategies – including supplemental screening in women with denser breasts – lowered breast cancer mortality or the risk of progression to advanced cancer. Further studies may show which approaches work best to reduce breast cancer deaths, the report said.
In this instance, ACOG agreed with USPSTF: “Based on the lack of data, ACOG does not recommend routine use of alternative or adjunctive tests to screening mammography in women with dense breasts who are asymptomatic and have no additional risk factors.”
Women with dense breasts should still be encouraged to receive regular screening mammography, even if the results they get may not be as accurate as those for women with less dense breasts, said Diana L. Miglioretti, PhD, of the University of California, Davis, who worked on a report for the USPSTF guidelines.
What’s next?
Despite ongoing debate and uncertainties surrounding some breast screening guidance, support for ending copay requirements for follow-up tests after a positive mammogram finding is widespread.
According to Dr. Fendrick, the USPSTF should expand coverage of follow-up testing after a positive mammogram to ensure people receive routine screening and any necessary diagnostic tests, as it did with colon cancer.
Before 2021, patients could face high costs for a colonoscopy following a positive stool-based Cologuard test. But in 2021, the USPSTF said that positive results on stool-based tests would require follow-up with colonoscopy, defining this follow-up as part of the screening benefit. In 2022, Medicare followed by setting a policy that ended the copay for these follow-up colonoscopies.
For breast screening, there are efforts underway in Congress to end copays for breast screening. In May, Rep. Rosa DeLauro (D-Conn.) introduced a bill, the Find It Early Act, that would require both private and government insurers to cover the out-of-pocket costs for many women receiving screening with ultrasound and MRI.
When the USPSTF finalizes its breast screening guidelines, the recommendations will be woven into discussions between primary care physicians and patients about breast cancer screening.
As guidelines and evidence evolve, “we’re learning to adjust” and communicate these changes to patients, said Tochi Iroku-Malize, MD, president of the American Academy of Family Physicians.
However, gaps in the guidance will leave some open-ended questions about optimal screening practices and how much screening may cost.
Given that, Dr. Iroku-Malize takes many factors into account when discussing screening options with her patients. Based on the new information and the patient’s information, she said she will tell her patients, “We’re going to adjust our guidance as to what you need.”
A version of this article first appeared on Medscape.com.
, while also renewing debates about the timing of these tests and the screening approaches used.
The U.S. Preventive Services Task Force is currently finalizing an update to its recommendations on breast cancer screening. In May, the task force released a proposed update that dropped the initial age for routine mammogram screening from 50 to 40.
The task force intends to give a “B” rating to this recommendation, which covers screening every other year up to age 74 for women deemed average risk for breast cancer.
The task force’s rating carries clout, A. Mark Fendrick, MD, director of the Value-Based Insurance Design at the University of Michigan, Ann Arbor, said in an interview.
For one, the Affordable Care Act requires that private insurers cover services that get top A or B marks from USPSTF without charging copays.
However, Dr. Fendrick noted, such coverage does not necessarily apply to follow-up testing when a routine mammogram comes back with a positive finding. The expense of follow-up testing may deter some women from seeking follow-up diagnostic imaging or biopsies after an abnormal result on a screening mammogram.
A recent analysis in JAMA Network Open found that women facing higher anticipated out-of-pocket costs for breast cancer diagnostic tests, based on their health insurance plan, were less likely to get that follow-up screening. For instance, the use of breast MRI decreased by nearly 24% between patients undergoing subsequent diagnostic testing in plans with the lowest out-of-pocket costs vs. those with the highest.
“The study’s central finding that some women who have an abnormal result on a mammogram may not get appropriate follow-up because of cost is worrisome,” said Dr. Fendrick and Ilana B. Richman, MD, MHS, in an accompanying commentary to the JAMA analysis. “On an individual level, high out-of-pocket costs may directly contribute to worse health outcomes or require individuals to use scarce financial resources that may otherwise be used for critical items such as food or rent.”
For patients to fully benefit from early detection, the USPSTF would also need to make clear that follow-up diagnostic mammograms are covered, Dr. Fendrick said.
The ongoing debates
Concerns over the costs of potential follow-up tests are not the only issues experts have highlighted since USPSTF released its updated draft guidance on screening mammography.
The task force’s proposed update has also reignited questions and uncertainties surrounding when to screen, how often, and what types are best.
When it comes to frequency, the major organizations that provide screening guidance don’t see eye to eye. The USPSTF recommends breast cancer screening every other year, while the American College of Radiology recommends screening every year because that approach leads to saves “the most lives.”
At this time, the American College of Obstetricians and Gynecologists guidance currently teeters in the middle, suggesting either annual or biennial screening and highlighting the pros and cons of either approach. According to ACOG, “annual screening intervals appear to result in the least number of breast cancer deaths, particularly in younger women, but at the cost of additional callbacks and biopsies.”
When to begin screening represents another point of contention. While some experts, such as ACOG, agree with the task force’s decision to lower the screening start age to 40, others point to the need for greater nuance on setting the appropriate screening age. The main issue: the task force’s draft sets a uniform age to begin screening, but the risk for breast cancer and breast cancer mortality is not uniform across different racial and ethnic groups.
A recent study published in JAMA Network Open found that, among women aged 40-49, breast cancer mortality was highest among Black women (27 deaths per 100,000 person-years) followed by White women (15 deaths per 100,000 person-years). Based on a recommended screening age of 50, the authors suggested that Black women should start screening at age 42, whereas White women could start at 51.
“These findings suggest that health policy makers and clinicians could consider an alternative, race and ethnicity–adapted approach in which Black female patients start screening earlier,” writes Tianhui Chen, PhD, of China’s Zhejiang Cancer Hospital and coauthor of the study.
Weighing in on the guidance, the nonprofit National Center for Health Research urged the task force to consider suggesting different screening schedules based on race and ethnicity data. That would mean the recommendation to start at age 40 should only apply to Black women and other groups with higher-than-average risk for breast cancer at a younger age.
“Women are capable of understanding why the age to start mammography screening may be different for women with different risk factors,” the National Center for Health Research wrote in a comment to USPSTF, provided to this news organization by request. “What is confusing is when some physician groups recommend annual mammograms for all women starting at age 40, even though the data do not support that recommendation.”
While the ACR agreed with the task force’s recommendation to lower the screening age, the organization suggested starting risk assessments based on racial variations in breast cancer incidence and death even earlier. Specifically, the ACR recommended that high-risk groups, such as Black women, get risk assessments by age 25 to determine whether mammography before age 40 is needed.
Screening options for women with dense breasts may be some of the most challenging to weigh. Having dense breasts increases an individual’s risk for breast cancer, and mammography alone is not as effective at identifying breast cancer among these women. However, the evidence on the benefits vs. harms of additional screening beyond mammography remains mixed.
As a result, the task force decided to maintain its “I” grade on additional screening beyond mammography for these women – a grade that indicates insufficient evidence to determine the benefits and harms for a service.
The task force largely based its decision on the findings of two key reports. One report from the Cancer Intervention and Surveillance Modeling Network, which modeled potential outcomes of different screening strategies, indicated that extra screening might reduce breast cancer mortality in those with dense breasts, but at a cost of more false-positive reports.
The second report, a review from the Kaiser Permanente Evidence-based Practice Center, reaffirmed the benefits of routine mammography for reducing deaths from breast cancer, but found no solid evidence that different strategies – including supplemental screening in women with denser breasts – lowered breast cancer mortality or the risk of progression to advanced cancer. Further studies may show which approaches work best to reduce breast cancer deaths, the report said.
In this instance, ACOG agreed with USPSTF: “Based on the lack of data, ACOG does not recommend routine use of alternative or adjunctive tests to screening mammography in women with dense breasts who are asymptomatic and have no additional risk factors.”
Women with dense breasts should still be encouraged to receive regular screening mammography, even if the results they get may not be as accurate as those for women with less dense breasts, said Diana L. Miglioretti, PhD, of the University of California, Davis, who worked on a report for the USPSTF guidelines.
What’s next?
Despite ongoing debate and uncertainties surrounding some breast screening guidance, support for ending copay requirements for follow-up tests after a positive mammogram finding is widespread.
According to Dr. Fendrick, the USPSTF should expand coverage of follow-up testing after a positive mammogram to ensure people receive routine screening and any necessary diagnostic tests, as it did with colon cancer.
Before 2021, patients could face high costs for a colonoscopy following a positive stool-based Cologuard test. But in 2021, the USPSTF said that positive results on stool-based tests would require follow-up with colonoscopy, defining this follow-up as part of the screening benefit. In 2022, Medicare followed by setting a policy that ended the copay for these follow-up colonoscopies.
For breast screening, there are efforts underway in Congress to end copays for breast screening. In May, Rep. Rosa DeLauro (D-Conn.) introduced a bill, the Find It Early Act, that would require both private and government insurers to cover the out-of-pocket costs for many women receiving screening with ultrasound and MRI.
When the USPSTF finalizes its breast screening guidelines, the recommendations will be woven into discussions between primary care physicians and patients about breast cancer screening.
As guidelines and evidence evolve, “we’re learning to adjust” and communicate these changes to patients, said Tochi Iroku-Malize, MD, president of the American Academy of Family Physicians.
However, gaps in the guidance will leave some open-ended questions about optimal screening practices and how much screening may cost.
Given that, Dr. Iroku-Malize takes many factors into account when discussing screening options with her patients. Based on the new information and the patient’s information, she said she will tell her patients, “We’re going to adjust our guidance as to what you need.”
A version of this article first appeared on Medscape.com.
Proposal to cap Part B pay on some drugs draws opposition
An influential panel proposed capping Medicare Part B pay for some drugs, arguing this would remove financial incentives to use more costly medicines when there are less expensive equivalents.
Medical groups have objected to both this recommendation from the Medicare Payment Advisory Commission (MedPAC) and the panel’s underlying premise. MedPAC said financial as well as clinical factors can come into play in clinicians’ choices of drugs for patients.
In an interview, Christina Downey, MD, chair of the Government Affairs Committee of the American College of Rheumatology, said physicians in her field cannot switch patients’ medicines to try to make a profit.
“Patients only respond to the drugs that they respond to,” Dr. Downey said. “It’s frankly very insulting to say that physicians just force patients to go on medicines that are going to make them a bunch of money.”
In a June report to Congress, MedPAC recommended reducing the add-on payment for many drugs given in hospitals and clinics, which are thus covered by Part B, as part of a package of suggestions for addressing rising costs. Part B drug spending grew about 9% annually between 2009 and 2021, rising from $15.4 billion to $42.9 billion, MedPAC said.
Medicare’s current Part B drug pricing model starts with the reported average sales price (ASP) and then adds about 4.3% or 6%, depending on current budget-sequester law, to the cost of medicines.
MedPAC members voted 17-0 in April in favor of a general recommendation to revise the Part B payment approach. In the June report, MedPAC fleshes out this idea. It mentions a model in which the add-on Part B payment would be the lesser of either 6% of the ASP, 3% plus $24, or $220.
The majority of Part B drug administrations are for very low-priced drugs, MedPAC said. But for some of the more costly ones, annual prices can be more than $400,000 per patient, and future launch prices may be even higher for certain types of products, such as gene therapies, MedPAC said.
“There is no evidence that the costs of a drug’s administration are proportionate to the price of the drug,” MedPAC said.
Concerns about how well Medicare covers the cost of drug administration should be addressed through other pathways, such as the American Medical
Association’s Specialty Society Relative Value Scale Update Committee (RUC), MedPAC said. AMA’s RUC advises the Centers for Medicare & Medicaid Services on the physician fee schedule.
Congress is not obliged to act on or to even consider MedPAC’s work. In general, lawmakers and CMS often pay heed to the panel’s recommendations, sometimes incorporating them into new policy.
But this new MedPAC Part B recommendation has drawn strong opposition, similar to the response to a 2016 CMS plan to cut the Part B add-on payment. That plan, which CMS later abandoned, would have cut the markup on Part B drugs to 2.5% and added a flat fee to cover administration costs.
Why not focus on PBMs instead?
The timing of the MedPAC recommendation is poor, given that CMS already is trying to implement the Inflation Reduction Act and create a new system of direct Medicare drug price negotiations, as ordered by Congress, said Madelaine A. Feldman, MD, a rheumatologist based in New Orleans.
A better approach for lowering drug prices would be to focus more on the operations of pharmacy benefit managers (PBMs), said Dr. Feldman, who also is vice president for advocacy and government affairs for the Coalition of State Rheumatology Organizations. A pending bipartisan Senate bill, for example, would prohibit PBM compensation based on the price of a drug as a condition of entering into a contract with a Medicare Part D plan.
Congress needs to take steps to unlink the profits of PBMs from higher drug prices, Dr. Feldman said.
“Until that happens, we can put all the lipstick we want on this big pig, but it’s not going to really fix the problem,” she said.
Reduced pay for drugs acquired through 340B program?
In an interview about the new MedPAC proposal, Ted Okon, executive director of the Community Oncology Alliance, urged renewed attention to what he sees as unintended consequences of the 340B discount drug program.
Under this program, certain hospitals can acquire drugs at steeply reduced prices, but they are not obliged to share those discounts with patients. Hospitals that participate in the 340B program can gain funds when patients and their insurers, including Medicare, pay more for the medicines hospitals and other organizations acquired with the 340B discount. Hospitals say they use the money from the 340B program to expand resources in their communities.
But rapid growth of the program in recent years has led to questions, especially about the role of contract pharmacies that manage the program. Congress created the 340B program in 1992 as a workaround to then new rules on Medicaid drug coverage.
In 2021, participating hospitals and clinics and organizations purchased about $44 billion worth of medicines through the 340B drug program. This was an increase of 16% from the previous year, according to a report from the nonprofit Commonwealth Fund. The number of sites, including hospitals and pharmacies, enrolled in the 340B program rose from 8,100 in 2000 to 50,000 by 2020, the report said.
MedPAC in 2016 urged CMS to reduce the amount Medicare pays for drugs acquired through the 340B program. CMS did so during the Trump administration, a policy later defended by the Biden administration.
But the U.S. Supreme Court last year said Medicare erred in its approach to making this cut, as earlier reported. Federal law required that the Department of Health and Human Services conduct a survey to support such a step, and HHS did not do this, the court said. CMS thus was ordered to return Medicare to the ASP+6% payment model for drugs purchased through the 340B discount program.
In the June report, though, MedPAC stuck by its 2016 recommendation that Medicare reduce its payments for drugs purchased through the 340B discount program despite this setback.
“We continue to believe that this approach is appropriate, and the specific level of payment reduction could be considered further as newer data become available,” MedPAC said.
Hospital, PhRMA split
Hospitals would certainly contest any renewed bid by CMS to drop Medicare’s pay for drugs purchased through the 340B program. The American Hospital Association objected to the MedPAC proposal regarding the add-on payment in Part B drug pricing.
MedPAC commissioners discussed this idea at a January meeting, prompting a February letter from the AHA to the panel. Like Dr. Feldman, AHA said it would be “premature” to launch into a revision of Part B drug pricing while the impact of the IRA on drug prices was still unclear.
AHA also noted that a reduction in Part B drug reimbursement would “shift the responsibility for the rapid increase in drug prices away from drug manufacturers, and instead places the burden on hospitals and patients.”
But the AHA gave a much warmer reception to another proposal MedPAC considered this year and that it included in its June report, which is a plan to address the high cost of certain drugs of as yet unconfirmed clinical benefit.
In April, the AHA said it supports a move toward a “value-based approach” in certain cases in which first-in-class medicines are sold under U.S. Food and Drug Administration’s accelerated approvals. Medicare could then cap payment for such drugs that have excessively high launch prices and uncertain clinical benefit, AHA said.
In the June report, MedPAC recommended that Medicare be able to place such a limit on Part B payments in certain cases, including ones in which companies do not meet FDA deadlines for postmarketing confirmatory trials.
The Pharmaceutical Research and Manufacturers of America (PhRMA) objected to this proposed change. The trade group for drugmakers said the FDA often revises and extends enrollment milestones for pending confirmatory trials when companies hit snags, such as challenges in enrolling patients, PhRMA said.
Reducing Part B payment for drugs for which confirmatory trials have been delayed would have a “disproportionate impact” on smaller and rural communities, where independent practices struggle to keep their doors open as it is, PhRMA spokeswoman Nicole Longo wrote in a blog post.
“If physicians can’t afford to administer a medicine, then they won’t and that means their patients won’t have access to them either,” Ms. Longo wrote.
A version of this article first appeared on Medscape.com.
An influential panel proposed capping Medicare Part B pay for some drugs, arguing this would remove financial incentives to use more costly medicines when there are less expensive equivalents.
Medical groups have objected to both this recommendation from the Medicare Payment Advisory Commission (MedPAC) and the panel’s underlying premise. MedPAC said financial as well as clinical factors can come into play in clinicians’ choices of drugs for patients.
In an interview, Christina Downey, MD, chair of the Government Affairs Committee of the American College of Rheumatology, said physicians in her field cannot switch patients’ medicines to try to make a profit.
“Patients only respond to the drugs that they respond to,” Dr. Downey said. “It’s frankly very insulting to say that physicians just force patients to go on medicines that are going to make them a bunch of money.”
In a June report to Congress, MedPAC recommended reducing the add-on payment for many drugs given in hospitals and clinics, which are thus covered by Part B, as part of a package of suggestions for addressing rising costs. Part B drug spending grew about 9% annually between 2009 and 2021, rising from $15.4 billion to $42.9 billion, MedPAC said.
Medicare’s current Part B drug pricing model starts with the reported average sales price (ASP) and then adds about 4.3% or 6%, depending on current budget-sequester law, to the cost of medicines.
MedPAC members voted 17-0 in April in favor of a general recommendation to revise the Part B payment approach. In the June report, MedPAC fleshes out this idea. It mentions a model in which the add-on Part B payment would be the lesser of either 6% of the ASP, 3% plus $24, or $220.
The majority of Part B drug administrations are for very low-priced drugs, MedPAC said. But for some of the more costly ones, annual prices can be more than $400,000 per patient, and future launch prices may be even higher for certain types of products, such as gene therapies, MedPAC said.
“There is no evidence that the costs of a drug’s administration are proportionate to the price of the drug,” MedPAC said.
Concerns about how well Medicare covers the cost of drug administration should be addressed through other pathways, such as the American Medical
Association’s Specialty Society Relative Value Scale Update Committee (RUC), MedPAC said. AMA’s RUC advises the Centers for Medicare & Medicaid Services on the physician fee schedule.
Congress is not obliged to act on or to even consider MedPAC’s work. In general, lawmakers and CMS often pay heed to the panel’s recommendations, sometimes incorporating them into new policy.
But this new MedPAC Part B recommendation has drawn strong opposition, similar to the response to a 2016 CMS plan to cut the Part B add-on payment. That plan, which CMS later abandoned, would have cut the markup on Part B drugs to 2.5% and added a flat fee to cover administration costs.
Why not focus on PBMs instead?
The timing of the MedPAC recommendation is poor, given that CMS already is trying to implement the Inflation Reduction Act and create a new system of direct Medicare drug price negotiations, as ordered by Congress, said Madelaine A. Feldman, MD, a rheumatologist based in New Orleans.
A better approach for lowering drug prices would be to focus more on the operations of pharmacy benefit managers (PBMs), said Dr. Feldman, who also is vice president for advocacy and government affairs for the Coalition of State Rheumatology Organizations. A pending bipartisan Senate bill, for example, would prohibit PBM compensation based on the price of a drug as a condition of entering into a contract with a Medicare Part D plan.
Congress needs to take steps to unlink the profits of PBMs from higher drug prices, Dr. Feldman said.
“Until that happens, we can put all the lipstick we want on this big pig, but it’s not going to really fix the problem,” she said.
Reduced pay for drugs acquired through 340B program?
In an interview about the new MedPAC proposal, Ted Okon, executive director of the Community Oncology Alliance, urged renewed attention to what he sees as unintended consequences of the 340B discount drug program.
Under this program, certain hospitals can acquire drugs at steeply reduced prices, but they are not obliged to share those discounts with patients. Hospitals that participate in the 340B program can gain funds when patients and their insurers, including Medicare, pay more for the medicines hospitals and other organizations acquired with the 340B discount. Hospitals say they use the money from the 340B program to expand resources in their communities.
But rapid growth of the program in recent years has led to questions, especially about the role of contract pharmacies that manage the program. Congress created the 340B program in 1992 as a workaround to then new rules on Medicaid drug coverage.
In 2021, participating hospitals and clinics and organizations purchased about $44 billion worth of medicines through the 340B drug program. This was an increase of 16% from the previous year, according to a report from the nonprofit Commonwealth Fund. The number of sites, including hospitals and pharmacies, enrolled in the 340B program rose from 8,100 in 2000 to 50,000 by 2020, the report said.
MedPAC in 2016 urged CMS to reduce the amount Medicare pays for drugs acquired through the 340B program. CMS did so during the Trump administration, a policy later defended by the Biden administration.
But the U.S. Supreme Court last year said Medicare erred in its approach to making this cut, as earlier reported. Federal law required that the Department of Health and Human Services conduct a survey to support such a step, and HHS did not do this, the court said. CMS thus was ordered to return Medicare to the ASP+6% payment model for drugs purchased through the 340B discount program.
In the June report, though, MedPAC stuck by its 2016 recommendation that Medicare reduce its payments for drugs purchased through the 340B discount program despite this setback.
“We continue to believe that this approach is appropriate, and the specific level of payment reduction could be considered further as newer data become available,” MedPAC said.
Hospital, PhRMA split
Hospitals would certainly contest any renewed bid by CMS to drop Medicare’s pay for drugs purchased through the 340B program. The American Hospital Association objected to the MedPAC proposal regarding the add-on payment in Part B drug pricing.
MedPAC commissioners discussed this idea at a January meeting, prompting a February letter from the AHA to the panel. Like Dr. Feldman, AHA said it would be “premature” to launch into a revision of Part B drug pricing while the impact of the IRA on drug prices was still unclear.
AHA also noted that a reduction in Part B drug reimbursement would “shift the responsibility for the rapid increase in drug prices away from drug manufacturers, and instead places the burden on hospitals and patients.”
But the AHA gave a much warmer reception to another proposal MedPAC considered this year and that it included in its June report, which is a plan to address the high cost of certain drugs of as yet unconfirmed clinical benefit.
In April, the AHA said it supports a move toward a “value-based approach” in certain cases in which first-in-class medicines are sold under U.S. Food and Drug Administration’s accelerated approvals. Medicare could then cap payment for such drugs that have excessively high launch prices and uncertain clinical benefit, AHA said.
In the June report, MedPAC recommended that Medicare be able to place such a limit on Part B payments in certain cases, including ones in which companies do not meet FDA deadlines for postmarketing confirmatory trials.
The Pharmaceutical Research and Manufacturers of America (PhRMA) objected to this proposed change. The trade group for drugmakers said the FDA often revises and extends enrollment milestones for pending confirmatory trials when companies hit snags, such as challenges in enrolling patients, PhRMA said.
Reducing Part B payment for drugs for which confirmatory trials have been delayed would have a “disproportionate impact” on smaller and rural communities, where independent practices struggle to keep their doors open as it is, PhRMA spokeswoman Nicole Longo wrote in a blog post.
“If physicians can’t afford to administer a medicine, then they won’t and that means their patients won’t have access to them either,” Ms. Longo wrote.
A version of this article first appeared on Medscape.com.
An influential panel proposed capping Medicare Part B pay for some drugs, arguing this would remove financial incentives to use more costly medicines when there are less expensive equivalents.
Medical groups have objected to both this recommendation from the Medicare Payment Advisory Commission (MedPAC) and the panel’s underlying premise. MedPAC said financial as well as clinical factors can come into play in clinicians’ choices of drugs for patients.
In an interview, Christina Downey, MD, chair of the Government Affairs Committee of the American College of Rheumatology, said physicians in her field cannot switch patients’ medicines to try to make a profit.
“Patients only respond to the drugs that they respond to,” Dr. Downey said. “It’s frankly very insulting to say that physicians just force patients to go on medicines that are going to make them a bunch of money.”
In a June report to Congress, MedPAC recommended reducing the add-on payment for many drugs given in hospitals and clinics, which are thus covered by Part B, as part of a package of suggestions for addressing rising costs. Part B drug spending grew about 9% annually between 2009 and 2021, rising from $15.4 billion to $42.9 billion, MedPAC said.
Medicare’s current Part B drug pricing model starts with the reported average sales price (ASP) and then adds about 4.3% or 6%, depending on current budget-sequester law, to the cost of medicines.
MedPAC members voted 17-0 in April in favor of a general recommendation to revise the Part B payment approach. In the June report, MedPAC fleshes out this idea. It mentions a model in which the add-on Part B payment would be the lesser of either 6% of the ASP, 3% plus $24, or $220.
The majority of Part B drug administrations are for very low-priced drugs, MedPAC said. But for some of the more costly ones, annual prices can be more than $400,000 per patient, and future launch prices may be even higher for certain types of products, such as gene therapies, MedPAC said.
“There is no evidence that the costs of a drug’s administration are proportionate to the price of the drug,” MedPAC said.
Concerns about how well Medicare covers the cost of drug administration should be addressed through other pathways, such as the American Medical
Association’s Specialty Society Relative Value Scale Update Committee (RUC), MedPAC said. AMA’s RUC advises the Centers for Medicare & Medicaid Services on the physician fee schedule.
Congress is not obliged to act on or to even consider MedPAC’s work. In general, lawmakers and CMS often pay heed to the panel’s recommendations, sometimes incorporating them into new policy.
But this new MedPAC Part B recommendation has drawn strong opposition, similar to the response to a 2016 CMS plan to cut the Part B add-on payment. That plan, which CMS later abandoned, would have cut the markup on Part B drugs to 2.5% and added a flat fee to cover administration costs.
Why not focus on PBMs instead?
The timing of the MedPAC recommendation is poor, given that CMS already is trying to implement the Inflation Reduction Act and create a new system of direct Medicare drug price negotiations, as ordered by Congress, said Madelaine A. Feldman, MD, a rheumatologist based in New Orleans.
A better approach for lowering drug prices would be to focus more on the operations of pharmacy benefit managers (PBMs), said Dr. Feldman, who also is vice president for advocacy and government affairs for the Coalition of State Rheumatology Organizations. A pending bipartisan Senate bill, for example, would prohibit PBM compensation based on the price of a drug as a condition of entering into a contract with a Medicare Part D plan.
Congress needs to take steps to unlink the profits of PBMs from higher drug prices, Dr. Feldman said.
“Until that happens, we can put all the lipstick we want on this big pig, but it’s not going to really fix the problem,” she said.
Reduced pay for drugs acquired through 340B program?
In an interview about the new MedPAC proposal, Ted Okon, executive director of the Community Oncology Alliance, urged renewed attention to what he sees as unintended consequences of the 340B discount drug program.
Under this program, certain hospitals can acquire drugs at steeply reduced prices, but they are not obliged to share those discounts with patients. Hospitals that participate in the 340B program can gain funds when patients and their insurers, including Medicare, pay more for the medicines hospitals and other organizations acquired with the 340B discount. Hospitals say they use the money from the 340B program to expand resources in their communities.
But rapid growth of the program in recent years has led to questions, especially about the role of contract pharmacies that manage the program. Congress created the 340B program in 1992 as a workaround to then new rules on Medicaid drug coverage.
In 2021, participating hospitals and clinics and organizations purchased about $44 billion worth of medicines through the 340B drug program. This was an increase of 16% from the previous year, according to a report from the nonprofit Commonwealth Fund. The number of sites, including hospitals and pharmacies, enrolled in the 340B program rose from 8,100 in 2000 to 50,000 by 2020, the report said.
MedPAC in 2016 urged CMS to reduce the amount Medicare pays for drugs acquired through the 340B program. CMS did so during the Trump administration, a policy later defended by the Biden administration.
But the U.S. Supreme Court last year said Medicare erred in its approach to making this cut, as earlier reported. Federal law required that the Department of Health and Human Services conduct a survey to support such a step, and HHS did not do this, the court said. CMS thus was ordered to return Medicare to the ASP+6% payment model for drugs purchased through the 340B discount program.
In the June report, though, MedPAC stuck by its 2016 recommendation that Medicare reduce its payments for drugs purchased through the 340B discount program despite this setback.
“We continue to believe that this approach is appropriate, and the specific level of payment reduction could be considered further as newer data become available,” MedPAC said.
Hospital, PhRMA split
Hospitals would certainly contest any renewed bid by CMS to drop Medicare’s pay for drugs purchased through the 340B program. The American Hospital Association objected to the MedPAC proposal regarding the add-on payment in Part B drug pricing.
MedPAC commissioners discussed this idea at a January meeting, prompting a February letter from the AHA to the panel. Like Dr. Feldman, AHA said it would be “premature” to launch into a revision of Part B drug pricing while the impact of the IRA on drug prices was still unclear.
AHA also noted that a reduction in Part B drug reimbursement would “shift the responsibility for the rapid increase in drug prices away from drug manufacturers, and instead places the burden on hospitals and patients.”
But the AHA gave a much warmer reception to another proposal MedPAC considered this year and that it included in its June report, which is a plan to address the high cost of certain drugs of as yet unconfirmed clinical benefit.
In April, the AHA said it supports a move toward a “value-based approach” in certain cases in which first-in-class medicines are sold under U.S. Food and Drug Administration’s accelerated approvals. Medicare could then cap payment for such drugs that have excessively high launch prices and uncertain clinical benefit, AHA said.
In the June report, MedPAC recommended that Medicare be able to place such a limit on Part B payments in certain cases, including ones in which companies do not meet FDA deadlines for postmarketing confirmatory trials.
The Pharmaceutical Research and Manufacturers of America (PhRMA) objected to this proposed change. The trade group for drugmakers said the FDA often revises and extends enrollment milestones for pending confirmatory trials when companies hit snags, such as challenges in enrolling patients, PhRMA said.
Reducing Part B payment for drugs for which confirmatory trials have been delayed would have a “disproportionate impact” on smaller and rural communities, where independent practices struggle to keep their doors open as it is, PhRMA spokeswoman Nicole Longo wrote in a blog post.
“If physicians can’t afford to administer a medicine, then they won’t and that means their patients won’t have access to them either,” Ms. Longo wrote.
A version of this article first appeared on Medscape.com.
FDA panel backs new COVID booster focusing only on variants
but questioned whether the population as a whole needs booster shots and how often they should be given.
The Vaccines and Related Biological Products Advisory Committee of the FDA voted 21-0 in favor of the recommendation about the strain to be used in the next crop of vaccines.
In the briefing document for the meeting, FDA staff said the available evidence suggests that a monovalent (single-strain) XBB-lineage vaccine “is warranted” for the 2023-2024 vaccination campaign and would replace the current bivalent vaccine, which targets the original version of the virus and two strains from the Omicron variant.
FDA staff also noted how such a shift would be in line with the World Health Organization toward targeting the XBB family of subvariants. European regulators have done this as well.
The FDA is not obligated to act on the panel’s recommendations. But the agency often does and is highly likely to do so in this case. Vaccine companies will need the recommendation from the FDA to begin making vaccines for the fall.
New shot every year?
The FDA asked its expert panel to vote only on the question about the makeup of future vaccines in terms of which strain to include.
But panelists also raised other questions during the meeting, including concerns about moves toward tying COVID vaccinations into the model of annual flu shots.
Paul Offit, MD, director of the Vaccine Education Center at the Children’s Hospital of Philadelphia, argued for greater focus on the response of T cells after vaccination, even in light of the already recognized waning of antibody protection.
In a recent Substack article, Dr. Offit called T cells the “unsung hero” of the pandemic. They take longer to develop after infection or vaccination than the antibodies that first attack the virus, but immune memory cells called B and T cells “are long-lived,” and their “protection against severe disease often lasts for years and sometimes decades.”
Dr. Offit said he was concerned about using a blanket approach for future recommendations for COVID vaccinations, following the one now in place for influenza vaccines.
The Centers for Disease Control and Prevention recommends flu shots for everyone 6 months and older, with rare exceptions.
“We need to continue to define who those high-risk groups are and not make this a recommendation for everybody every season,” he said.
Dr. Offit offered his own experience as an example. While he had been vaccinated against the virus’s early Wuhan strain, he still was infected, most likely with a variant that emerged later.
“That was a drifted virus. That’s why I had a mild infection but I didn’t have a severe infection, because presumably I had T cells which prevented that severe infection, which may last for years,” Dr. Offit said.
Pfizer and Moderna, the two companies that make mRNA-based COVID vaccines, are working on experimental products meant to protect against both flu and SARS-COv-2 in one shot. Novavax, maker of a more traditional protein-based COVID shot, is doing the same.
The idea of these combination products is to make it more convenient for people to protect against both viruses, while also offering companies some marketing advantages.
But without referring to these drugmakers’ plans for future combo flu-COVID shots, members of the FDA panel raised objections to an assumption of routine annual vaccines against variants of SARS-CoV-2.
Among the panelists who expressed concerns was Henry H. Bernstein, DO, a former member of the CDC’s Advisory Committee on Immunization Practices.
Bernstein questioned the approach of dubbing these the “2023-2024 formulas,” as this approach conveyed a sense of an expectation for a need for annual vaccines, as happens with flu.
“It’s not clear to me that this is a seasonal virus yet,” said Dr. Bernstein, who is also a professor of pediatrics at Hofstra University, Hempstead, N.Y..
In response to Dr. Bernstein’s point, Arnold Monto, MD, the acting chair of the FDA panel, suggested such a pattern could emerge, while also agreeing that it’s too soon to say for sure.
A professor emeritus at the University of Michigan, Ann Arbor, Dr. Monto’s career included pandemic planning and emergency response to virus outbreaks, including the 1968 Hong Kong influenza pandemic, avian influenza, and the original SARS.
“I think it’s premature to say that this virus will not become seasonal,” Dr. Monto said about SARS-CoV-2. “I agree. We’re not there yet, but we may be.”
At the end of the meeting, Dr. Monto recapped the meeting’s key points, noting that there was a general consensus that the XBB.1.5 subvariant would be the best to use in future COVID shots.
He also noted that Novavax, which makes the more traditional protein-based vaccine, along with Pfizer and Moderna, already have honed in on this subvariant, which would allow for rapid development of updated COVID vaccines.
“The fact that most of the manufacturers are ready to work on an XBB 1.5 [vaccine] is an added reason to select this strain or this variant, given the immunologic data,” Dr. Monto said.
Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said the demands involved in manufacturing vaccines tilts toward annual changes.
“Practically, we’re going to have one update per year, barring a heroic effort to deal with a strain that pops up that is essentially so different that it requires us to mobilize tremendous resources to address that strain change,” he said.
Dr. Marks questioned the panelists’ concerns about likening flu and COVID vaccination practices. The FDA staff’s intent was to try to help the public understand the need for follow-on vaccination.
“I’m really having trouble understanding that committee’s need to bristle against something that’s similar to influenza. People understand a yearly influenza vaccine,” Dr. Marks said.
And it’s not certain when another major change in the COVID virus will follow the XBB subvariant, but it’s likely one will – and soon, Dr. Marks said.
“It looks like, probably by next fall, there’ll be further drift from this,” he said.
Informing the public
Dr. Marks also stressed the need to better convey the benefits of vaccination to people in the United States.
CDC data estimate that 70% of the U.S. population completed an initial series of the original monovalent vaccines, with only 17% then getting bivalent shots. There’s even a decline among people ages 65 and older. CDC estimates 94% of this group completed their primary series, but only 43% got the bivalent booster dose.
“We have to do better because we have not done a good job today communicating to the American public what’s going on here,” Marks said.
Researchers also are still trying to determine the best timing for people to get additional COVID shots. Finding the “sweet spot” where people can maximize additional protection is tricky, with people most protected if they happen to get shot near the beginning of an uptick in viral spread, the CDC’s Ruth Link-Gelles, PhD, MPH, told the panel during a presentation.
“You’re going to get the best incremental benefit if it’s been longer since your last vaccine,” she said. “But of course, if you wait too long since your last vaccine, you’re left with very little protection, and so you’re at higher risk of severe illness.”
Like Dr. Marks, Dr. Link-Gelles stressed the need for persuading more people to get follow-on vaccines.
“Most Americans, at this point, haven’t even received the bivalent and so are a year or more out from their monovalent dose and so have relatively little protection left,” she said.
A version of this article first appeared on WebMD.com.
but questioned whether the population as a whole needs booster shots and how often they should be given.
The Vaccines and Related Biological Products Advisory Committee of the FDA voted 21-0 in favor of the recommendation about the strain to be used in the next crop of vaccines.
In the briefing document for the meeting, FDA staff said the available evidence suggests that a monovalent (single-strain) XBB-lineage vaccine “is warranted” for the 2023-2024 vaccination campaign and would replace the current bivalent vaccine, which targets the original version of the virus and two strains from the Omicron variant.
FDA staff also noted how such a shift would be in line with the World Health Organization toward targeting the XBB family of subvariants. European regulators have done this as well.
The FDA is not obligated to act on the panel’s recommendations. But the agency often does and is highly likely to do so in this case. Vaccine companies will need the recommendation from the FDA to begin making vaccines for the fall.
New shot every year?
The FDA asked its expert panel to vote only on the question about the makeup of future vaccines in terms of which strain to include.
But panelists also raised other questions during the meeting, including concerns about moves toward tying COVID vaccinations into the model of annual flu shots.
Paul Offit, MD, director of the Vaccine Education Center at the Children’s Hospital of Philadelphia, argued for greater focus on the response of T cells after vaccination, even in light of the already recognized waning of antibody protection.
In a recent Substack article, Dr. Offit called T cells the “unsung hero” of the pandemic. They take longer to develop after infection or vaccination than the antibodies that first attack the virus, but immune memory cells called B and T cells “are long-lived,” and their “protection against severe disease often lasts for years and sometimes decades.”
Dr. Offit said he was concerned about using a blanket approach for future recommendations for COVID vaccinations, following the one now in place for influenza vaccines.
The Centers for Disease Control and Prevention recommends flu shots for everyone 6 months and older, with rare exceptions.
“We need to continue to define who those high-risk groups are and not make this a recommendation for everybody every season,” he said.
Dr. Offit offered his own experience as an example. While he had been vaccinated against the virus’s early Wuhan strain, he still was infected, most likely with a variant that emerged later.
“That was a drifted virus. That’s why I had a mild infection but I didn’t have a severe infection, because presumably I had T cells which prevented that severe infection, which may last for years,” Dr. Offit said.
Pfizer and Moderna, the two companies that make mRNA-based COVID vaccines, are working on experimental products meant to protect against both flu and SARS-COv-2 in one shot. Novavax, maker of a more traditional protein-based COVID shot, is doing the same.
The idea of these combination products is to make it more convenient for people to protect against both viruses, while also offering companies some marketing advantages.
But without referring to these drugmakers’ plans for future combo flu-COVID shots, members of the FDA panel raised objections to an assumption of routine annual vaccines against variants of SARS-CoV-2.
Among the panelists who expressed concerns was Henry H. Bernstein, DO, a former member of the CDC’s Advisory Committee on Immunization Practices.
Bernstein questioned the approach of dubbing these the “2023-2024 formulas,” as this approach conveyed a sense of an expectation for a need for annual vaccines, as happens with flu.
“It’s not clear to me that this is a seasonal virus yet,” said Dr. Bernstein, who is also a professor of pediatrics at Hofstra University, Hempstead, N.Y..
In response to Dr. Bernstein’s point, Arnold Monto, MD, the acting chair of the FDA panel, suggested such a pattern could emerge, while also agreeing that it’s too soon to say for sure.
A professor emeritus at the University of Michigan, Ann Arbor, Dr. Monto’s career included pandemic planning and emergency response to virus outbreaks, including the 1968 Hong Kong influenza pandemic, avian influenza, and the original SARS.
“I think it’s premature to say that this virus will not become seasonal,” Dr. Monto said about SARS-CoV-2. “I agree. We’re not there yet, but we may be.”
At the end of the meeting, Dr. Monto recapped the meeting’s key points, noting that there was a general consensus that the XBB.1.5 subvariant would be the best to use in future COVID shots.
He also noted that Novavax, which makes the more traditional protein-based vaccine, along with Pfizer and Moderna, already have honed in on this subvariant, which would allow for rapid development of updated COVID vaccines.
“The fact that most of the manufacturers are ready to work on an XBB 1.5 [vaccine] is an added reason to select this strain or this variant, given the immunologic data,” Dr. Monto said.
Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said the demands involved in manufacturing vaccines tilts toward annual changes.
“Practically, we’re going to have one update per year, barring a heroic effort to deal with a strain that pops up that is essentially so different that it requires us to mobilize tremendous resources to address that strain change,” he said.
Dr. Marks questioned the panelists’ concerns about likening flu and COVID vaccination practices. The FDA staff’s intent was to try to help the public understand the need for follow-on vaccination.
“I’m really having trouble understanding that committee’s need to bristle against something that’s similar to influenza. People understand a yearly influenza vaccine,” Dr. Marks said.
And it’s not certain when another major change in the COVID virus will follow the XBB subvariant, but it’s likely one will – and soon, Dr. Marks said.
“It looks like, probably by next fall, there’ll be further drift from this,” he said.
Informing the public
Dr. Marks also stressed the need to better convey the benefits of vaccination to people in the United States.
CDC data estimate that 70% of the U.S. population completed an initial series of the original monovalent vaccines, with only 17% then getting bivalent shots. There’s even a decline among people ages 65 and older. CDC estimates 94% of this group completed their primary series, but only 43% got the bivalent booster dose.
“We have to do better because we have not done a good job today communicating to the American public what’s going on here,” Marks said.
Researchers also are still trying to determine the best timing for people to get additional COVID shots. Finding the “sweet spot” where people can maximize additional protection is tricky, with people most protected if they happen to get shot near the beginning of an uptick in viral spread, the CDC’s Ruth Link-Gelles, PhD, MPH, told the panel during a presentation.
“You’re going to get the best incremental benefit if it’s been longer since your last vaccine,” she said. “But of course, if you wait too long since your last vaccine, you’re left with very little protection, and so you’re at higher risk of severe illness.”
Like Dr. Marks, Dr. Link-Gelles stressed the need for persuading more people to get follow-on vaccines.
“Most Americans, at this point, haven’t even received the bivalent and so are a year or more out from their monovalent dose and so have relatively little protection left,” she said.
A version of this article first appeared on WebMD.com.
but questioned whether the population as a whole needs booster shots and how often they should be given.
The Vaccines and Related Biological Products Advisory Committee of the FDA voted 21-0 in favor of the recommendation about the strain to be used in the next crop of vaccines.
In the briefing document for the meeting, FDA staff said the available evidence suggests that a monovalent (single-strain) XBB-lineage vaccine “is warranted” for the 2023-2024 vaccination campaign and would replace the current bivalent vaccine, which targets the original version of the virus and two strains from the Omicron variant.
FDA staff also noted how such a shift would be in line with the World Health Organization toward targeting the XBB family of subvariants. European regulators have done this as well.
The FDA is not obligated to act on the panel’s recommendations. But the agency often does and is highly likely to do so in this case. Vaccine companies will need the recommendation from the FDA to begin making vaccines for the fall.
New shot every year?
The FDA asked its expert panel to vote only on the question about the makeup of future vaccines in terms of which strain to include.
But panelists also raised other questions during the meeting, including concerns about moves toward tying COVID vaccinations into the model of annual flu shots.
Paul Offit, MD, director of the Vaccine Education Center at the Children’s Hospital of Philadelphia, argued for greater focus on the response of T cells after vaccination, even in light of the already recognized waning of antibody protection.
In a recent Substack article, Dr. Offit called T cells the “unsung hero” of the pandemic. They take longer to develop after infection or vaccination than the antibodies that first attack the virus, but immune memory cells called B and T cells “are long-lived,” and their “protection against severe disease often lasts for years and sometimes decades.”
Dr. Offit said he was concerned about using a blanket approach for future recommendations for COVID vaccinations, following the one now in place for influenza vaccines.
The Centers for Disease Control and Prevention recommends flu shots for everyone 6 months and older, with rare exceptions.
“We need to continue to define who those high-risk groups are and not make this a recommendation for everybody every season,” he said.
Dr. Offit offered his own experience as an example. While he had been vaccinated against the virus’s early Wuhan strain, he still was infected, most likely with a variant that emerged later.
“That was a drifted virus. That’s why I had a mild infection but I didn’t have a severe infection, because presumably I had T cells which prevented that severe infection, which may last for years,” Dr. Offit said.
Pfizer and Moderna, the two companies that make mRNA-based COVID vaccines, are working on experimental products meant to protect against both flu and SARS-COv-2 in one shot. Novavax, maker of a more traditional protein-based COVID shot, is doing the same.
The idea of these combination products is to make it more convenient for people to protect against both viruses, while also offering companies some marketing advantages.
But without referring to these drugmakers’ plans for future combo flu-COVID shots, members of the FDA panel raised objections to an assumption of routine annual vaccines against variants of SARS-CoV-2.
Among the panelists who expressed concerns was Henry H. Bernstein, DO, a former member of the CDC’s Advisory Committee on Immunization Practices.
Bernstein questioned the approach of dubbing these the “2023-2024 formulas,” as this approach conveyed a sense of an expectation for a need for annual vaccines, as happens with flu.
“It’s not clear to me that this is a seasonal virus yet,” said Dr. Bernstein, who is also a professor of pediatrics at Hofstra University, Hempstead, N.Y..
In response to Dr. Bernstein’s point, Arnold Monto, MD, the acting chair of the FDA panel, suggested such a pattern could emerge, while also agreeing that it’s too soon to say for sure.
A professor emeritus at the University of Michigan, Ann Arbor, Dr. Monto’s career included pandemic planning and emergency response to virus outbreaks, including the 1968 Hong Kong influenza pandemic, avian influenza, and the original SARS.
“I think it’s premature to say that this virus will not become seasonal,” Dr. Monto said about SARS-CoV-2. “I agree. We’re not there yet, but we may be.”
At the end of the meeting, Dr. Monto recapped the meeting’s key points, noting that there was a general consensus that the XBB.1.5 subvariant would be the best to use in future COVID shots.
He also noted that Novavax, which makes the more traditional protein-based vaccine, along with Pfizer and Moderna, already have honed in on this subvariant, which would allow for rapid development of updated COVID vaccines.
“The fact that most of the manufacturers are ready to work on an XBB 1.5 [vaccine] is an added reason to select this strain or this variant, given the immunologic data,” Dr. Monto said.
Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said the demands involved in manufacturing vaccines tilts toward annual changes.
“Practically, we’re going to have one update per year, barring a heroic effort to deal with a strain that pops up that is essentially so different that it requires us to mobilize tremendous resources to address that strain change,” he said.
Dr. Marks questioned the panelists’ concerns about likening flu and COVID vaccination practices. The FDA staff’s intent was to try to help the public understand the need for follow-on vaccination.
“I’m really having trouble understanding that committee’s need to bristle against something that’s similar to influenza. People understand a yearly influenza vaccine,” Dr. Marks said.
And it’s not certain when another major change in the COVID virus will follow the XBB subvariant, but it’s likely one will – and soon, Dr. Marks said.
“It looks like, probably by next fall, there’ll be further drift from this,” he said.
Informing the public
Dr. Marks also stressed the need to better convey the benefits of vaccination to people in the United States.
CDC data estimate that 70% of the U.S. population completed an initial series of the original monovalent vaccines, with only 17% then getting bivalent shots. There’s even a decline among people ages 65 and older. CDC estimates 94% of this group completed their primary series, but only 43% got the bivalent booster dose.
“We have to do better because we have not done a good job today communicating to the American public what’s going on here,” Marks said.
Researchers also are still trying to determine the best timing for people to get additional COVID shots. Finding the “sweet spot” where people can maximize additional protection is tricky, with people most protected if they happen to get shot near the beginning of an uptick in viral spread, the CDC’s Ruth Link-Gelles, PhD, MPH, told the panel during a presentation.
“You’re going to get the best incremental benefit if it’s been longer since your last vaccine,” she said. “But of course, if you wait too long since your last vaccine, you’re left with very little protection, and so you’re at higher risk of severe illness.”
Like Dr. Marks, Dr. Link-Gelles stressed the need for persuading more people to get follow-on vaccines.
“Most Americans, at this point, haven’t even received the bivalent and so are a year or more out from their monovalent dose and so have relatively little protection left,” she said.
A version of this article first appeared on WebMD.com.
New USPSTF draft suggests mammography start at 40, not 50
The major change: USPSTF proposed reducing the recommended start age for routine screening mammograms from age 50 to age 40. The latest recommendation, which carries a B grade, also calls for screening every other year and sets a cutoff age of 74.
The task force’s A and B ratings indicate strong confidence in the evidence for benefit, meaning that clinicians should encourage their patients to get these services as appropriate.
The influential federal advisory panel last updated these recommendations in 2016. At the time, USPSTF recommended routine screening mammograms starting at age 50, and gave a C grade to starting before that.
In the 2016 recommendations, “we felt a woman could start screening in her 40s depending on how she feels about the harms and benefits in an individualized personal decision,” USPSTF member John Wong, MD, chief of clinical decision making and a primary care physician at Tufts Medical Center in Boston, said in an interview. “In this draft recommendation, we now recommend that all women get screened starting at age 40.”
Two major factors prompted the change, explained Dr. Wong. One is that more women are being diagnosed with breast cancer in their 40s. The other is that a growing body of evidence showing that Black women get breast cancer younger, are more likely to die of breast cancer, and would benefit from earlier screening.
“It is now clear that screening every other year starting at age 40 has the potential to save about 20% more lives among all women and there is even greater potential benefit for Black women, who are much more likely to die from breast cancer,” Dr. Wong said.
The American Cancer Society (ACS) called the draft recommendations a “significant positive change,” while noting that the task force recommendations only apply to women at average risk for breast cancer.
The American College of Radiology (ACR) already recommends yearly mammograms for average risk women starting at age 40. Its latest guidelines on mammography call for women at higher-than-average risk for breast cancer to undergo a risk assessment by age 25 to determine if screening before age 40 is needed.
When asked about the differing views, Debra Monticciolo, MD, division chief for breast imaging at Massachusetts General Hospital, said annual screenings that follow ACR recommendations would save more lives than the every-other-year approach backed by the task force. Dr. Monticciolo also highlighted that the available scientific evidence supports earlier assessment as well as augmented and earlier-than-age-40 screening of many women – particularly Black women.
“These evidence-based updates should spur more-informed doctor–patient conversations and help providers save more lives,” Dr. Monticciolo said in a press release.
Insurance access
Typically, upgrading a USPSTF recommendation from C to B leads to better access and insurance coverage for patients. The Affordable Care Act (ACA) of 2010 requires insurers to cover the cost of services that get A and B recommendations from the USPSTF without charging copays – a mandate intended to promote greater use for highly regarded services.
But Congress created a special workaround that effectively makes the ACA mandate apply to the 2002 task force recommendations on mammography. In those recommendations, the task force gave a B grade to screening mammograms every 1 or 2 years starting at age 40 without an age limit.
Federal lawmakers have sought to provide copay-free access to mammograms for this entire population even when the USPSTF recommendations in 2009 and 2016 gave a C grade to routine screening for women under 50.
Still, “it is important to note that our recommendation is based solely on the science of what works to prevent breast cancer and it is not a recommendation for or against insurance coverage,” the task force acknowledged when unveiling the new draft update. “Coverage decisions involve considerations beyond the evidence about clinical benefit, and in the end, these decisions are the responsibility of payors, regulators, and legislators.”
Uncertainties persist
The new draft recommendations also highlight the persistent gaps in knowledge about the uses of mammography, despite years of widespread use of this screening tool.
The updated draft recommendations emphasize the lack of sufficient evidence to address major areas of concern related to screening and treating Black women, older women, women with dense breasts, and those with ductal carcinoma in situ (DCIS).
The task force called for more research addressing the underlying causes of elevated breast cancer mortality rates among Black women.
The USPSTF also issued an ‘I’ statement for providing women with dense breasts additional screening with breast ultrasound or MRI and for screening women older than 75 for breast cancer. Such statements indicate that the available evidence is lacking, poor quality, or conflicting, and thus the USPSTF can’t assess the benefits and harms or make a recommendation for or against providing the preventive service.
“Nearly half of all women have dense breasts, which increases their risk for breast cancer and means that mammograms may not work as well for them. We need to know more about whether and how additional screening might help women with dense breasts stay healthy,” the task force explained.
The task force also called for more research on approaches to reduce the risk for overdiagnosis and overtreatment for breast lesions, such as DCIS, which are identified through screening.
One analysis – the COMET study – is currently underway to assess whether women could be spared surgery for DCIS and opt for watchful waiting instead.
“If we can find that monitoring them carefully, either with or without some sort of endocrine therapy, is just as effective in keeping patients free of invasive cancer as surgery, then I think we could help to de-escalate treatment for this very low-risk group of patients,” Shelley Hwang, MD, MPH, principal investigator of the COMET study, told this news organization in December.
The task force will accept comments from the public on this draft update through June 5.
A version of this article first appeared on Medscape.com.
The major change: USPSTF proposed reducing the recommended start age for routine screening mammograms from age 50 to age 40. The latest recommendation, which carries a B grade, also calls for screening every other year and sets a cutoff age of 74.
The task force’s A and B ratings indicate strong confidence in the evidence for benefit, meaning that clinicians should encourage their patients to get these services as appropriate.
The influential federal advisory panel last updated these recommendations in 2016. At the time, USPSTF recommended routine screening mammograms starting at age 50, and gave a C grade to starting before that.
In the 2016 recommendations, “we felt a woman could start screening in her 40s depending on how she feels about the harms and benefits in an individualized personal decision,” USPSTF member John Wong, MD, chief of clinical decision making and a primary care physician at Tufts Medical Center in Boston, said in an interview. “In this draft recommendation, we now recommend that all women get screened starting at age 40.”
Two major factors prompted the change, explained Dr. Wong. One is that more women are being diagnosed with breast cancer in their 40s. The other is that a growing body of evidence showing that Black women get breast cancer younger, are more likely to die of breast cancer, and would benefit from earlier screening.
“It is now clear that screening every other year starting at age 40 has the potential to save about 20% more lives among all women and there is even greater potential benefit for Black women, who are much more likely to die from breast cancer,” Dr. Wong said.
The American Cancer Society (ACS) called the draft recommendations a “significant positive change,” while noting that the task force recommendations only apply to women at average risk for breast cancer.
The American College of Radiology (ACR) already recommends yearly mammograms for average risk women starting at age 40. Its latest guidelines on mammography call for women at higher-than-average risk for breast cancer to undergo a risk assessment by age 25 to determine if screening before age 40 is needed.
When asked about the differing views, Debra Monticciolo, MD, division chief for breast imaging at Massachusetts General Hospital, said annual screenings that follow ACR recommendations would save more lives than the every-other-year approach backed by the task force. Dr. Monticciolo also highlighted that the available scientific evidence supports earlier assessment as well as augmented and earlier-than-age-40 screening of many women – particularly Black women.
“These evidence-based updates should spur more-informed doctor–patient conversations and help providers save more lives,” Dr. Monticciolo said in a press release.
Insurance access
Typically, upgrading a USPSTF recommendation from C to B leads to better access and insurance coverage for patients. The Affordable Care Act (ACA) of 2010 requires insurers to cover the cost of services that get A and B recommendations from the USPSTF without charging copays – a mandate intended to promote greater use for highly regarded services.
But Congress created a special workaround that effectively makes the ACA mandate apply to the 2002 task force recommendations on mammography. In those recommendations, the task force gave a B grade to screening mammograms every 1 or 2 years starting at age 40 without an age limit.
Federal lawmakers have sought to provide copay-free access to mammograms for this entire population even when the USPSTF recommendations in 2009 and 2016 gave a C grade to routine screening for women under 50.
Still, “it is important to note that our recommendation is based solely on the science of what works to prevent breast cancer and it is not a recommendation for or against insurance coverage,” the task force acknowledged when unveiling the new draft update. “Coverage decisions involve considerations beyond the evidence about clinical benefit, and in the end, these decisions are the responsibility of payors, regulators, and legislators.”
Uncertainties persist
The new draft recommendations also highlight the persistent gaps in knowledge about the uses of mammography, despite years of widespread use of this screening tool.
The updated draft recommendations emphasize the lack of sufficient evidence to address major areas of concern related to screening and treating Black women, older women, women with dense breasts, and those with ductal carcinoma in situ (DCIS).
The task force called for more research addressing the underlying causes of elevated breast cancer mortality rates among Black women.
The USPSTF also issued an ‘I’ statement for providing women with dense breasts additional screening with breast ultrasound or MRI and for screening women older than 75 for breast cancer. Such statements indicate that the available evidence is lacking, poor quality, or conflicting, and thus the USPSTF can’t assess the benefits and harms or make a recommendation for or against providing the preventive service.
“Nearly half of all women have dense breasts, which increases their risk for breast cancer and means that mammograms may not work as well for them. We need to know more about whether and how additional screening might help women with dense breasts stay healthy,” the task force explained.
The task force also called for more research on approaches to reduce the risk for overdiagnosis and overtreatment for breast lesions, such as DCIS, which are identified through screening.
One analysis – the COMET study – is currently underway to assess whether women could be spared surgery for DCIS and opt for watchful waiting instead.
“If we can find that monitoring them carefully, either with or without some sort of endocrine therapy, is just as effective in keeping patients free of invasive cancer as surgery, then I think we could help to de-escalate treatment for this very low-risk group of patients,” Shelley Hwang, MD, MPH, principal investigator of the COMET study, told this news organization in December.
The task force will accept comments from the public on this draft update through June 5.
A version of this article first appeared on Medscape.com.
The major change: USPSTF proposed reducing the recommended start age for routine screening mammograms from age 50 to age 40. The latest recommendation, which carries a B grade, also calls for screening every other year and sets a cutoff age of 74.
The task force’s A and B ratings indicate strong confidence in the evidence for benefit, meaning that clinicians should encourage their patients to get these services as appropriate.
The influential federal advisory panel last updated these recommendations in 2016. At the time, USPSTF recommended routine screening mammograms starting at age 50, and gave a C grade to starting before that.
In the 2016 recommendations, “we felt a woman could start screening in her 40s depending on how she feels about the harms and benefits in an individualized personal decision,” USPSTF member John Wong, MD, chief of clinical decision making and a primary care physician at Tufts Medical Center in Boston, said in an interview. “In this draft recommendation, we now recommend that all women get screened starting at age 40.”
Two major factors prompted the change, explained Dr. Wong. One is that more women are being diagnosed with breast cancer in their 40s. The other is that a growing body of evidence showing that Black women get breast cancer younger, are more likely to die of breast cancer, and would benefit from earlier screening.
“It is now clear that screening every other year starting at age 40 has the potential to save about 20% more lives among all women and there is even greater potential benefit for Black women, who are much more likely to die from breast cancer,” Dr. Wong said.
The American Cancer Society (ACS) called the draft recommendations a “significant positive change,” while noting that the task force recommendations only apply to women at average risk for breast cancer.
The American College of Radiology (ACR) already recommends yearly mammograms for average risk women starting at age 40. Its latest guidelines on mammography call for women at higher-than-average risk for breast cancer to undergo a risk assessment by age 25 to determine if screening before age 40 is needed.
When asked about the differing views, Debra Monticciolo, MD, division chief for breast imaging at Massachusetts General Hospital, said annual screenings that follow ACR recommendations would save more lives than the every-other-year approach backed by the task force. Dr. Monticciolo also highlighted that the available scientific evidence supports earlier assessment as well as augmented and earlier-than-age-40 screening of many women – particularly Black women.
“These evidence-based updates should spur more-informed doctor–patient conversations and help providers save more lives,” Dr. Monticciolo said in a press release.
Insurance access
Typically, upgrading a USPSTF recommendation from C to B leads to better access and insurance coverage for patients. The Affordable Care Act (ACA) of 2010 requires insurers to cover the cost of services that get A and B recommendations from the USPSTF without charging copays – a mandate intended to promote greater use for highly regarded services.
But Congress created a special workaround that effectively makes the ACA mandate apply to the 2002 task force recommendations on mammography. In those recommendations, the task force gave a B grade to screening mammograms every 1 or 2 years starting at age 40 without an age limit.
Federal lawmakers have sought to provide copay-free access to mammograms for this entire population even when the USPSTF recommendations in 2009 and 2016 gave a C grade to routine screening for women under 50.
Still, “it is important to note that our recommendation is based solely on the science of what works to prevent breast cancer and it is not a recommendation for or against insurance coverage,” the task force acknowledged when unveiling the new draft update. “Coverage decisions involve considerations beyond the evidence about clinical benefit, and in the end, these decisions are the responsibility of payors, regulators, and legislators.”
Uncertainties persist
The new draft recommendations also highlight the persistent gaps in knowledge about the uses of mammography, despite years of widespread use of this screening tool.
The updated draft recommendations emphasize the lack of sufficient evidence to address major areas of concern related to screening and treating Black women, older women, women with dense breasts, and those with ductal carcinoma in situ (DCIS).
The task force called for more research addressing the underlying causes of elevated breast cancer mortality rates among Black women.
The USPSTF also issued an ‘I’ statement for providing women with dense breasts additional screening with breast ultrasound or MRI and for screening women older than 75 for breast cancer. Such statements indicate that the available evidence is lacking, poor quality, or conflicting, and thus the USPSTF can’t assess the benefits and harms or make a recommendation for or against providing the preventive service.
“Nearly half of all women have dense breasts, which increases their risk for breast cancer and means that mammograms may not work as well for them. We need to know more about whether and how additional screening might help women with dense breasts stay healthy,” the task force explained.
The task force also called for more research on approaches to reduce the risk for overdiagnosis and overtreatment for breast lesions, such as DCIS, which are identified through screening.
One analysis – the COMET study – is currently underway to assess whether women could be spared surgery for DCIS and opt for watchful waiting instead.
“If we can find that monitoring them carefully, either with or without some sort of endocrine therapy, is just as effective in keeping patients free of invasive cancer as surgery, then I think we could help to de-escalate treatment for this very low-risk group of patients,” Shelley Hwang, MD, MPH, principal investigator of the COMET study, told this news organization in December.
The task force will accept comments from the public on this draft update through June 5.
A version of this article first appeared on Medscape.com.
Panel backs limited new olaparib use in prostate cancer
A panel of independent advisers recently almost unanimously recommended to restrict a new indication for olaparib (Lynparza) alongside abiraterone (Zytiga) in patients with metastatic castration-resistant prostate cancer.
On April 28, members of the Oncologic Drugs Advisory Committee voted 11 to 1, with one abstention, that only patients whose tumors have a BRCA mutation should receive olaparib as part of the combination first-line treatment for metastatic castration-resistant prostate cancer.
Olaparib is already cleared by the Food and Drug Administration for various ovarian, breast, and pancreatic cancer indications as well as later-line use in certain more advanced prostate cancers. AstraZeneca recently applied for an additional, broad indication for the poly (ADP-ribose) polymerase (PARP) inhibitor as an initial therapy that would include patients without BRCA or homologous recombination repair (HRR) mutations.
In reviewing the application, a phrase agency staff used in their briefing document.
Given these concerns, the FDA reviewers asked the independent ODAC panel to vote on the following question: “As FDA reviews the proposed indication for olaparib in combination with abiraterone for initial treatment of [metastatic castration-resistant prostate cancer], should the indication be restricted to patients whose tumors have a BRCA mutation?”
The ODAC panel voted 11 to 1 in favor of a restricted expansion of olaparib plus abiraterone and prednisone or prednisolone to patients whose tumors have a BRCA mutation. One member – Ravi A. Madan, MD, of the National Cancer Institute – abstained. The FDA staff asked panelists to abstain if they felt the combination treatment should not be approved for any indication.
Overall, the ODAC panel agreed with the FDA staff’s criticism of the research supporting the application: the PROpel study. The trial randomized 796 patients with previously untreated metastatic castration-resistant prostate cancer to olaparib plus abiraterone or abiraterone plus placebo. The median time for radiographic progression-free survival – the study’s primary endpoint – was nearly 25 months in the olaparib group versus 16.6 months in the placebo group.
The combination also demonstrated a 19% reduced risk of death, which was not statistically significant (hazard ratio, 0.81; P = .0544), and a median improvement of 7.4 months in the combination arm (42.1 vs. 34.7 months).
Although the study met its primary endpoint, the results were difficult to interpret given the lack of information about genetic variability in the participants’ tumors. Participants were not prospectively assessed for either BRCA or HRR status. But given the importance of BRCA status as a predictive biomarker for PARP inhibitor efficacy, “this trial design would be considered inappropriate today as the biomarker should have been prospectively evaluated,” the FDA wrote in its briefing document.
In a post hoc analysis performed by the FDA, the agency found that BRCA-positive patients accounted for most of the survival benefit of the combination, though made up only 11% of the PROpel population.
That meant the ODAC members were being asked to evaluate a drug based on “suboptimal data” resulting from a “suboptimal study design,” said Dr. Madan, who is head of the prostate cancer clinical research section in the NCI’s Center for Cancer Research.
Dr. Madan also emphasized concerns the FDA raised about the potential harms for patients whose prostate cancer was not tied to BRCA mutations. In the FDA’s briefing document, the agency said patients treated in the first-line metastatic castration-resistant prostate cancer setting generally have few symptoms at baseline. Adding olaparib among patients lacking the BRCA mutation could expose them to a drug with known side effects but minimal chance to help them.
The PROpel trial also found that patients who received olaparib and abiraterone experienced greater toxicity than those who received abiraterone. These adverse events included venous thromboembolic events, myelosuppression, requirement for blood transfusions, nausea, vomiting, and diarrhea.
Although the FDA is not compelled to follow the recommendations of its advisory committees, it often does.
AstraZeneca expressed some disappointment about the recommendation in a press release. Susan Galbraith, executive vice president of Oncology R&D at AstraZeneca, said, “while we are pleased with the recognition of the benefit of Lynparza plus abiraterone for patients with BRCA-mutated metastatic castration-resistant prostate cancer, we are disappointed with the outcome of today’s ODAC meeting. We strongly believe in the results of the PROpel trial, which demonstrated the clinically meaningful benefit for this combination in a broad population of patients regardless of biomarker status.”
One ODAC member, Jorge J. Nieva, MD, did support the expanded approval for olaparib, casting the single “no” vote. Dr. Nieva, an oncologist at the University of Southern California, Los Angeles, disagreed with the FDA’s question about limiting use of the olaparib-abiraterone combination to patients with known BRCA mutations, citing the positive result from the PROpel trial.
People are aware that olaparib provides a great deal more benefit in the BRCA-positive group and may give “only minimal benefit if these tests are not positive,” but “these risks and benefits can be addressed at the patient and physician level,” he said.
But Terrence M. Kungel, MBA, who served as ODAC’s patient representative for the meeting, offered a counterpoint to Dr. Nieva’s assessment. Patients now often struggle to assess the options available to them and then pay for these medicines, with financial toxicity affecting many people with cancer.
“Prostate cancer patients need more treatments that are effective, not more choices,” said Mr. Kungel, who voted with the majority.
A version of this article first appeared on Medscape.com.
A panel of independent advisers recently almost unanimously recommended to restrict a new indication for olaparib (Lynparza) alongside abiraterone (Zytiga) in patients with metastatic castration-resistant prostate cancer.
On April 28, members of the Oncologic Drugs Advisory Committee voted 11 to 1, with one abstention, that only patients whose tumors have a BRCA mutation should receive olaparib as part of the combination first-line treatment for metastatic castration-resistant prostate cancer.
Olaparib is already cleared by the Food and Drug Administration for various ovarian, breast, and pancreatic cancer indications as well as later-line use in certain more advanced prostate cancers. AstraZeneca recently applied for an additional, broad indication for the poly (ADP-ribose) polymerase (PARP) inhibitor as an initial therapy that would include patients without BRCA or homologous recombination repair (HRR) mutations.
In reviewing the application, a phrase agency staff used in their briefing document.
Given these concerns, the FDA reviewers asked the independent ODAC panel to vote on the following question: “As FDA reviews the proposed indication for olaparib in combination with abiraterone for initial treatment of [metastatic castration-resistant prostate cancer], should the indication be restricted to patients whose tumors have a BRCA mutation?”
The ODAC panel voted 11 to 1 in favor of a restricted expansion of olaparib plus abiraterone and prednisone or prednisolone to patients whose tumors have a BRCA mutation. One member – Ravi A. Madan, MD, of the National Cancer Institute – abstained. The FDA staff asked panelists to abstain if they felt the combination treatment should not be approved for any indication.
Overall, the ODAC panel agreed with the FDA staff’s criticism of the research supporting the application: the PROpel study. The trial randomized 796 patients with previously untreated metastatic castration-resistant prostate cancer to olaparib plus abiraterone or abiraterone plus placebo. The median time for radiographic progression-free survival – the study’s primary endpoint – was nearly 25 months in the olaparib group versus 16.6 months in the placebo group.
The combination also demonstrated a 19% reduced risk of death, which was not statistically significant (hazard ratio, 0.81; P = .0544), and a median improvement of 7.4 months in the combination arm (42.1 vs. 34.7 months).
Although the study met its primary endpoint, the results were difficult to interpret given the lack of information about genetic variability in the participants’ tumors. Participants were not prospectively assessed for either BRCA or HRR status. But given the importance of BRCA status as a predictive biomarker for PARP inhibitor efficacy, “this trial design would be considered inappropriate today as the biomarker should have been prospectively evaluated,” the FDA wrote in its briefing document.
In a post hoc analysis performed by the FDA, the agency found that BRCA-positive patients accounted for most of the survival benefit of the combination, though made up only 11% of the PROpel population.
That meant the ODAC members were being asked to evaluate a drug based on “suboptimal data” resulting from a “suboptimal study design,” said Dr. Madan, who is head of the prostate cancer clinical research section in the NCI’s Center for Cancer Research.
Dr. Madan also emphasized concerns the FDA raised about the potential harms for patients whose prostate cancer was not tied to BRCA mutations. In the FDA’s briefing document, the agency said patients treated in the first-line metastatic castration-resistant prostate cancer setting generally have few symptoms at baseline. Adding olaparib among patients lacking the BRCA mutation could expose them to a drug with known side effects but minimal chance to help them.
The PROpel trial also found that patients who received olaparib and abiraterone experienced greater toxicity than those who received abiraterone. These adverse events included venous thromboembolic events, myelosuppression, requirement for blood transfusions, nausea, vomiting, and diarrhea.
Although the FDA is not compelled to follow the recommendations of its advisory committees, it often does.
AstraZeneca expressed some disappointment about the recommendation in a press release. Susan Galbraith, executive vice president of Oncology R&D at AstraZeneca, said, “while we are pleased with the recognition of the benefit of Lynparza plus abiraterone for patients with BRCA-mutated metastatic castration-resistant prostate cancer, we are disappointed with the outcome of today’s ODAC meeting. We strongly believe in the results of the PROpel trial, which demonstrated the clinically meaningful benefit for this combination in a broad population of patients regardless of biomarker status.”
One ODAC member, Jorge J. Nieva, MD, did support the expanded approval for olaparib, casting the single “no” vote. Dr. Nieva, an oncologist at the University of Southern California, Los Angeles, disagreed with the FDA’s question about limiting use of the olaparib-abiraterone combination to patients with known BRCA mutations, citing the positive result from the PROpel trial.
People are aware that olaparib provides a great deal more benefit in the BRCA-positive group and may give “only minimal benefit if these tests are not positive,” but “these risks and benefits can be addressed at the patient and physician level,” he said.
But Terrence M. Kungel, MBA, who served as ODAC’s patient representative for the meeting, offered a counterpoint to Dr. Nieva’s assessment. Patients now often struggle to assess the options available to them and then pay for these medicines, with financial toxicity affecting many people with cancer.
“Prostate cancer patients need more treatments that are effective, not more choices,” said Mr. Kungel, who voted with the majority.
A version of this article first appeared on Medscape.com.
A panel of independent advisers recently almost unanimously recommended to restrict a new indication for olaparib (Lynparza) alongside abiraterone (Zytiga) in patients with metastatic castration-resistant prostate cancer.
On April 28, members of the Oncologic Drugs Advisory Committee voted 11 to 1, with one abstention, that only patients whose tumors have a BRCA mutation should receive olaparib as part of the combination first-line treatment for metastatic castration-resistant prostate cancer.
Olaparib is already cleared by the Food and Drug Administration for various ovarian, breast, and pancreatic cancer indications as well as later-line use in certain more advanced prostate cancers. AstraZeneca recently applied for an additional, broad indication for the poly (ADP-ribose) polymerase (PARP) inhibitor as an initial therapy that would include patients without BRCA or homologous recombination repair (HRR) mutations.
In reviewing the application, a phrase agency staff used in their briefing document.
Given these concerns, the FDA reviewers asked the independent ODAC panel to vote on the following question: “As FDA reviews the proposed indication for olaparib in combination with abiraterone for initial treatment of [metastatic castration-resistant prostate cancer], should the indication be restricted to patients whose tumors have a BRCA mutation?”
The ODAC panel voted 11 to 1 in favor of a restricted expansion of olaparib plus abiraterone and prednisone or prednisolone to patients whose tumors have a BRCA mutation. One member – Ravi A. Madan, MD, of the National Cancer Institute – abstained. The FDA staff asked panelists to abstain if they felt the combination treatment should not be approved for any indication.
Overall, the ODAC panel agreed with the FDA staff’s criticism of the research supporting the application: the PROpel study. The trial randomized 796 patients with previously untreated metastatic castration-resistant prostate cancer to olaparib plus abiraterone or abiraterone plus placebo. The median time for radiographic progression-free survival – the study’s primary endpoint – was nearly 25 months in the olaparib group versus 16.6 months in the placebo group.
The combination also demonstrated a 19% reduced risk of death, which was not statistically significant (hazard ratio, 0.81; P = .0544), and a median improvement of 7.4 months in the combination arm (42.1 vs. 34.7 months).
Although the study met its primary endpoint, the results were difficult to interpret given the lack of information about genetic variability in the participants’ tumors. Participants were not prospectively assessed for either BRCA or HRR status. But given the importance of BRCA status as a predictive biomarker for PARP inhibitor efficacy, “this trial design would be considered inappropriate today as the biomarker should have been prospectively evaluated,” the FDA wrote in its briefing document.
In a post hoc analysis performed by the FDA, the agency found that BRCA-positive patients accounted for most of the survival benefit of the combination, though made up only 11% of the PROpel population.
That meant the ODAC members were being asked to evaluate a drug based on “suboptimal data” resulting from a “suboptimal study design,” said Dr. Madan, who is head of the prostate cancer clinical research section in the NCI’s Center for Cancer Research.
Dr. Madan also emphasized concerns the FDA raised about the potential harms for patients whose prostate cancer was not tied to BRCA mutations. In the FDA’s briefing document, the agency said patients treated in the first-line metastatic castration-resistant prostate cancer setting generally have few symptoms at baseline. Adding olaparib among patients lacking the BRCA mutation could expose them to a drug with known side effects but minimal chance to help them.
The PROpel trial also found that patients who received olaparib and abiraterone experienced greater toxicity than those who received abiraterone. These adverse events included venous thromboembolic events, myelosuppression, requirement for blood transfusions, nausea, vomiting, and diarrhea.
Although the FDA is not compelled to follow the recommendations of its advisory committees, it often does.
AstraZeneca expressed some disappointment about the recommendation in a press release. Susan Galbraith, executive vice president of Oncology R&D at AstraZeneca, said, “while we are pleased with the recognition of the benefit of Lynparza plus abiraterone for patients with BRCA-mutated metastatic castration-resistant prostate cancer, we are disappointed with the outcome of today’s ODAC meeting. We strongly believe in the results of the PROpel trial, which demonstrated the clinically meaningful benefit for this combination in a broad population of patients regardless of biomarker status.”
One ODAC member, Jorge J. Nieva, MD, did support the expanded approval for olaparib, casting the single “no” vote. Dr. Nieva, an oncologist at the University of Southern California, Los Angeles, disagreed with the FDA’s question about limiting use of the olaparib-abiraterone combination to patients with known BRCA mutations, citing the positive result from the PROpel trial.
People are aware that olaparib provides a great deal more benefit in the BRCA-positive group and may give “only minimal benefit if these tests are not positive,” but “these risks and benefits can be addressed at the patient and physician level,” he said.
But Terrence M. Kungel, MBA, who served as ODAC’s patient representative for the meeting, offered a counterpoint to Dr. Nieva’s assessment. Patients now often struggle to assess the options available to them and then pay for these medicines, with financial toxicity affecting many people with cancer.
“Prostate cancer patients need more treatments that are effective, not more choices,” said Mr. Kungel, who voted with the majority.
A version of this article first appeared on Medscape.com.
New Medicare rule streamlines prior authorization in Medicare Advantage plans
A new federal rule seeks to reduce Medicare Advantage insurance plans’ prior authorization burdens on physicians while also ensuring that enrollees have the same access to necessary care that they would receive under traditional fee-for-service Medicare.
The prior authorization changes, announced this week, are part of the Centers for Medicare & Medicaid Services’ 2024 update of policy changes for Medicare Advantage and Part D pharmacy plans
Medicare Advantage plans’ business practices have raised significant concerns in recent years. More than 28 million Americans were enrolled in a Medicare Advantage plan in 2022, which is nearly half of all Medicare enrollees, according to the Kaiser Family Foundation.
Medicare pays a fixed amount per enrollee per year to these privately run managed care plans, in contrast to traditional fee-for-service Medicare. Medicare Advantage plans have been criticized for aggressive marketing, for overbilling the federal government for care, and for using prior authorization to inappropriately deny needed care to patients.
About 13% of prior authorization requests that are denied by Medicare Advantage plans actually met Medicare coverage rules and should have been approved, the Office of the Inspector General at the U.S. Department of Health & Human Services reported in 2022.
The newly finalized rule now requires Medicare Advantage plans to do the following.
- Ensure that a prior authorization approval, once granted, remains valid for as long as medically necessary to avoid disruptions in care.
- Conduct an annual review of utilization management policies.
- Ensure that coverage denials based on medical necessity be reviewed by health care professionals with relevant expertise before a denial can be issued.
Physician groups welcomed the changes. In a statement, the American Medical Association said that an initial reading of the rule suggested CMS had “taken important steps toward right-sizing the prior authorization process.”
The Medical Group Management Association praised CMS in a statement for having limited “dangerous disruptions and delays to necessary patient care” resulting from the cumbersome processes of prior approval. With the new rules, CMS will provide greater consistency across Advantage plans as well as traditional Medicare, said Anders Gilberg, MGMA’s senior vice president of government affairs, in a statement.
Peer consideration
The final rule did disappoint physician groups in one key way. CMS rebuffed requests to have CMS require Advantage plans to use reviewers of the same specialty as treating physicians in handling disputes about prior authorization. CMS said it expects plans to exercise judgment in finding reviewers with “sufficient expertise to make an informed and supportable decision.”
“In some instances, we expect that plans will use a physician or other health care professional of the same specialty or subspecialty as the treating physician,” CMS said. “In other instances, we expect that plans will utilize a reviewer with specialized training, certification, or clinical experience in the applicable field of medicine.”
Medicare Advantage marketing ‘sowing confusion’
With this final rule, CMS also sought to protect consumers from “potentially misleading marketing practices” used in promoting Medicare Advantage and Part D prescription drug plans.
The agency said it had received complaints about people who have received official-looking promotional materials for Medicare that directed them not to government sources of information but to Medicare Advantage and Part D plans or their agents and brokers.
Ads now must mention a specific plan name, and they cannot use the Medicare name, CMS logo, Medicare card, or other government information in a misleading way, CMS said.
“CMS can see no value or purpose in a non-governmental entity’s use of the Medicare logo or HHS logo except for the express purpose of sowing confusion and misrepresenting itself as the government,” the agency said.
A version of this article first appeared on Medscape.com.
A new federal rule seeks to reduce Medicare Advantage insurance plans’ prior authorization burdens on physicians while also ensuring that enrollees have the same access to necessary care that they would receive under traditional fee-for-service Medicare.
The prior authorization changes, announced this week, are part of the Centers for Medicare & Medicaid Services’ 2024 update of policy changes for Medicare Advantage and Part D pharmacy plans
Medicare Advantage plans’ business practices have raised significant concerns in recent years. More than 28 million Americans were enrolled in a Medicare Advantage plan in 2022, which is nearly half of all Medicare enrollees, according to the Kaiser Family Foundation.
Medicare pays a fixed amount per enrollee per year to these privately run managed care plans, in contrast to traditional fee-for-service Medicare. Medicare Advantage plans have been criticized for aggressive marketing, for overbilling the federal government for care, and for using prior authorization to inappropriately deny needed care to patients.
About 13% of prior authorization requests that are denied by Medicare Advantage plans actually met Medicare coverage rules and should have been approved, the Office of the Inspector General at the U.S. Department of Health & Human Services reported in 2022.
The newly finalized rule now requires Medicare Advantage plans to do the following.
- Ensure that a prior authorization approval, once granted, remains valid for as long as medically necessary to avoid disruptions in care.
- Conduct an annual review of utilization management policies.
- Ensure that coverage denials based on medical necessity be reviewed by health care professionals with relevant expertise before a denial can be issued.
Physician groups welcomed the changes. In a statement, the American Medical Association said that an initial reading of the rule suggested CMS had “taken important steps toward right-sizing the prior authorization process.”
The Medical Group Management Association praised CMS in a statement for having limited “dangerous disruptions and delays to necessary patient care” resulting from the cumbersome processes of prior approval. With the new rules, CMS will provide greater consistency across Advantage plans as well as traditional Medicare, said Anders Gilberg, MGMA’s senior vice president of government affairs, in a statement.
Peer consideration
The final rule did disappoint physician groups in one key way. CMS rebuffed requests to have CMS require Advantage plans to use reviewers of the same specialty as treating physicians in handling disputes about prior authorization. CMS said it expects plans to exercise judgment in finding reviewers with “sufficient expertise to make an informed and supportable decision.”
“In some instances, we expect that plans will use a physician or other health care professional of the same specialty or subspecialty as the treating physician,” CMS said. “In other instances, we expect that plans will utilize a reviewer with specialized training, certification, or clinical experience in the applicable field of medicine.”
Medicare Advantage marketing ‘sowing confusion’
With this final rule, CMS also sought to protect consumers from “potentially misleading marketing practices” used in promoting Medicare Advantage and Part D prescription drug plans.
The agency said it had received complaints about people who have received official-looking promotional materials for Medicare that directed them not to government sources of information but to Medicare Advantage and Part D plans or their agents and brokers.
Ads now must mention a specific plan name, and they cannot use the Medicare name, CMS logo, Medicare card, or other government information in a misleading way, CMS said.
“CMS can see no value or purpose in a non-governmental entity’s use of the Medicare logo or HHS logo except for the express purpose of sowing confusion and misrepresenting itself as the government,” the agency said.
A version of this article first appeared on Medscape.com.
A new federal rule seeks to reduce Medicare Advantage insurance plans’ prior authorization burdens on physicians while also ensuring that enrollees have the same access to necessary care that they would receive under traditional fee-for-service Medicare.
The prior authorization changes, announced this week, are part of the Centers for Medicare & Medicaid Services’ 2024 update of policy changes for Medicare Advantage and Part D pharmacy plans
Medicare Advantage plans’ business practices have raised significant concerns in recent years. More than 28 million Americans were enrolled in a Medicare Advantage plan in 2022, which is nearly half of all Medicare enrollees, according to the Kaiser Family Foundation.
Medicare pays a fixed amount per enrollee per year to these privately run managed care plans, in contrast to traditional fee-for-service Medicare. Medicare Advantage plans have been criticized for aggressive marketing, for overbilling the federal government for care, and for using prior authorization to inappropriately deny needed care to patients.
About 13% of prior authorization requests that are denied by Medicare Advantage plans actually met Medicare coverage rules and should have been approved, the Office of the Inspector General at the U.S. Department of Health & Human Services reported in 2022.
The newly finalized rule now requires Medicare Advantage plans to do the following.
- Ensure that a prior authorization approval, once granted, remains valid for as long as medically necessary to avoid disruptions in care.
- Conduct an annual review of utilization management policies.
- Ensure that coverage denials based on medical necessity be reviewed by health care professionals with relevant expertise before a denial can be issued.
Physician groups welcomed the changes. In a statement, the American Medical Association said that an initial reading of the rule suggested CMS had “taken important steps toward right-sizing the prior authorization process.”
The Medical Group Management Association praised CMS in a statement for having limited “dangerous disruptions and delays to necessary patient care” resulting from the cumbersome processes of prior approval. With the new rules, CMS will provide greater consistency across Advantage plans as well as traditional Medicare, said Anders Gilberg, MGMA’s senior vice president of government affairs, in a statement.
Peer consideration
The final rule did disappoint physician groups in one key way. CMS rebuffed requests to have CMS require Advantage plans to use reviewers of the same specialty as treating physicians in handling disputes about prior authorization. CMS said it expects plans to exercise judgment in finding reviewers with “sufficient expertise to make an informed and supportable decision.”
“In some instances, we expect that plans will use a physician or other health care professional of the same specialty or subspecialty as the treating physician,” CMS said. “In other instances, we expect that plans will utilize a reviewer with specialized training, certification, or clinical experience in the applicable field of medicine.”
Medicare Advantage marketing ‘sowing confusion’
With this final rule, CMS also sought to protect consumers from “potentially misleading marketing practices” used in promoting Medicare Advantage and Part D prescription drug plans.
The agency said it had received complaints about people who have received official-looking promotional materials for Medicare that directed them not to government sources of information but to Medicare Advantage and Part D plans or their agents and brokers.
Ads now must mention a specific plan name, and they cannot use the Medicare name, CMS logo, Medicare card, or other government information in a misleading way, CMS said.
“CMS can see no value or purpose in a non-governmental entity’s use of the Medicare logo or HHS logo except for the express purpose of sowing confusion and misrepresenting itself as the government,” the agency said.
A version of this article first appeared on Medscape.com.
CRC blood tests: A future without screening colonoscopies?
Most recently, Guardant Health announced the completion of its U.S. premarket approval application for its Shield blood test to screen for CRC. Approval by the Food and Drug Administration would position Guardant to later secure Medicare coverage for its test.
Rival companies, including CellMax Life, Freenome, and Exact Sciences, which already offers the stool-based Cologuard product, are pursuing similar paths in their development of blood tests for CRC.
If these companies succeed, clinicians and patients could have a choice of several FDA-approved tests in a few years.
“They’re coming, and they will be increasingly widely used,” said David A. Johnson, MD, professor of medicine and chief of gastroenterology at Eastern Virginia Medical School, Norfolk, who earlier in his career helped win broader insurance coverage of colonoscopy.
Blood tests for CRC have the potential to cause a shift in screening for colon cancer.
Screening colonoscopies ultimately could be largely phased out in the years ahead in favor of highly sensitive noninvasive tests, if the blood tests do as well as expected, said John M. Carethers, MD, AGAF, president of the American Gastroenterological Association.
‘Holy grail?’
“A blood test for cancer screening has been the ‘holy grail’ ever since the carcinoembryonic antigen blood test in the 1960s was claimed to have nearly 100% sensitivity and specificity – but turned out not to – for colorectal cancer,” wrote David F. Ransohoff, MD, a gastroenterologist at the University of North Carolina at Chapel Hill, in a 2021 article. Dr. Ransohoff has studied noninvasive CRC screening for decades.
There is a great allure in the idea of such multi-cancer detection (MCD) tests. “MCD technology offers the potential to detect asymptomatic cancer at several organ sites with a simple blood test, often called a liquid biopsy, ” according to a National Cancer Institute FY24 budget request report.
Several companies are selling MCD tests, some of which include CRC components. Among the best-known MCD tests now sold is Grail’s Galleri. At this time, however, the Galleri test, which tests for 50 types of cancer, should be used in addition to recommended colon cancer screening tests, such as colonoscopy, the company’s website says.
Guardant has also noted that its CRC-specific blood test should only complement screening tools, including colonoscopy, not replace them.
The prospect of phasing out more commonly used CRC screening – such as colonoscopy – may be appealing, but it would require a big shift for a field in which procedures have dominated. According to a report from the Centers for Disease Control and Prevention, in 2018, 67% of U.S. adults aged 50-75 years met the U.S. Preventive Services Task Force recommendations for CRC screening, and overall, 60.6% had a colonoscopy in the past 10 years.
Still, the NCI and the FDA have signaled the potential they see in MCD tests. The NCI highlighted its plans to aid MCD test development as part of its budget request for fiscal year 2024. The NCI is preparing to launch a 4-year pilot study for MCD tests to enroll 24,000 people aged 45-70 years. The study is intended as groundwork for a randomized controlled trial that will enroll 225,000 people.
The FDA has shown an interest in helping companies bring blood tests for cancer detection to market through its breakthrough device designation – a sign that the FDA places great priority on a product and seeks to streamline the application and review process.
CellMax Life appears to be the only CRC-specific screening blood test to have received a breakthrough device designation from the FDA, Atul Sharan, MS, MBA, cofounder and chief executive officer of CellMax Life, said in an email.
Lance Baldo, MD, Freenome’s chief medical officer, said in an interview that the FDA may be reviewing parts of their application in 2024, allowing for a potential 2025 launch of a blood test for asymptomatic people at average risk for CRC.
A spotty track record
Before anyone gets too excited about the prospect of phasing out screening colonoscopy, it’s important to remember that CRC blood tests have proven disappointing in the past.
Germany’s Epigenomics, for example, secured the first FDA approval for a CRC blood test, Epi ProColon, in 2016. But the company did not receive Medicare coverage for the test. In a 2021 memo explaining the decision, the Centers for Medicare & Medicaid Services noted that, given more reliable alternatives, including stool-based tests, the Epi ProColon would result in harm to some patients.
CMS also does not cover Grail’s blood test, which has a list price of $949, though the company has secured reimbursement arrangements with several self-insured employers and insurers, such as Point32Health.
But CMS officials have acknowledged the strong interest in CRC blood tests.
In that 2021 memo, the agency also outlined its requirements for Medicare coverage. CMS said it will cover blood-based screening tests for certain patients if these products meet the following standards:
- Receive FDA market authorization with an indication for CRC screening.
- Have proven test performance characteristics for a blood-based screening test with sensitivity of at least 74% and specificity of at least 90% in the detection of CRC, compared with the recognized standard (which at this time is colonoscopy) as minimal threshold levels, based on the pivotal studies included in the FDA labeling.
In February 2023, CellLife Max presented data at ASCO Gastrointestinal Cancers Symposium that its blood test had sensitivity of 92.1% for detection of CRC and 54.5% for detection of advanced adenomas, at 91% specificity.
Prior to that, in December 2022, Guardant issued a press release with study results that met the CMS standard. The test had sensitivity of 83% in detecting individuals with CRC. Specificity was 90%, the company said. That translates to a false positive rate of just 10%.
While such results look promising, Asad Umar, DVM, PhD, the chief of gastrointestinal and other cancers at NCI’s division of cancer prevention, said physicians should be cautious when giving advice or answering questions about MCD tests, given limited data from prospective studies about their effect on health outcomes.
Even among physicians already using some MCD tests to screen patients, there is a lot of concern about false-positive results that require diagnostic workup and false negative results that lead to a false sense of assurance, Dr. Umar said.
“Screening is a process and not just a test. The process involves follow-up testing for any positive test findings,” Dr. Umar said. “At this point, doctors should inform patients that there is not sufficient data to know how best to use these tests.”
Hurdles to broad acceptance
For companies seeking broad acceptance of a CRC blood test, two of the three major steps needed are securing FDA approval and Medicare coverage. The last step would be getting an A or B recommendation from the USPSTF, which would mandate coverage by health plans.
This is the “big trifecta,” Dr. Baldo said.
In the USPSTF’s current colon cancer screening recommendations, issued in 2021, it gave an A grade for CRC screening for adults aged 50-75 years and a B grade for those aged 45-49 years.
The USPSTF’s recommended forms of screening include colonoscopy, high-sensitivity guaiac fecal occult blood (gFOBT), fecal immunochemical test (FIT), flexible sigmoidoscopy (FS), stool DNA, and/or computed tomographic colonography (CTC).
The USPSTF says more research is needed to establish the accuracy and effectiveness of emerging screening technologies, such as blood or serum tests.
If CRC blood tests eventually win FDA approval, the USPSTF would likely provide guidance to clinicians on how patients can use them as a screening option.
Dr. Ransohoff noted that the mission of the USPSTF is different from that of the FDA and CMS. The FDA’s approach on medical tests is to consider overall safety and efficacy, as does CMS, but neither agency makes recommendations, nor does it perform its own rigorous quantitative assessment of benefit versus harm. The USPSTF, however, does its own detailed evidence-based reviews of the benefit versus harm of products, Dr. Ransohoff said.
“To me, the Task Force is the gold standard,” Dr. Ransohoff said. “You have to jump through the hoops with the FDA and CMS for making claims, to enable use, and to help get payment. But the Task Force looks at the choices and the consequences in a quantitative way and makes specific practice recommendations.”
What the future may hold
Dr. Carethers sees a future in which highly sensitive blood tests are able to largely replace screening colonoscopies. He said that colonoscopies would be used for people who are most in need of diagnosis and treatment. Dr. Carethers addressed these points during an AGA podcast released in January 2023.
In 20-25 years, colonoscopies may be only a therapeutic procedure, much like endoscopic retrograde cholangiopancreatography is now, Dr. Carethers added.
Even if CRC-specific blood tests prove to be effective screening tools, Dr. Ransohoff stressed that colonoscopy will survive. Many people will eventually need to undergo colonoscopy as a diagnostic procedure following a positive blood-based test result, and some may also opt for screening colonoscopies in lieu of frequent blood tests.
And, overall, physicians and patients will need to weigh the trade-offs of a noninvasive test that can only diagnose CRC versus a screening colonoscopy that offers preventative treatment as well.
“The best intent for screening is prevention of cancer, not detection of cancer,” said Dr. Johnson.
Dr. Carethers, Dr. Johnson, and Dr. Ransohoff reporting having no relevant financial conflicts of interest.
A version of this article originally appeared on Medscape.com.
Most recently, Guardant Health announced the completion of its U.S. premarket approval application for its Shield blood test to screen for CRC. Approval by the Food and Drug Administration would position Guardant to later secure Medicare coverage for its test.
Rival companies, including CellMax Life, Freenome, and Exact Sciences, which already offers the stool-based Cologuard product, are pursuing similar paths in their development of blood tests for CRC.
If these companies succeed, clinicians and patients could have a choice of several FDA-approved tests in a few years.
“They’re coming, and they will be increasingly widely used,” said David A. Johnson, MD, professor of medicine and chief of gastroenterology at Eastern Virginia Medical School, Norfolk, who earlier in his career helped win broader insurance coverage of colonoscopy.
Blood tests for CRC have the potential to cause a shift in screening for colon cancer.
Screening colonoscopies ultimately could be largely phased out in the years ahead in favor of highly sensitive noninvasive tests, if the blood tests do as well as expected, said John M. Carethers, MD, AGAF, president of the American Gastroenterological Association.
‘Holy grail?’
“A blood test for cancer screening has been the ‘holy grail’ ever since the carcinoembryonic antigen blood test in the 1960s was claimed to have nearly 100% sensitivity and specificity – but turned out not to – for colorectal cancer,” wrote David F. Ransohoff, MD, a gastroenterologist at the University of North Carolina at Chapel Hill, in a 2021 article. Dr. Ransohoff has studied noninvasive CRC screening for decades.
There is a great allure in the idea of such multi-cancer detection (MCD) tests. “MCD technology offers the potential to detect asymptomatic cancer at several organ sites with a simple blood test, often called a liquid biopsy, ” according to a National Cancer Institute FY24 budget request report.
Several companies are selling MCD tests, some of which include CRC components. Among the best-known MCD tests now sold is Grail’s Galleri. At this time, however, the Galleri test, which tests for 50 types of cancer, should be used in addition to recommended colon cancer screening tests, such as colonoscopy, the company’s website says.
Guardant has also noted that its CRC-specific blood test should only complement screening tools, including colonoscopy, not replace them.
The prospect of phasing out more commonly used CRC screening – such as colonoscopy – may be appealing, but it would require a big shift for a field in which procedures have dominated. According to a report from the Centers for Disease Control and Prevention, in 2018, 67% of U.S. adults aged 50-75 years met the U.S. Preventive Services Task Force recommendations for CRC screening, and overall, 60.6% had a colonoscopy in the past 10 years.
Still, the NCI and the FDA have signaled the potential they see in MCD tests. The NCI highlighted its plans to aid MCD test development as part of its budget request for fiscal year 2024. The NCI is preparing to launch a 4-year pilot study for MCD tests to enroll 24,000 people aged 45-70 years. The study is intended as groundwork for a randomized controlled trial that will enroll 225,000 people.
The FDA has shown an interest in helping companies bring blood tests for cancer detection to market through its breakthrough device designation – a sign that the FDA places great priority on a product and seeks to streamline the application and review process.
CellMax Life appears to be the only CRC-specific screening blood test to have received a breakthrough device designation from the FDA, Atul Sharan, MS, MBA, cofounder and chief executive officer of CellMax Life, said in an email.
Lance Baldo, MD, Freenome’s chief medical officer, said in an interview that the FDA may be reviewing parts of their application in 2024, allowing for a potential 2025 launch of a blood test for asymptomatic people at average risk for CRC.
A spotty track record
Before anyone gets too excited about the prospect of phasing out screening colonoscopy, it’s important to remember that CRC blood tests have proven disappointing in the past.
Germany’s Epigenomics, for example, secured the first FDA approval for a CRC blood test, Epi ProColon, in 2016. But the company did not receive Medicare coverage for the test. In a 2021 memo explaining the decision, the Centers for Medicare & Medicaid Services noted that, given more reliable alternatives, including stool-based tests, the Epi ProColon would result in harm to some patients.
CMS also does not cover Grail’s blood test, which has a list price of $949, though the company has secured reimbursement arrangements with several self-insured employers and insurers, such as Point32Health.
But CMS officials have acknowledged the strong interest in CRC blood tests.
In that 2021 memo, the agency also outlined its requirements for Medicare coverage. CMS said it will cover blood-based screening tests for certain patients if these products meet the following standards:
- Receive FDA market authorization with an indication for CRC screening.
- Have proven test performance characteristics for a blood-based screening test with sensitivity of at least 74% and specificity of at least 90% in the detection of CRC, compared with the recognized standard (which at this time is colonoscopy) as minimal threshold levels, based on the pivotal studies included in the FDA labeling.
In February 2023, CellLife Max presented data at ASCO Gastrointestinal Cancers Symposium that its blood test had sensitivity of 92.1% for detection of CRC and 54.5% for detection of advanced adenomas, at 91% specificity.
Prior to that, in December 2022, Guardant issued a press release with study results that met the CMS standard. The test had sensitivity of 83% in detecting individuals with CRC. Specificity was 90%, the company said. That translates to a false positive rate of just 10%.
While such results look promising, Asad Umar, DVM, PhD, the chief of gastrointestinal and other cancers at NCI’s division of cancer prevention, said physicians should be cautious when giving advice or answering questions about MCD tests, given limited data from prospective studies about their effect on health outcomes.
Even among physicians already using some MCD tests to screen patients, there is a lot of concern about false-positive results that require diagnostic workup and false negative results that lead to a false sense of assurance, Dr. Umar said.
“Screening is a process and not just a test. The process involves follow-up testing for any positive test findings,” Dr. Umar said. “At this point, doctors should inform patients that there is not sufficient data to know how best to use these tests.”
Hurdles to broad acceptance
For companies seeking broad acceptance of a CRC blood test, two of the three major steps needed are securing FDA approval and Medicare coverage. The last step would be getting an A or B recommendation from the USPSTF, which would mandate coverage by health plans.
This is the “big trifecta,” Dr. Baldo said.
In the USPSTF’s current colon cancer screening recommendations, issued in 2021, it gave an A grade for CRC screening for adults aged 50-75 years and a B grade for those aged 45-49 years.
The USPSTF’s recommended forms of screening include colonoscopy, high-sensitivity guaiac fecal occult blood (gFOBT), fecal immunochemical test (FIT), flexible sigmoidoscopy (FS), stool DNA, and/or computed tomographic colonography (CTC).
The USPSTF says more research is needed to establish the accuracy and effectiveness of emerging screening technologies, such as blood or serum tests.
If CRC blood tests eventually win FDA approval, the USPSTF would likely provide guidance to clinicians on how patients can use them as a screening option.
Dr. Ransohoff noted that the mission of the USPSTF is different from that of the FDA and CMS. The FDA’s approach on medical tests is to consider overall safety and efficacy, as does CMS, but neither agency makes recommendations, nor does it perform its own rigorous quantitative assessment of benefit versus harm. The USPSTF, however, does its own detailed evidence-based reviews of the benefit versus harm of products, Dr. Ransohoff said.
“To me, the Task Force is the gold standard,” Dr. Ransohoff said. “You have to jump through the hoops with the FDA and CMS for making claims, to enable use, and to help get payment. But the Task Force looks at the choices and the consequences in a quantitative way and makes specific practice recommendations.”
What the future may hold
Dr. Carethers sees a future in which highly sensitive blood tests are able to largely replace screening colonoscopies. He said that colonoscopies would be used for people who are most in need of diagnosis and treatment. Dr. Carethers addressed these points during an AGA podcast released in January 2023.
In 20-25 years, colonoscopies may be only a therapeutic procedure, much like endoscopic retrograde cholangiopancreatography is now, Dr. Carethers added.
Even if CRC-specific blood tests prove to be effective screening tools, Dr. Ransohoff stressed that colonoscopy will survive. Many people will eventually need to undergo colonoscopy as a diagnostic procedure following a positive blood-based test result, and some may also opt for screening colonoscopies in lieu of frequent blood tests.
And, overall, physicians and patients will need to weigh the trade-offs of a noninvasive test that can only diagnose CRC versus a screening colonoscopy that offers preventative treatment as well.
“The best intent for screening is prevention of cancer, not detection of cancer,” said Dr. Johnson.
Dr. Carethers, Dr. Johnson, and Dr. Ransohoff reporting having no relevant financial conflicts of interest.
A version of this article originally appeared on Medscape.com.
Most recently, Guardant Health announced the completion of its U.S. premarket approval application for its Shield blood test to screen for CRC. Approval by the Food and Drug Administration would position Guardant to later secure Medicare coverage for its test.
Rival companies, including CellMax Life, Freenome, and Exact Sciences, which already offers the stool-based Cologuard product, are pursuing similar paths in their development of blood tests for CRC.
If these companies succeed, clinicians and patients could have a choice of several FDA-approved tests in a few years.
“They’re coming, and they will be increasingly widely used,” said David A. Johnson, MD, professor of medicine and chief of gastroenterology at Eastern Virginia Medical School, Norfolk, who earlier in his career helped win broader insurance coverage of colonoscopy.
Blood tests for CRC have the potential to cause a shift in screening for colon cancer.
Screening colonoscopies ultimately could be largely phased out in the years ahead in favor of highly sensitive noninvasive tests, if the blood tests do as well as expected, said John M. Carethers, MD, AGAF, president of the American Gastroenterological Association.
‘Holy grail?’
“A blood test for cancer screening has been the ‘holy grail’ ever since the carcinoembryonic antigen blood test in the 1960s was claimed to have nearly 100% sensitivity and specificity – but turned out not to – for colorectal cancer,” wrote David F. Ransohoff, MD, a gastroenterologist at the University of North Carolina at Chapel Hill, in a 2021 article. Dr. Ransohoff has studied noninvasive CRC screening for decades.
There is a great allure in the idea of such multi-cancer detection (MCD) tests. “MCD technology offers the potential to detect asymptomatic cancer at several organ sites with a simple blood test, often called a liquid biopsy, ” according to a National Cancer Institute FY24 budget request report.
Several companies are selling MCD tests, some of which include CRC components. Among the best-known MCD tests now sold is Grail’s Galleri. At this time, however, the Galleri test, which tests for 50 types of cancer, should be used in addition to recommended colon cancer screening tests, such as colonoscopy, the company’s website says.
Guardant has also noted that its CRC-specific blood test should only complement screening tools, including colonoscopy, not replace them.
The prospect of phasing out more commonly used CRC screening – such as colonoscopy – may be appealing, but it would require a big shift for a field in which procedures have dominated. According to a report from the Centers for Disease Control and Prevention, in 2018, 67% of U.S. adults aged 50-75 years met the U.S. Preventive Services Task Force recommendations for CRC screening, and overall, 60.6% had a colonoscopy in the past 10 years.
Still, the NCI and the FDA have signaled the potential they see in MCD tests. The NCI highlighted its plans to aid MCD test development as part of its budget request for fiscal year 2024. The NCI is preparing to launch a 4-year pilot study for MCD tests to enroll 24,000 people aged 45-70 years. The study is intended as groundwork for a randomized controlled trial that will enroll 225,000 people.
The FDA has shown an interest in helping companies bring blood tests for cancer detection to market through its breakthrough device designation – a sign that the FDA places great priority on a product and seeks to streamline the application and review process.
CellMax Life appears to be the only CRC-specific screening blood test to have received a breakthrough device designation from the FDA, Atul Sharan, MS, MBA, cofounder and chief executive officer of CellMax Life, said in an email.
Lance Baldo, MD, Freenome’s chief medical officer, said in an interview that the FDA may be reviewing parts of their application in 2024, allowing for a potential 2025 launch of a blood test for asymptomatic people at average risk for CRC.
A spotty track record
Before anyone gets too excited about the prospect of phasing out screening colonoscopy, it’s important to remember that CRC blood tests have proven disappointing in the past.
Germany’s Epigenomics, for example, secured the first FDA approval for a CRC blood test, Epi ProColon, in 2016. But the company did not receive Medicare coverage for the test. In a 2021 memo explaining the decision, the Centers for Medicare & Medicaid Services noted that, given more reliable alternatives, including stool-based tests, the Epi ProColon would result in harm to some patients.
CMS also does not cover Grail’s blood test, which has a list price of $949, though the company has secured reimbursement arrangements with several self-insured employers and insurers, such as Point32Health.
But CMS officials have acknowledged the strong interest in CRC blood tests.
In that 2021 memo, the agency also outlined its requirements for Medicare coverage. CMS said it will cover blood-based screening tests for certain patients if these products meet the following standards:
- Receive FDA market authorization with an indication for CRC screening.
- Have proven test performance characteristics for a blood-based screening test with sensitivity of at least 74% and specificity of at least 90% in the detection of CRC, compared with the recognized standard (which at this time is colonoscopy) as minimal threshold levels, based on the pivotal studies included in the FDA labeling.
In February 2023, CellLife Max presented data at ASCO Gastrointestinal Cancers Symposium that its blood test had sensitivity of 92.1% for detection of CRC and 54.5% for detection of advanced adenomas, at 91% specificity.
Prior to that, in December 2022, Guardant issued a press release with study results that met the CMS standard. The test had sensitivity of 83% in detecting individuals with CRC. Specificity was 90%, the company said. That translates to a false positive rate of just 10%.
While such results look promising, Asad Umar, DVM, PhD, the chief of gastrointestinal and other cancers at NCI’s division of cancer prevention, said physicians should be cautious when giving advice or answering questions about MCD tests, given limited data from prospective studies about their effect on health outcomes.
Even among physicians already using some MCD tests to screen patients, there is a lot of concern about false-positive results that require diagnostic workup and false negative results that lead to a false sense of assurance, Dr. Umar said.
“Screening is a process and not just a test. The process involves follow-up testing for any positive test findings,” Dr. Umar said. “At this point, doctors should inform patients that there is not sufficient data to know how best to use these tests.”
Hurdles to broad acceptance
For companies seeking broad acceptance of a CRC blood test, two of the three major steps needed are securing FDA approval and Medicare coverage. The last step would be getting an A or B recommendation from the USPSTF, which would mandate coverage by health plans.
This is the “big trifecta,” Dr. Baldo said.
In the USPSTF’s current colon cancer screening recommendations, issued in 2021, it gave an A grade for CRC screening for adults aged 50-75 years and a B grade for those aged 45-49 years.
The USPSTF’s recommended forms of screening include colonoscopy, high-sensitivity guaiac fecal occult blood (gFOBT), fecal immunochemical test (FIT), flexible sigmoidoscopy (FS), stool DNA, and/or computed tomographic colonography (CTC).
The USPSTF says more research is needed to establish the accuracy and effectiveness of emerging screening technologies, such as blood or serum tests.
If CRC blood tests eventually win FDA approval, the USPSTF would likely provide guidance to clinicians on how patients can use them as a screening option.
Dr. Ransohoff noted that the mission of the USPSTF is different from that of the FDA and CMS. The FDA’s approach on medical tests is to consider overall safety and efficacy, as does CMS, but neither agency makes recommendations, nor does it perform its own rigorous quantitative assessment of benefit versus harm. The USPSTF, however, does its own detailed evidence-based reviews of the benefit versus harm of products, Dr. Ransohoff said.
“To me, the Task Force is the gold standard,” Dr. Ransohoff said. “You have to jump through the hoops with the FDA and CMS for making claims, to enable use, and to help get payment. But the Task Force looks at the choices and the consequences in a quantitative way and makes specific practice recommendations.”
What the future may hold
Dr. Carethers sees a future in which highly sensitive blood tests are able to largely replace screening colonoscopies. He said that colonoscopies would be used for people who are most in need of diagnosis and treatment. Dr. Carethers addressed these points during an AGA podcast released in January 2023.
In 20-25 years, colonoscopies may be only a therapeutic procedure, much like endoscopic retrograde cholangiopancreatography is now, Dr. Carethers added.
Even if CRC-specific blood tests prove to be effective screening tools, Dr. Ransohoff stressed that colonoscopy will survive. Many people will eventually need to undergo colonoscopy as a diagnostic procedure following a positive blood-based test result, and some may also opt for screening colonoscopies in lieu of frequent blood tests.
And, overall, physicians and patients will need to weigh the trade-offs of a noninvasive test that can only diagnose CRC versus a screening colonoscopy that offers preventative treatment as well.
“The best intent for screening is prevention of cancer, not detection of cancer,” said Dr. Johnson.
Dr. Carethers, Dr. Johnson, and Dr. Ransohoff reporting having no relevant financial conflicts of interest.
A version of this article originally appeared on Medscape.com.
New challenge for docs: End of COVID federal public health emergency
The Biden administration intends to end by May 11 certain COVID-19 emergency measures used to aid in the response to the pandemic, while many others will remain in place.
A separate declaration covers the Food and Drug Administration’s emergency use authorizations (EUAs) for COVID medicines and tests. That would not be affected by the May 11 deadline, the FDA said. In addition, Congress and state lawmakers have extended some COVID response measures.
The result is a patchwork of emergency COVID-19 measures with different end dates.
The American Medical Association and the American Academy of Family Physicians (AAFP) are assessing how best to advise their members about the end of the public health emergency.
Several waivers regarding copays and coverage and policies regarding controlled substances will expire, Claire Ernst, director of government affairs at the Medical Group Management Association, told this news organization.
The impact of the unwinding “will vary based on some factors, such as what state the practice resides in,” Ms. Ernst said. “Fortunately, Congress provided some predictability for practices by extending many of the telehealth waivers through the end of 2024.”
The AAFP told this news organization that it has joined several other groups in calling for the release of proposed Drug Enforcement Administration (DEA) regulations meant to permanently allow prescriptions of buprenorphine treatment for opioid use disorder via telehealth. The AAFP and other groups want to review these proposals and, if needed, urge the DEA to modify or finalize before there are any disruptions in access to medications for opioid use disorder.
Patients’ questions
Clinicians can expect to field patients’ questions about their insurance coverage and what they need to pay, said Nancy Foster, vice president for quality and patient safety policy at the American Hospital Association (AHA).
“Your doctor’s office, that clinic you typically get care at, that is the face of medicine to you,” Ms. Foster told this news organization. “Many doctors and their staff will be asked, ‘What’s happening with Medicaid?’ ‘What about my Medicare coverage?’ ‘Can I still access care in the same way that I did before?’ ”
Physicians will need to be ready to answers those question, or point patients to where they can get answers, Ms. Foster said.
For example, Medicaid will no longer cover postpartum care for some enrollees after giving birth, said Taylor Platt, health policy manager for the American College of Obstetricians and Gynecologists.
The federal response to the pandemic created “a de facto postpartum coverage extension for Medicaid enrollees,” which will be lost in some states, Ms. Platt told this news organization. However, 28 states and the District of Columbia have taken separate measures to extend postpartum coverage to 1 year.
“This coverage has been critical for postpartum individuals to address health needs like substance use and mental health treatment and chronic conditions,” Ms. Platt said.
States significantly changed Medicaid policy to expand access to care during the pandemic.
All 50 states and the District of Columbia, for example, expanded coverage or access to telehealth services in Medicaid during the pandemic, according to a Jan. 31 report from the Kaiser Family Foundation (KFF). These expansions expire under various deadlines, although most states have made or are planning to make some Medicaid telehealth flexibilities permanent, KFF said.
The KFF report notes that all states and the District of Columbia temporarily waived some aspects of state licensure requirements, so that clinicians with equivalent licenses in other states could practice via telehealth.
In some states, these waivers are still active and are tied to the end of the federal emergency declaration. In others, they expired, with some states allowing for long-term or permanent interstate telemedicine, KFF said. (The Federation of State Medical Boards has a detailed summary of these modifications.)
The end of free COVID vaccines, testing for some patients
The AAFP has also raised concerns about continued access to COVID-19 vaccines, particularly for uninsured adults. Ashish Jha, MD, MPH, the White House COVID-19 Response Coordinator, said in a tweet that this transition, however, wouldn’t happen until a few months after the public health emergency ends.
After those few months, there will be a transition from U.S. government–distributed vaccines and treatments to ones purchased through the regular health care system, the “way we do for every other vaccine and treatment,” Dr. Jha added.
But that raises the same kind of difficult questions that permeate U.S. health care, with a potential to keep COVID active, said Patricia Jackson, RN, president of the Association for Professionals in Infection Control and Epidemiology (APIC).
People who don’t have insurance may lose access to COVID testing and vaccines.
“Will that lead to increases in transmission? Who knows,” Ms. Jackson told this news organization. “We will have to see. There are some health equity issues that potentially arise.”
Future FDA actions
Biden’s May 11 deadline applies to emergency provisions made under a Section 319 declaration, which allow the Department of Health and Human Services to respond to crises.
But a separate flexibility, known as a Section 564 declaration, covers the FDA’s EUAs, which can remain in effect even as the other declarations end.
The best-known EUAs for the pandemic were used to bring COVID vaccines and treatments to market. Many of these have since been converted to normal approvals as companies presented more evidence to support the initial emergency approvals. In other cases, EUAs have been withdrawn owing to disappointing research results, changing virus strains, and evolving medical treatments.
The FDA also used many EUAs to cover new uses of ventilators and other hospital equipment and expand these supplies in response to the pandemic, said Mark Howell, AHA’s director of policy and patient safety.
The FDA should examine the EUAs issued during the pandemic to see what greater flexibilities might be used to deal with future serious shortages of critical supplies. International incidents such as the war in Ukraine show how fragile the supply chain can be. The FDA should consider its recent experience with EUAs to address this, Mr. Howell said.
“What do we do coming out of the pandemic? And how do we think about being more proactive in this space to ensure that our supply doesn’t bottleneck, that we continue to make sure that providers have access to supply that’s not only safe and effective, but that they can use?” Mr. Howell told this news organization.
Such planning might also help prepare the country for the next pandemic, which is a near certainty, APIC’s Ms. Jackson said. The nation needs a nimbler response to the next major outbreak of an infectious disease, she said.
“There is going to be a next time,” Ms. Jackson said. “We are going to have another pandemic.”
A version of this article first appeared on Medscape.com.
The Biden administration intends to end by May 11 certain COVID-19 emergency measures used to aid in the response to the pandemic, while many others will remain in place.
A separate declaration covers the Food and Drug Administration’s emergency use authorizations (EUAs) for COVID medicines and tests. That would not be affected by the May 11 deadline, the FDA said. In addition, Congress and state lawmakers have extended some COVID response measures.
The result is a patchwork of emergency COVID-19 measures with different end dates.
The American Medical Association and the American Academy of Family Physicians (AAFP) are assessing how best to advise their members about the end of the public health emergency.
Several waivers regarding copays and coverage and policies regarding controlled substances will expire, Claire Ernst, director of government affairs at the Medical Group Management Association, told this news organization.
The impact of the unwinding “will vary based on some factors, such as what state the practice resides in,” Ms. Ernst said. “Fortunately, Congress provided some predictability for practices by extending many of the telehealth waivers through the end of 2024.”
The AAFP told this news organization that it has joined several other groups in calling for the release of proposed Drug Enforcement Administration (DEA) regulations meant to permanently allow prescriptions of buprenorphine treatment for opioid use disorder via telehealth. The AAFP and other groups want to review these proposals and, if needed, urge the DEA to modify or finalize before there are any disruptions in access to medications for opioid use disorder.
Patients’ questions
Clinicians can expect to field patients’ questions about their insurance coverage and what they need to pay, said Nancy Foster, vice president for quality and patient safety policy at the American Hospital Association (AHA).
“Your doctor’s office, that clinic you typically get care at, that is the face of medicine to you,” Ms. Foster told this news organization. “Many doctors and their staff will be asked, ‘What’s happening with Medicaid?’ ‘What about my Medicare coverage?’ ‘Can I still access care in the same way that I did before?’ ”
Physicians will need to be ready to answers those question, or point patients to where they can get answers, Ms. Foster said.
For example, Medicaid will no longer cover postpartum care for some enrollees after giving birth, said Taylor Platt, health policy manager for the American College of Obstetricians and Gynecologists.
The federal response to the pandemic created “a de facto postpartum coverage extension for Medicaid enrollees,” which will be lost in some states, Ms. Platt told this news organization. However, 28 states and the District of Columbia have taken separate measures to extend postpartum coverage to 1 year.
“This coverage has been critical for postpartum individuals to address health needs like substance use and mental health treatment and chronic conditions,” Ms. Platt said.
States significantly changed Medicaid policy to expand access to care during the pandemic.
All 50 states and the District of Columbia, for example, expanded coverage or access to telehealth services in Medicaid during the pandemic, according to a Jan. 31 report from the Kaiser Family Foundation (KFF). These expansions expire under various deadlines, although most states have made or are planning to make some Medicaid telehealth flexibilities permanent, KFF said.
The KFF report notes that all states and the District of Columbia temporarily waived some aspects of state licensure requirements, so that clinicians with equivalent licenses in other states could practice via telehealth.
In some states, these waivers are still active and are tied to the end of the federal emergency declaration. In others, they expired, with some states allowing for long-term or permanent interstate telemedicine, KFF said. (The Federation of State Medical Boards has a detailed summary of these modifications.)
The end of free COVID vaccines, testing for some patients
The AAFP has also raised concerns about continued access to COVID-19 vaccines, particularly for uninsured adults. Ashish Jha, MD, MPH, the White House COVID-19 Response Coordinator, said in a tweet that this transition, however, wouldn’t happen until a few months after the public health emergency ends.
After those few months, there will be a transition from U.S. government–distributed vaccines and treatments to ones purchased through the regular health care system, the “way we do for every other vaccine and treatment,” Dr. Jha added.
But that raises the same kind of difficult questions that permeate U.S. health care, with a potential to keep COVID active, said Patricia Jackson, RN, president of the Association for Professionals in Infection Control and Epidemiology (APIC).
People who don’t have insurance may lose access to COVID testing and vaccines.
“Will that lead to increases in transmission? Who knows,” Ms. Jackson told this news organization. “We will have to see. There are some health equity issues that potentially arise.”
Future FDA actions
Biden’s May 11 deadline applies to emergency provisions made under a Section 319 declaration, which allow the Department of Health and Human Services to respond to crises.
But a separate flexibility, known as a Section 564 declaration, covers the FDA’s EUAs, which can remain in effect even as the other declarations end.
The best-known EUAs for the pandemic were used to bring COVID vaccines and treatments to market. Many of these have since been converted to normal approvals as companies presented more evidence to support the initial emergency approvals. In other cases, EUAs have been withdrawn owing to disappointing research results, changing virus strains, and evolving medical treatments.
The FDA also used many EUAs to cover new uses of ventilators and other hospital equipment and expand these supplies in response to the pandemic, said Mark Howell, AHA’s director of policy and patient safety.
The FDA should examine the EUAs issued during the pandemic to see what greater flexibilities might be used to deal with future serious shortages of critical supplies. International incidents such as the war in Ukraine show how fragile the supply chain can be. The FDA should consider its recent experience with EUAs to address this, Mr. Howell said.
“What do we do coming out of the pandemic? And how do we think about being more proactive in this space to ensure that our supply doesn’t bottleneck, that we continue to make sure that providers have access to supply that’s not only safe and effective, but that they can use?” Mr. Howell told this news organization.
Such planning might also help prepare the country for the next pandemic, which is a near certainty, APIC’s Ms. Jackson said. The nation needs a nimbler response to the next major outbreak of an infectious disease, she said.
“There is going to be a next time,” Ms. Jackson said. “We are going to have another pandemic.”
A version of this article first appeared on Medscape.com.
The Biden administration intends to end by May 11 certain COVID-19 emergency measures used to aid in the response to the pandemic, while many others will remain in place.
A separate declaration covers the Food and Drug Administration’s emergency use authorizations (EUAs) for COVID medicines and tests. That would not be affected by the May 11 deadline, the FDA said. In addition, Congress and state lawmakers have extended some COVID response measures.
The result is a patchwork of emergency COVID-19 measures with different end dates.
The American Medical Association and the American Academy of Family Physicians (AAFP) are assessing how best to advise their members about the end of the public health emergency.
Several waivers regarding copays and coverage and policies regarding controlled substances will expire, Claire Ernst, director of government affairs at the Medical Group Management Association, told this news organization.
The impact of the unwinding “will vary based on some factors, such as what state the practice resides in,” Ms. Ernst said. “Fortunately, Congress provided some predictability for practices by extending many of the telehealth waivers through the end of 2024.”
The AAFP told this news organization that it has joined several other groups in calling for the release of proposed Drug Enforcement Administration (DEA) regulations meant to permanently allow prescriptions of buprenorphine treatment for opioid use disorder via telehealth. The AAFP and other groups want to review these proposals and, if needed, urge the DEA to modify or finalize before there are any disruptions in access to medications for opioid use disorder.
Patients’ questions
Clinicians can expect to field patients’ questions about their insurance coverage and what they need to pay, said Nancy Foster, vice president for quality and patient safety policy at the American Hospital Association (AHA).
“Your doctor’s office, that clinic you typically get care at, that is the face of medicine to you,” Ms. Foster told this news organization. “Many doctors and their staff will be asked, ‘What’s happening with Medicaid?’ ‘What about my Medicare coverage?’ ‘Can I still access care in the same way that I did before?’ ”
Physicians will need to be ready to answers those question, or point patients to where they can get answers, Ms. Foster said.
For example, Medicaid will no longer cover postpartum care for some enrollees after giving birth, said Taylor Platt, health policy manager for the American College of Obstetricians and Gynecologists.
The federal response to the pandemic created “a de facto postpartum coverage extension for Medicaid enrollees,” which will be lost in some states, Ms. Platt told this news organization. However, 28 states and the District of Columbia have taken separate measures to extend postpartum coverage to 1 year.
“This coverage has been critical for postpartum individuals to address health needs like substance use and mental health treatment and chronic conditions,” Ms. Platt said.
States significantly changed Medicaid policy to expand access to care during the pandemic.
All 50 states and the District of Columbia, for example, expanded coverage or access to telehealth services in Medicaid during the pandemic, according to a Jan. 31 report from the Kaiser Family Foundation (KFF). These expansions expire under various deadlines, although most states have made or are planning to make some Medicaid telehealth flexibilities permanent, KFF said.
The KFF report notes that all states and the District of Columbia temporarily waived some aspects of state licensure requirements, so that clinicians with equivalent licenses in other states could practice via telehealth.
In some states, these waivers are still active and are tied to the end of the federal emergency declaration. In others, they expired, with some states allowing for long-term or permanent interstate telemedicine, KFF said. (The Federation of State Medical Boards has a detailed summary of these modifications.)
The end of free COVID vaccines, testing for some patients
The AAFP has also raised concerns about continued access to COVID-19 vaccines, particularly for uninsured adults. Ashish Jha, MD, MPH, the White House COVID-19 Response Coordinator, said in a tweet that this transition, however, wouldn’t happen until a few months after the public health emergency ends.
After those few months, there will be a transition from U.S. government–distributed vaccines and treatments to ones purchased through the regular health care system, the “way we do for every other vaccine and treatment,” Dr. Jha added.
But that raises the same kind of difficult questions that permeate U.S. health care, with a potential to keep COVID active, said Patricia Jackson, RN, president of the Association for Professionals in Infection Control and Epidemiology (APIC).
People who don’t have insurance may lose access to COVID testing and vaccines.
“Will that lead to increases in transmission? Who knows,” Ms. Jackson told this news organization. “We will have to see. There are some health equity issues that potentially arise.”
Future FDA actions
Biden’s May 11 deadline applies to emergency provisions made under a Section 319 declaration, which allow the Department of Health and Human Services to respond to crises.
But a separate flexibility, known as a Section 564 declaration, covers the FDA’s EUAs, which can remain in effect even as the other declarations end.
The best-known EUAs for the pandemic were used to bring COVID vaccines and treatments to market. Many of these have since been converted to normal approvals as companies presented more evidence to support the initial emergency approvals. In other cases, EUAs have been withdrawn owing to disappointing research results, changing virus strains, and evolving medical treatments.
The FDA also used many EUAs to cover new uses of ventilators and other hospital equipment and expand these supplies in response to the pandemic, said Mark Howell, AHA’s director of policy and patient safety.
The FDA should examine the EUAs issued during the pandemic to see what greater flexibilities might be used to deal with future serious shortages of critical supplies. International incidents such as the war in Ukraine show how fragile the supply chain can be. The FDA should consider its recent experience with EUAs to address this, Mr. Howell said.
“What do we do coming out of the pandemic? And how do we think about being more proactive in this space to ensure that our supply doesn’t bottleneck, that we continue to make sure that providers have access to supply that’s not only safe and effective, but that they can use?” Mr. Howell told this news organization.
Such planning might also help prepare the country for the next pandemic, which is a near certainty, APIC’s Ms. Jackson said. The nation needs a nimbler response to the next major outbreak of an infectious disease, she said.
“There is going to be a next time,” Ms. Jackson said. “We are going to have another pandemic.”
A version of this article first appeared on Medscape.com.