Kerry Dooley Young

The Trump administration is sticking with a plan to boost certain Medicare pay for many primary care and other specialties focused heavily on office visits while lowering that for other groups to balance these increased costs.

On Aug. 4, the Centers for Medicare & Medicaid Services posted on the Federal Register draft versions of two of its major annual payment measures: the physician fee schedule and the payment rule for hospital outpatient services. On Aug. 3, the CMS informally posted a copy of the physician fee schedule on its own website, allowing medical groups to begin reading the more than 1,300-page rule.

Federal officials normally use annual Medicare payment rules to make many revisions to policies as well as adjust reimbursement.

The draft 2021 physician fee schedule, for example, calls for broadening the authority of clinicians other than physicians to authorize testing of people enrolled in Medicare.

The CMS intends to allow nurse practitioners, physician assistants, and certain other health care professionals to more widely supervise diagnostic psychological and neuropsychological tests, in keeping with applicable state laws.

The draft 2021 hospital outpatient rule proposes a gradual changeover to allow more procedures to be performed on an outpatient basis. This shift could save money for Medicare as well as for the people enrolled in the giant federal health program who need these services, the CMS explained.

Medicare would begin with a change in status for almost 300 musculoskeletal-related services, making them eligible for payment in the hospital outpatient setting when appropriate, CMS wrote in a fact sheet.

The initial reaction to Medicare’s proposed 2021 rules centered on its planned redistribution of funds among medical specialties. The CMS had outlined this plan last year. It is part of longstanding efforts to boost pay for primary care specialists and other physicians whose practice centers more around office visits than procedures.

There is broad support in health policy circles for raising pay for these specialties, but there also are strong objections to the cuts the CMS plans to offset the cost of rising pay for some fields.

Susan R. Bailey, MD, president of the American Medical Association, addressed both of these ideas in an AMA news release on the proposed 2021 physician fee schedule. The increase in pay for office visits, covered under evaluation and management services (E/M), stems from recommendations on resource costs from the AMA/Specialty Society RVS Update Committee, Dr. Bailey said.

“Unfortunately, these office visit payment increases, and a multitude of other new CMS proposed payment increases, are required by statute to be offset by payment reductions to other services, through an unsustainable reduction of nearly 11% to the Medicare conversion factor,” Dr. Bailey explained.

In the news release, Dr. Bailey asked Congress to waive Medicare’s budget-neutrality requirements to allow increases without the cuts.

“Physicians are already experiencing substantial economic hardships due to COVID-19, so these pay cuts could not come at a worse time,” she said.

Winners and losers

The CMS details the possible winners and losers in its payment reshuffle in Table 90 of the proposed 2021 physician fee schedule. In the proposed rule, CMS notes in the draft that these figures are based upon estimates of aggregate allowed charges across all services furnished by physicians and other clinicians.

“Therefore, they are averages, and may not necessarily be representative of what is happening to the particular services furnished by a single practitioner within any given specialty,” the CMS said.

Specialties in line for increases under the 2021 draft rule include allergy/immunology (9%), endocrinology (17%), family practice (13%), general practice (8%), geriatrics (4%), hematology/oncology (14%), internal medicine (4%), nephrology (6%), physician assistants (8%), psychiatry (8%), rheumatology (16%), and urology (8%).

In line for cuts would be anesthesiology (–8%), cardiac surgery (–9%), emergency medicine (–6%), general surgery (–7%), infectious disease (–4%), neurosurgery (–7%), physical/occupational therapy (–9%), plastic surgery (–7%), radiology (–11%), and thoracic surgery (–8%).

An umbrella group, the Surgical Care Coalition, on Aug. 3 had a quick statement ready about the CMS proposal. Writing on behalf of the group was David B. Hoyt, MD, executive director of the American College of Surgeons.

“Today’s proposed rule ignores both patients and the surgeons who care for them. The middle of a pandemic is no time for cuts to any form of health care, but today’s announcement moves ahead as if nothing has changed,” Hoyt said in the statement. “The Surgical Care Coalition believes no physician should see payment cuts that will reduce patients’ access to care.”

The Surgical Care Coalition already has been asking Congress to waive budget-neutrality requirements. Making a similar request Aug. 4 in a unified statement were the American Physical Therapy Association (APTA), the American Occupational Therapy Association (AOTA), and the American Speech-Language-Hearing Association (ASHA).

“Our organizations call on Congress and CMS to advance well-reasoned fee schedule payment policies and waive budget neutrality,” the groups said. “While APTA, AOTA, and ASHA do not oppose payment increases for primary care physicians, we believe these increases can be implemented without imposing payment reductions on other providers.”
 

A version of this article originally appeared on Medscape.com.

The Trump administration is sticking with a plan to boost certain Medicare pay for many primary care and other specialties focused heavily on office visits while lowering that for other groups to balance these increased costs.

On Aug. 4, the Centers for Medicare & Medicaid Services posted on the Federal Register draft versions of two of its major annual payment measures: the physician fee schedule and the payment rule for hospital outpatient services. On Aug. 3, the CMS informally posted a copy of the physician fee schedule on its own website, allowing medical groups to begin reading the more than 1,300-page rule.

Federal officials normally use annual Medicare payment rules to make many revisions to policies as well as adjust reimbursement.

The draft 2021 physician fee schedule, for example, calls for broadening the authority of clinicians other than physicians to authorize testing of people enrolled in Medicare.

The CMS intends to allow nurse practitioners, physician assistants, and certain other health care professionals to more widely supervise diagnostic psychological and neuropsychological tests, in keeping with applicable state laws.

The draft 2021 hospital outpatient rule proposes a gradual changeover to allow more procedures to be performed on an outpatient basis. This shift could save money for Medicare as well as for the people enrolled in the giant federal health program who need these services, the CMS explained.

Medicare would begin with a change in status for almost 300 musculoskeletal-related services, making them eligible for payment in the hospital outpatient setting when appropriate, CMS wrote in a fact sheet.

The initial reaction to Medicare’s proposed 2021 rules centered on its planned redistribution of funds among medical specialties. The CMS had outlined this plan last year. It is part of longstanding efforts to boost pay for primary care specialists and other physicians whose practice centers more around office visits than procedures.

There is broad support in health policy circles for raising pay for these specialties, but there also are strong objections to the cuts the CMS plans to offset the cost of rising pay for some fields.

Susan R. Bailey, MD, president of the American Medical Association, addressed both of these ideas in an AMA news release on the proposed 2021 physician fee schedule. The increase in pay for office visits, covered under evaluation and management services (E/M), stems from recommendations on resource costs from the AMA/Specialty Society RVS Update Committee, Dr. Bailey said.

“Unfortunately, these office visit payment increases, and a multitude of other new CMS proposed payment increases, are required by statute to be offset by payment reductions to other services, through an unsustainable reduction of nearly 11% to the Medicare conversion factor,” Dr. Bailey explained.

In the news release, Dr. Bailey asked Congress to waive Medicare’s budget-neutrality requirements to allow increases without the cuts.

“Physicians are already experiencing substantial economic hardships due to COVID-19, so these pay cuts could not come at a worse time,” she said.

Winners and losers

The CMS details the possible winners and losers in its payment reshuffle in Table 90 of the proposed 2021 physician fee schedule. In the proposed rule, CMS notes in the draft that these figures are based upon estimates of aggregate allowed charges across all services furnished by physicians and other clinicians.

“Therefore, they are averages, and may not necessarily be representative of what is happening to the particular services furnished by a single practitioner within any given specialty,” the CMS said.

Specialties in line for increases under the 2021 draft rule include allergy/immunology (9%), endocrinology (17%), family practice (13%), general practice (8%), geriatrics (4%), hematology/oncology (14%), internal medicine (4%), nephrology (6%), physician assistants (8%), psychiatry (8%), rheumatology (16%), and urology (8%).

In line for cuts would be anesthesiology (–8%), cardiac surgery (–9%), emergency medicine (–6%), general surgery (–7%), infectious disease (–4%), neurosurgery (–7%), physical/occupational therapy (–9%), plastic surgery (–7%), radiology (–11%), and thoracic surgery (–8%).

An umbrella group, the Surgical Care Coalition, on Aug. 3 had a quick statement ready about the CMS proposal. Writing on behalf of the group was David B. Hoyt, MD, executive director of the American College of Surgeons.

“Today’s proposed rule ignores both patients and the surgeons who care for them. The middle of a pandemic is no time for cuts to any form of health care, but today’s announcement moves ahead as if nothing has changed,” Hoyt said in the statement. “The Surgical Care Coalition believes no physician should see payment cuts that will reduce patients’ access to care.”

The Surgical Care Coalition already has been asking Congress to waive budget-neutrality requirements. Making a similar request Aug. 4 in a unified statement were the American Physical Therapy Association (APTA), the American Occupational Therapy Association (AOTA), and the American Speech-Language-Hearing Association (ASHA).

“Our organizations call on Congress and CMS to advance well-reasoned fee schedule payment policies and waive budget neutrality,” the groups said. “While APTA, AOTA, and ASHA do not oppose payment increases for primary care physicians, we believe these increases can be implemented without imposing payment reductions on other providers.”
 

A version of this article originally appeared on Medscape.com.

The Trump administration is sticking with a plan to boost certain Medicare pay for many primary care and other specialties focused heavily on office visits while lowering that for other groups to balance these increased costs.

On Aug. 4, the Centers for Medicare & Medicaid Services posted on the Federal Register draft versions of two of its major annual payment measures: the physician fee schedule and the payment rule for hospital outpatient services. On Aug. 3, the CMS informally posted a copy of the physician fee schedule on its own website, allowing medical groups to begin reading the more than 1,300-page rule.

Federal officials normally use annual Medicare payment rules to make many revisions to policies as well as adjust reimbursement.

The draft 2021 physician fee schedule, for example, calls for broadening the authority of clinicians other than physicians to authorize testing of people enrolled in Medicare.

The CMS intends to allow nurse practitioners, physician assistants, and certain other health care professionals to more widely supervise diagnostic psychological and neuropsychological tests, in keeping with applicable state laws.

The draft 2021 hospital outpatient rule proposes a gradual changeover to allow more procedures to be performed on an outpatient basis. This shift could save money for Medicare as well as for the people enrolled in the giant federal health program who need these services, the CMS explained.

Medicare would begin with a change in status for almost 300 musculoskeletal-related services, making them eligible for payment in the hospital outpatient setting when appropriate, CMS wrote in a fact sheet.

The initial reaction to Medicare’s proposed 2021 rules centered on its planned redistribution of funds among medical specialties. The CMS had outlined this plan last year. It is part of longstanding efforts to boost pay for primary care specialists and other physicians whose practice centers more around office visits than procedures.

There is broad support in health policy circles for raising pay for these specialties, but there also are strong objections to the cuts the CMS plans to offset the cost of rising pay for some fields.

Susan R. Bailey, MD, president of the American Medical Association, addressed both of these ideas in an AMA news release on the proposed 2021 physician fee schedule. The increase in pay for office visits, covered under evaluation and management services (E/M), stems from recommendations on resource costs from the AMA/Specialty Society RVS Update Committee, Dr. Bailey said.

“Unfortunately, these office visit payment increases, and a multitude of other new CMS proposed payment increases, are required by statute to be offset by payment reductions to other services, through an unsustainable reduction of nearly 11% to the Medicare conversion factor,” Dr. Bailey explained.

In the news release, Dr. Bailey asked Congress to waive Medicare’s budget-neutrality requirements to allow increases without the cuts.

“Physicians are already experiencing substantial economic hardships due to COVID-19, so these pay cuts could not come at a worse time,” she said.

Winners and losers

The CMS details the possible winners and losers in its payment reshuffle in Table 90 of the proposed 2021 physician fee schedule. In the proposed rule, CMS notes in the draft that these figures are based upon estimates of aggregate allowed charges across all services furnished by physicians and other clinicians.

“Therefore, they are averages, and may not necessarily be representative of what is happening to the particular services furnished by a single practitioner within any given specialty,” the CMS said.

Specialties in line for increases under the 2021 draft rule include allergy/immunology (9%), endocrinology (17%), family practice (13%), general practice (8%), geriatrics (4%), hematology/oncology (14%), internal medicine (4%), nephrology (6%), physician assistants (8%), psychiatry (8%), rheumatology (16%), and urology (8%).

In line for cuts would be anesthesiology (–8%), cardiac surgery (–9%), emergency medicine (–6%), general surgery (–7%), infectious disease (–4%), neurosurgery (–7%), physical/occupational therapy (–9%), plastic surgery (–7%), radiology (–11%), and thoracic surgery (–8%).

An umbrella group, the Surgical Care Coalition, on Aug. 3 had a quick statement ready about the CMS proposal. Writing on behalf of the group was David B. Hoyt, MD, executive director of the American College of Surgeons.

“Today’s proposed rule ignores both patients and the surgeons who care for them. The middle of a pandemic is no time for cuts to any form of health care, but today’s announcement moves ahead as if nothing has changed,” Hoyt said in the statement. “The Surgical Care Coalition believes no physician should see payment cuts that will reduce patients’ access to care.”

The Surgical Care Coalition already has been asking Congress to waive budget-neutrality requirements. Making a similar request Aug. 4 in a unified statement were the American Physical Therapy Association (APTA), the American Occupational Therapy Association (AOTA), and the American Speech-Language-Hearing Association (ASHA).

“Our organizations call on Congress and CMS to advance well-reasoned fee schedule payment policies and waive budget neutrality,” the groups said. “While APTA, AOTA, and ASHA do not oppose payment increases for primary care physicians, we believe these increases can be implemented without imposing payment reductions on other providers.”
 

A version of this article originally appeared on Medscape.com.

Several federal lawmakers on June 30 questioned state policies that require disclosure of mental health treatment as part of medical licensing applications and renewals, citing concerns about creating barriers to psychiatric care for clinicians.

Mental health–related questions on state medical boards’ licensing applications are especially worrisome with many clinicians, including ED staff, immersed in the physical and emotional challenges involved in treating waves of people with COVID-19, lawmakers said during a hearing of the House Energy and Commerce Committee’s health panel.

“We must consider the mental health of the providers on the front lines of the pandemic,” said Rep. Morgan Griffith, a Virginia Republican.

The issue of state medical boards’ disclosure rules was not on the official agenda for the House Energy and Commerce health subcommittee’s hearing. And there was no discussion of any specific state medical board’s regulations. The Energy and Commerce health subcommittee is working on more than 20 bills related to mental health, including measures intended to aid first responders, such as firemen and emergency medical personnel, and students.

This hearing marked an early stage in the process for a planned package of mental health legislation, said Rep. Michael C. Burgess, MD, of Texas, who is the top Republican on the Energy and Commerce health subcommittee. There may be opportunities as this legislation advances to add provisions intended to aid physicians, said Dr. Burgess, who practiced for many years as an ob.gyn. before being elected to Congress.

“We knew that suicide was a problem among our colleagues prior to the onset of this coronavirus epidemic and I know it is more pronounced now,” he said.

Dr. Burgess then solicited specific recommendations from the hearing’s witnesses on steps needed to help clinicians’ mental health.

The first suggestion offered in reply by Jeffrey L. Geller, MD, MPH, appearing in his role as president of the American Psychiatric Association, was that Congress should look for ways to encourage states to alter their licensing procedures.

The hearing comes on the heels of the APA, the American Academy of Family Physicians, and more than 40 other groups having jointly signed a statement calling for changes to disclosure rules about mental health.

“Licensing and credentialing applications by covered entities should only employ narrowly focused questions that address current functional impairment,” the statement said. “Additionally, we strongly support The Joint Commission (TJC) statement on Removing Barriers to Mental Health Care for Clinicians and Health Care Staff. TJC ‘supports the removal of any barriers that inhibit clinicians and health care staff from accessing mental health care services.’ ”

Physicians and other clinicians must be able to safely secure treatment for mental or other health issues, just as any other individual,” the groups wrote. “A provider’s history of mental illness or substance use disorder should not be used as any indication of their current or future ability to practice competently and without impairment.”

Also among the signers to this statement was the Federation of State Medical Boards, which has been leading an effort for years to change licensing.

In 2018, the FSMB recommended state medical boards reconsider whether it is necessary to include probing questions about a physician applicant’s mental health, addiction, or substance use on applications for medical licensure or their renewal. While the intent of these questions may be to protect patients, these queries can discourage physicians from getting needed help, the FSMB said.

Several states have since revised or considered revising their license applications and renewals. In May 2020, The Joint Commission urged broader adoption of recommendations from the FSMB and the American Medical Association to limit queries about clinicians’ mental health to “conditions that currently impair the clinicians’ ability to perform their job.”

“We strongly encourage organizations to not ask about past history of mental health conditions or treatment,” said The Joint Commission, which accredits hospitals, in a statement. “It is critical that we ensure health care workers can feel free to access mental health resources.”

Rep. Susan Brooks, an Indiana Republican who is an attorney, suggested there may need to be a broader look at how state officials pose questions about past mental health treatment to people in many professions, including her own.

“It does build on the stigma on accessing services” to know a state or licensing authority may question a professional about receiving treatment for mental health, she said.

Also at the hearing, Rep. Nanette Diaz Barragán, a California Democrat, spoke of her own reaction to seeing a question about mental health treatment while applying for a White House internship. During her college years, Rep. Barragán had to cope with her father’s terminal illness.

“I remember thinking to myself: ‘Jeez, if I end up seeing a mental health expert maybe one day I couldn’t work in government,’ ” she said.

Several federal lawmakers on June 30 questioned state policies that require disclosure of mental health treatment as part of medical licensing applications and renewals, citing concerns about creating barriers to psychiatric care for clinicians.

Mental health–related questions on state medical boards’ licensing applications are especially worrisome with many clinicians, including ED staff, immersed in the physical and emotional challenges involved in treating waves of people with COVID-19, lawmakers said during a hearing of the House Energy and Commerce Committee’s health panel.

“We must consider the mental health of the providers on the front lines of the pandemic,” said Rep. Morgan Griffith, a Virginia Republican.

The issue of state medical boards’ disclosure rules was not on the official agenda for the House Energy and Commerce health subcommittee’s hearing. And there was no discussion of any specific state medical board’s regulations. The Energy and Commerce health subcommittee is working on more than 20 bills related to mental health, including measures intended to aid first responders, such as firemen and emergency medical personnel, and students.

This hearing marked an early stage in the process for a planned package of mental health legislation, said Rep. Michael C. Burgess, MD, of Texas, who is the top Republican on the Energy and Commerce health subcommittee. There may be opportunities as this legislation advances to add provisions intended to aid physicians, said Dr. Burgess, who practiced for many years as an ob.gyn. before being elected to Congress.

“We knew that suicide was a problem among our colleagues prior to the onset of this coronavirus epidemic and I know it is more pronounced now,” he said.

Dr. Burgess then solicited specific recommendations from the hearing’s witnesses on steps needed to help clinicians’ mental health.

The first suggestion offered in reply by Jeffrey L. Geller, MD, MPH, appearing in his role as president of the American Psychiatric Association, was that Congress should look for ways to encourage states to alter their licensing procedures.

The hearing comes on the heels of the APA, the American Academy of Family Physicians, and more than 40 other groups having jointly signed a statement calling for changes to disclosure rules about mental health.

“Licensing and credentialing applications by covered entities should only employ narrowly focused questions that address current functional impairment,” the statement said. “Additionally, we strongly support The Joint Commission (TJC) statement on Removing Barriers to Mental Health Care for Clinicians and Health Care Staff. TJC ‘supports the removal of any barriers that inhibit clinicians and health care staff from accessing mental health care services.’ ”

Physicians and other clinicians must be able to safely secure treatment for mental or other health issues, just as any other individual,” the groups wrote. “A provider’s history of mental illness or substance use disorder should not be used as any indication of their current or future ability to practice competently and without impairment.”

Also among the signers to this statement was the Federation of State Medical Boards, which has been leading an effort for years to change licensing.

In 2018, the FSMB recommended state medical boards reconsider whether it is necessary to include probing questions about a physician applicant’s mental health, addiction, or substance use on applications for medical licensure or their renewal. While the intent of these questions may be to protect patients, these queries can discourage physicians from getting needed help, the FSMB said.

Several states have since revised or considered revising their license applications and renewals. In May 2020, The Joint Commission urged broader adoption of recommendations from the FSMB and the American Medical Association to limit queries about clinicians’ mental health to “conditions that currently impair the clinicians’ ability to perform their job.”

“We strongly encourage organizations to not ask about past history of mental health conditions or treatment,” said The Joint Commission, which accredits hospitals, in a statement. “It is critical that we ensure health care workers can feel free to access mental health resources.”

Rep. Susan Brooks, an Indiana Republican who is an attorney, suggested there may need to be a broader look at how state officials pose questions about past mental health treatment to people in many professions, including her own.

“It does build on the stigma on accessing services” to know a state or licensing authority may question a professional about receiving treatment for mental health, she said.

Also at the hearing, Rep. Nanette Diaz Barragán, a California Democrat, spoke of her own reaction to seeing a question about mental health treatment while applying for a White House internship. During her college years, Rep. Barragán had to cope with her father’s terminal illness.

“I remember thinking to myself: ‘Jeez, if I end up seeing a mental health expert maybe one day I couldn’t work in government,’ ” she said.

Several federal lawmakers on June 30 questioned state policies that require disclosure of mental health treatment as part of medical licensing applications and renewals, citing concerns about creating barriers to psychiatric care for clinicians.

Mental health–related questions on state medical boards’ licensing applications are especially worrisome with many clinicians, including ED staff, immersed in the physical and emotional challenges involved in treating waves of people with COVID-19, lawmakers said during a hearing of the House Energy and Commerce Committee’s health panel.

“We must consider the mental health of the providers on the front lines of the pandemic,” said Rep. Morgan Griffith, a Virginia Republican.

The issue of state medical boards’ disclosure rules was not on the official agenda for the House Energy and Commerce health subcommittee’s hearing. And there was no discussion of any specific state medical board’s regulations. The Energy and Commerce health subcommittee is working on more than 20 bills related to mental health, including measures intended to aid first responders, such as firemen and emergency medical personnel, and students.

This hearing marked an early stage in the process for a planned package of mental health legislation, said Rep. Michael C. Burgess, MD, of Texas, who is the top Republican on the Energy and Commerce health subcommittee. There may be opportunities as this legislation advances to add provisions intended to aid physicians, said Dr. Burgess, who practiced for many years as an ob.gyn. before being elected to Congress.

“We knew that suicide was a problem among our colleagues prior to the onset of this coronavirus epidemic and I know it is more pronounced now,” he said.

Dr. Burgess then solicited specific recommendations from the hearing’s witnesses on steps needed to help clinicians’ mental health.

The first suggestion offered in reply by Jeffrey L. Geller, MD, MPH, appearing in his role as president of the American Psychiatric Association, was that Congress should look for ways to encourage states to alter their licensing procedures.

The hearing comes on the heels of the APA, the American Academy of Family Physicians, and more than 40 other groups having jointly signed a statement calling for changes to disclosure rules about mental health.

“Licensing and credentialing applications by covered entities should only employ narrowly focused questions that address current functional impairment,” the statement said. “Additionally, we strongly support The Joint Commission (TJC) statement on Removing Barriers to Mental Health Care for Clinicians and Health Care Staff. TJC ‘supports the removal of any barriers that inhibit clinicians and health care staff from accessing mental health care services.’ ”

Physicians and other clinicians must be able to safely secure treatment for mental or other health issues, just as any other individual,” the groups wrote. “A provider’s history of mental illness or substance use disorder should not be used as any indication of their current or future ability to practice competently and without impairment.”

Also among the signers to this statement was the Federation of State Medical Boards, which has been leading an effort for years to change licensing.

In 2018, the FSMB recommended state medical boards reconsider whether it is necessary to include probing questions about a physician applicant’s mental health, addiction, or substance use on applications for medical licensure or their renewal. While the intent of these questions may be to protect patients, these queries can discourage physicians from getting needed help, the FSMB said.

Several states have since revised or considered revising their license applications and renewals. In May 2020, The Joint Commission urged broader adoption of recommendations from the FSMB and the American Medical Association to limit queries about clinicians’ mental health to “conditions that currently impair the clinicians’ ability to perform their job.”

“We strongly encourage organizations to not ask about past history of mental health conditions or treatment,” said The Joint Commission, which accredits hospitals, in a statement. “It is critical that we ensure health care workers can feel free to access mental health resources.”

Rep. Susan Brooks, an Indiana Republican who is an attorney, suggested there may need to be a broader look at how state officials pose questions about past mental health treatment to people in many professions, including her own.

“It does build on the stigma on accessing services” to know a state or licensing authority may question a professional about receiving treatment for mental health, she said.

Also at the hearing, Rep. Nanette Diaz Barragán, a California Democrat, spoke of her own reaction to seeing a question about mental health treatment while applying for a White House internship. During her college years, Rep. Barragán had to cope with her father’s terminal illness.

“I remember thinking to myself: ‘Jeez, if I end up seeing a mental health expert maybe one day I couldn’t work in government,’ ” she said.

A blood test detected 11 of 11 cases of colorectal cancer in a study involving 354 patients, and also spotted a majority of cases – 40 out of 53 – in which participants had advanced adenomas, an investigator said.

Results from a single-center study of CellMax Life’s FirstSight blood test were released as a poster as part of the annual Digestive Disease Week^®, which was canceled because of COVID-19.

For a study conducted at one site, the Veterans Affairs Palo Alto (Calif.) Healthcare System, Shai Friedland, MD, and colleagues recruited 354 patients between ages 45 and 80 who were scheduled for elective colonoscopy. The researchers excluded people with a personal history of cancer or inflammatory bowel disease. They used CellMax’s FirstSight test on blood samples from the study participants.

The FirstSight test result was positive for colorectal cancer in all 11 patients in the study who were found by colonoscopy to have this condition, said Dr. Friedland, who is a professor of medicine at Stanford (Calif.) University and chief of gastroenterology at the VA Palo Alto Healthcare System. Thus, the test showed a sensitivity of 100% in this instance.

Among the 53 study participants found by colonoscopy to have advanced adenoma, 40 were positive on FirstSight; thus, so the test has a sensitivity of 75.5% for this result.

Among 79 patients who had negative colonoscopy results, meaning they were judged free of cancer or polyps, the test showed 8 as having signs of disease or growths.

“If you had a large adenoma that was removed years ago and now you have a negative colonoscopy, your score might still be high,” Dr. Friedland said in a recorded presentation for DDW. “In other words, the changes that are detectable in your blood might persist even after the polypectomy.”

He said there are plans to soon start a large-scale multicenter study of the CellMax assay.

“The blood test has the potential to fill an unmet need by giving patients a highly sensitive convenient option for colorectal cancer screening,” he said.

CellMax already is seeking to position its test as a more convenient alternative to either colonoscopy or the Cologuard screening test. Many patients put off cancer screening because of the need to take time off from work and the invasive nature of colonoscopy. Exact Sciences has used direct-to-consumer advertising to promote its Cologuard home-based test as a more convenient alternative to colonoscopy, but its product requires patients to collect their own stool samples and mail them to a lab, a process many people find off-putting.

Public health advocates, including the U.S. Preventive Services Task Force (USPSTF), have for years been pressing for wider screening of American adults for colon cancer. USPSTF is in the midst of updating its recommendations on colon cancer. In announcing its latest update of these recommendations in 2016, USPSTF said “the best screening test is the one that gets done” (JAMA. 2016;315[23]:2564-75).

USPSTF pressed for maximizing the total proportion of the eligible population, a point Dr. Friedland echoed in a CellMax press release.

“For colon cancer screening to be most effective, it is essential to detect precancerous polyps and then perform a colonoscopy to remove the polyps,” said Dr. Friedland in the CellMax press release. “Giving patients the option of getting a blood test for screening would undoubtedly increase compliance and thereby reduce mortality from colorectal cancer.”

In the DDW presentation, Dr. Friedland and colleagues also said the CellMax test showed greater sensitivity (100%) for colorectal cancer and advanced precancerous lesions (75.5%) than did Cologuard (92.3% for colorectal cancer and 42.4% for advanced precancerous lesions).

Cara Connelly, Director of Public Relations and Corporate Communications for Exact Sciences said that the company “is dedicated to getting more people screened for colorectal cancer and applaud the researchers for their efforts. We look forward to hearing more about the performance of this test in a prospective multisite study with nonsymptomatic patients.”

Naresh T. Gunaratnam, MD, a gastroenterologist and research director at Huron Gastro in Ypsilanti, Mich., said he is concerned that aggressive promotion of alternative tests may obscure the benefits of colonoscopy. Dr. Gunaratnam, a 2019 winner of the American Gastroenterological Association (AGA) Distinguished Clinician Award, has been a public critic of the marketing of colon cancer tests, which emphasize the convenience of these products. When asked by MDedge to comment on the CellMax-funded study, Dr. Gunaratnam said alternative tests do have a place for the care of patients who cannot or will not have a colonoscopy.

“But if you convince a patient who would be willing to have a colonoscopy not to, that’s a disservice,” he said.

“If you want the best test, the one that is best at finding cancers and finding polyps and the only one that can remove the polyp, that’s colonoscopy,” Dr. Gunaratnam added. “One day there may be a pill you can swallow that blows up the polyps, but we’re not there yet. We have to mechanically remove them.”

SOURCE: Friedland S et al. DDW 2020, eposter 575.

A blood test detected 11 of 11 cases of colorectal cancer in a study involving 354 patients, and also spotted a majority of cases – 40 out of 53 – in which participants had advanced adenomas, an investigator said.

Results from a single-center study of CellMax Life’s FirstSight blood test were released as a poster as part of the annual Digestive Disease Week^®, which was canceled because of COVID-19.

For a study conducted at one site, the Veterans Affairs Palo Alto (Calif.) Healthcare System, Shai Friedland, MD, and colleagues recruited 354 patients between ages 45 and 80 who were scheduled for elective colonoscopy. The researchers excluded people with a personal history of cancer or inflammatory bowel disease. They used CellMax’s FirstSight test on blood samples from the study participants.

The FirstSight test result was positive for colorectal cancer in all 11 patients in the study who were found by colonoscopy to have this condition, said Dr. Friedland, who is a professor of medicine at Stanford (Calif.) University and chief of gastroenterology at the VA Palo Alto Healthcare System. Thus, the test showed a sensitivity of 100% in this instance.

Among the 53 study participants found by colonoscopy to have advanced adenoma, 40 were positive on FirstSight; thus, so the test has a sensitivity of 75.5% for this result.

Among 79 patients who had negative colonoscopy results, meaning they were judged free of cancer or polyps, the test showed 8 as having signs of disease or growths.

“If you had a large adenoma that was removed years ago and now you have a negative colonoscopy, your score might still be high,” Dr. Friedland said in a recorded presentation for DDW. “In other words, the changes that are detectable in your blood might persist even after the polypectomy.”

He said there are plans to soon start a large-scale multicenter study of the CellMax assay.

“The blood test has the potential to fill an unmet need by giving patients a highly sensitive convenient option for colorectal cancer screening,” he said.

CellMax already is seeking to position its test as a more convenient alternative to either colonoscopy or the Cologuard screening test. Many patients put off cancer screening because of the need to take time off from work and the invasive nature of colonoscopy. Exact Sciences has used direct-to-consumer advertising to promote its Cologuard home-based test as a more convenient alternative to colonoscopy, but its product requires patients to collect their own stool samples and mail them to a lab, a process many people find off-putting.

Public health advocates, including the U.S. Preventive Services Task Force (USPSTF), have for years been pressing for wider screening of American adults for colon cancer. USPSTF is in the midst of updating its recommendations on colon cancer. In announcing its latest update of these recommendations in 2016, USPSTF said “the best screening test is the one that gets done” (JAMA. 2016;315[23]:2564-75).

USPSTF pressed for maximizing the total proportion of the eligible population, a point Dr. Friedland echoed in a CellMax press release.

“For colon cancer screening to be most effective, it is essential to detect precancerous polyps and then perform a colonoscopy to remove the polyps,” said Dr. Friedland in the CellMax press release. “Giving patients the option of getting a blood test for screening would undoubtedly increase compliance and thereby reduce mortality from colorectal cancer.”

In the DDW presentation, Dr. Friedland and colleagues also said the CellMax test showed greater sensitivity (100%) for colorectal cancer and advanced precancerous lesions (75.5%) than did Cologuard (92.3% for colorectal cancer and 42.4% for advanced precancerous lesions).

Cara Connelly, Director of Public Relations and Corporate Communications for Exact Sciences said that the company “is dedicated to getting more people screened for colorectal cancer and applaud the researchers for their efforts. We look forward to hearing more about the performance of this test in a prospective multisite study with nonsymptomatic patients.”

Naresh T. Gunaratnam, MD, a gastroenterologist and research director at Huron Gastro in Ypsilanti, Mich., said he is concerned that aggressive promotion of alternative tests may obscure the benefits of colonoscopy. Dr. Gunaratnam, a 2019 winner of the American Gastroenterological Association (AGA) Distinguished Clinician Award, has been a public critic of the marketing of colon cancer tests, which emphasize the convenience of these products. When asked by MDedge to comment on the CellMax-funded study, Dr. Gunaratnam said alternative tests do have a place for the care of patients who cannot or will not have a colonoscopy.

“But if you convince a patient who would be willing to have a colonoscopy not to, that’s a disservice,” he said.

“If you want the best test, the one that is best at finding cancers and finding polyps and the only one that can remove the polyp, that’s colonoscopy,” Dr. Gunaratnam added. “One day there may be a pill you can swallow that blows up the polyps, but we’re not there yet. We have to mechanically remove them.”

SOURCE: Friedland S et al. DDW 2020, eposter 575.

A blood test detected 11 of 11 cases of colorectal cancer in a study involving 354 patients, and also spotted a majority of cases – 40 out of 53 – in which participants had advanced adenomas, an investigator said.

Results from a single-center study of CellMax Life’s FirstSight blood test were released as a poster as part of the annual Digestive Disease Week^®, which was canceled because of COVID-19.

For a study conducted at one site, the Veterans Affairs Palo Alto (Calif.) Healthcare System, Shai Friedland, MD, and colleagues recruited 354 patients between ages 45 and 80 who were scheduled for elective colonoscopy. The researchers excluded people with a personal history of cancer or inflammatory bowel disease. They used CellMax’s FirstSight test on blood samples from the study participants.

The FirstSight test result was positive for colorectal cancer in all 11 patients in the study who were found by colonoscopy to have this condition, said Dr. Friedland, who is a professor of medicine at Stanford (Calif.) University and chief of gastroenterology at the VA Palo Alto Healthcare System. Thus, the test showed a sensitivity of 100% in this instance.

Among the 53 study participants found by colonoscopy to have advanced adenoma, 40 were positive on FirstSight; thus, so the test has a sensitivity of 75.5% for this result.

Among 79 patients who had negative colonoscopy results, meaning they were judged free of cancer or polyps, the test showed 8 as having signs of disease or growths.

“If you had a large adenoma that was removed years ago and now you have a negative colonoscopy, your score might still be high,” Dr. Friedland said in a recorded presentation for DDW. “In other words, the changes that are detectable in your blood might persist even after the polypectomy.”

He said there are plans to soon start a large-scale multicenter study of the CellMax assay.

“The blood test has the potential to fill an unmet need by giving patients a highly sensitive convenient option for colorectal cancer screening,” he said.

CellMax already is seeking to position its test as a more convenient alternative to either colonoscopy or the Cologuard screening test. Many patients put off cancer screening because of the need to take time off from work and the invasive nature of colonoscopy. Exact Sciences has used direct-to-consumer advertising to promote its Cologuard home-based test as a more convenient alternative to colonoscopy, but its product requires patients to collect their own stool samples and mail them to a lab, a process many people find off-putting.

Public health advocates, including the U.S. Preventive Services Task Force (USPSTF), have for years been pressing for wider screening of American adults for colon cancer. USPSTF is in the midst of updating its recommendations on colon cancer. In announcing its latest update of these recommendations in 2016, USPSTF said “the best screening test is the one that gets done” (JAMA. 2016;315[23]:2564-75).

USPSTF pressed for maximizing the total proportion of the eligible population, a point Dr. Friedland echoed in a CellMax press release.

“For colon cancer screening to be most effective, it is essential to detect precancerous polyps and then perform a colonoscopy to remove the polyps,” said Dr. Friedland in the CellMax press release. “Giving patients the option of getting a blood test for screening would undoubtedly increase compliance and thereby reduce mortality from colorectal cancer.”

In the DDW presentation, Dr. Friedland and colleagues also said the CellMax test showed greater sensitivity (100%) for colorectal cancer and advanced precancerous lesions (75.5%) than did Cologuard (92.3% for colorectal cancer and 42.4% for advanced precancerous lesions).

Cara Connelly, Director of Public Relations and Corporate Communications for Exact Sciences said that the company “is dedicated to getting more people screened for colorectal cancer and applaud the researchers for their efforts. We look forward to hearing more about the performance of this test in a prospective multisite study with nonsymptomatic patients.”

Naresh T. Gunaratnam, MD, a gastroenterologist and research director at Huron Gastro in Ypsilanti, Mich., said he is concerned that aggressive promotion of alternative tests may obscure the benefits of colonoscopy. Dr. Gunaratnam, a 2019 winner of the American Gastroenterological Association (AGA) Distinguished Clinician Award, has been a public critic of the marketing of colon cancer tests, which emphasize the convenience of these products. When asked by MDedge to comment on the CellMax-funded study, Dr. Gunaratnam said alternative tests do have a place for the care of patients who cannot or will not have a colonoscopy.

“But if you convince a patient who would be willing to have a colonoscopy not to, that’s a disservice,” he said.

“If you want the best test, the one that is best at finding cancers and finding polyps and the only one that can remove the polyp, that’s colonoscopy,” Dr. Gunaratnam added. “One day there may be a pill you can swallow that blows up the polyps, but we’re not there yet. We have to mechanically remove them.”

SOURCE: Friedland S et al. DDW 2020, eposter 575.

A study using deidentified medical data for 65 million people found those who had used the recalled H₂ blocker, ranitidine, were not more likely than those who had taken the similar famotidine to develop cancer. People who had taken either of these drugs had a higher risk for cancer, but they also were more likely to have risk factors for this disease such as obesity or a history of smoking, an investigator said.

The findings from Nabeeha Mohyuddin, MD, an internal medicine resident at Allegheny Health Network in Pittsburgh, appear to be at odds with warnings from the U.S. Food and Drug Administration. The FDA in April called on manufacturers to pull all versions of ranitidine because of a problem with probable contamination by human carcinogen n-nitrosodimethylamine (NDMA). The FDA and the European Medicines Agency last year announced investigations of this NDMA contamination.

Dr. Mohyuddin and colleagues used IBM’s Explorys database, which includes data collected from EMRs from more than 40 U.S. health systems, to see if ranitidine use appeared connected to cancer diagnoses. Dr. Mohyuddin presented the findings in an abstract released as part of the annual Digestive Disease Week^®, which was canceled because of COVID-19. The researchers identified records for 1.62 million users of ranitidine, 3.37 million users of famotidine, and 59.63 million people who did not use either H₂ blocker.

The incidence of cancer was respectively 14.69%, 21.24%, and 5.38% for the ranitidine group, the famotidine group, and the group representing the general population without use of either H₂ blocker. Among subjects without risk factors including smoking, obesity, alcohol use, family history, cirrhosis, and gastroesophageal reflux disease, ranitidine users did not have an increased risk of cancer, compared with famotidine users (odds ratio, 0.77; 95% confidence interval, 0.76-0.77; P < .001), Dr. Mohyuddin said in an email interview after her presentation.

“The results need to be interpreted with caution given that this is a retrospective study and it’s the first of its kind,” she said. “Further studies will be definitely needed on this to definitively answer the question, ‘does ranitidine have an increased risk of cancer?’ ”

People in the study who used H₂ blockers tended to be older and were more likely to have other risk factors for cancers, according to the abstract:

• Of the ranitidine group, about 33.6% were older than 65, 74.4% were smokers, and 8.9% had a body mass index above 30 kg/m².
• Of the famotidine group, about 38.3% were older than 65, 76.9% were smokers and 10.8% had a BMI above 30.
• Of the general population, about 23.9% were older than 65, 27.5% were smokers, and 1.69% were obese.

The Explorys database accounts for only 15%-19% of the entire U.S. population and that could be an explanation for why the percentage for obesity in the population seems spuriously low, Dr. Mohyuddin said in an email exchange.

Additionally, it pulls in patients through diagnosis codes, and if a different code for obesity was entered, those patients may not have been accounted for, she said.

Dhyanesh A. Patel, MD, assistant professor of medicine at Vanderbilt University, Nashville, Tenn., said it was a surprise to see the rate of cancer reported for famotidine users in the abstract presented at DDW.

“But these databases are so prone to multiple biases that it’s really hard to tease out. You’d really have to do prospective studies where you follow these patients and then control for variables to know the true risk,” Dr. Patel said in an interview.

In many cases, for example, patients may start on H₂ blockers after reporting symptoms that sound like indigestion, Patel said.

“And then the next week, they actually get an upper endoscopy and they get diagnosed with gastric cancer,” Patel said. “In the database, it may seem to be that ranitidine was started and the patient was diagnosed with gastric cancer. So now the gastric cancer gets associated with ranitidine.”

Physicians have had many options to use instead of ranitidine since the FDA last year announced its concerns about the drug, Dr. Patel said.

“Patients can easily switch over to a similar histamine blocker or you can use one of the proton pump inhibitors that haven’t been found to have this impurity in them,” he said. “Both of those are good options for patients.”
 

Further research

Manufacturers have been withdrawing their ranitidine products from the market since the reports of contamination surfaced last year. Several drugmakers, including Pfizer and Perrigo, have reported facing lawsuits connected with claims of cancer or increased cancer risk from ranitidine. In February 2020, a federal judicial panel opted to use a procedure, known as multidistrict litigation (MDL), to streamline the handling of these many cases. They were put before the U.S. District Court for the Southern District of Florida, labeled as In Re: Zantac (Ranitidine) NDMA Litigation.

A spokesman for FDA declined MDedge’s request for comment on Dr. Mohyuddin’s DDW presentation. He said the agency tends not to offer its views on work done by scientists outside of the FDA.

Valisure, an online pharmacy that runs quality checks on the medicines it dispenses, in September 2019 petitioned the FDA for a withdrawal of ranitidine due to concerns about NDMA. In its petition, Valisure reported finding notable levels of NDMA in ranitidine tablets, but not detecting it in testing of famotidine and several similar drugs. In an emailed statement, David Light, founder and CEO of Valisure, said the structure of Dr. Mohyuddin’s research limited its ability to detect cancer correlations because of the large and generalized study population.

“When comparing millions of people on medications which are both over-the-counter and prescription, any epidemiological impact will very likely be eclipsed by the sheer variations of exposure and a wide variety of confounding factors,” Mr. Light wrote.

There are a “vast number of variables that aren’t controlled for in such a massive and broadly defined cohort,” he added.

More focused and controlled studies will be needed to best evaluate NDMA and ranitidine, according to Mr. Light. “We are also investigating this issue with researchers at Memorial Sloan Kettering Cancer Center and plan to publish results soon,” he said.

Dr. Mohyuddin and Dr. Patel did not disclose financial conflicts in connection with ranitidine.

SOURCE: Mohyuddin N et al. DDW 2020. Abstract Tu1360.

A study using deidentified medical data for 65 million people found those who had used the recalled H₂ blocker, ranitidine, were not more likely than those who had taken the similar famotidine to develop cancer. People who had taken either of these drugs had a higher risk for cancer, but they also were more likely to have risk factors for this disease such as obesity or a history of smoking, an investigator said.

The findings from Nabeeha Mohyuddin, MD, an internal medicine resident at Allegheny Health Network in Pittsburgh, appear to be at odds with warnings from the U.S. Food and Drug Administration. The FDA in April called on manufacturers to pull all versions of ranitidine because of a problem with probable contamination by human carcinogen n-nitrosodimethylamine (NDMA). The FDA and the European Medicines Agency last year announced investigations of this NDMA contamination.

Dr. Mohyuddin and colleagues used IBM’s Explorys database, which includes data collected from EMRs from more than 40 U.S. health systems, to see if ranitidine use appeared connected to cancer diagnoses. Dr. Mohyuddin presented the findings in an abstract released as part of the annual Digestive Disease Week^®, which was canceled because of COVID-19. The researchers identified records for 1.62 million users of ranitidine, 3.37 million users of famotidine, and 59.63 million people who did not use either H₂ blocker.

The incidence of cancer was respectively 14.69%, 21.24%, and 5.38% for the ranitidine group, the famotidine group, and the group representing the general population without use of either H₂ blocker. Among subjects without risk factors including smoking, obesity, alcohol use, family history, cirrhosis, and gastroesophageal reflux disease, ranitidine users did not have an increased risk of cancer, compared with famotidine users (odds ratio, 0.77; 95% confidence interval, 0.76-0.77; P < .001), Dr. Mohyuddin said in an email interview after her presentation.

“The results need to be interpreted with caution given that this is a retrospective study and it’s the first of its kind,” she said. “Further studies will be definitely needed on this to definitively answer the question, ‘does ranitidine have an increased risk of cancer?’ ”

People in the study who used H₂ blockers tended to be older and were more likely to have other risk factors for cancers, according to the abstract:

• Of the ranitidine group, about 33.6% were older than 65, 74.4% were smokers, and 8.9% had a body mass index above 30 kg/m².
• Of the famotidine group, about 38.3% were older than 65, 76.9% were smokers and 10.8% had a BMI above 30.
• Of the general population, about 23.9% were older than 65, 27.5% were smokers, and 1.69% were obese.

The Explorys database accounts for only 15%-19% of the entire U.S. population and that could be an explanation for why the percentage for obesity in the population seems spuriously low, Dr. Mohyuddin said in an email exchange.

Additionally, it pulls in patients through diagnosis codes, and if a different code for obesity was entered, those patients may not have been accounted for, she said.

Dhyanesh A. Patel, MD, assistant professor of medicine at Vanderbilt University, Nashville, Tenn., said it was a surprise to see the rate of cancer reported for famotidine users in the abstract presented at DDW.

“But these databases are so prone to multiple biases that it’s really hard to tease out. You’d really have to do prospective studies where you follow these patients and then control for variables to know the true risk,” Dr. Patel said in an interview.

In many cases, for example, patients may start on H₂ blockers after reporting symptoms that sound like indigestion, Patel said.

“And then the next week, they actually get an upper endoscopy and they get diagnosed with gastric cancer,” Patel said. “In the database, it may seem to be that ranitidine was started and the patient was diagnosed with gastric cancer. So now the gastric cancer gets associated with ranitidine.”

Physicians have had many options to use instead of ranitidine since the FDA last year announced its concerns about the drug, Dr. Patel said.

“Patients can easily switch over to a similar histamine blocker or you can use one of the proton pump inhibitors that haven’t been found to have this impurity in them,” he said. “Both of those are good options for patients.”
 

Further research

Manufacturers have been withdrawing their ranitidine products from the market since the reports of contamination surfaced last year. Several drugmakers, including Pfizer and Perrigo, have reported facing lawsuits connected with claims of cancer or increased cancer risk from ranitidine. In February 2020, a federal judicial panel opted to use a procedure, known as multidistrict litigation (MDL), to streamline the handling of these many cases. They were put before the U.S. District Court for the Southern District of Florida, labeled as In Re: Zantac (Ranitidine) NDMA Litigation.

A spokesman for FDA declined MDedge’s request for comment on Dr. Mohyuddin’s DDW presentation. He said the agency tends not to offer its views on work done by scientists outside of the FDA.

Valisure, an online pharmacy that runs quality checks on the medicines it dispenses, in September 2019 petitioned the FDA for a withdrawal of ranitidine due to concerns about NDMA. In its petition, Valisure reported finding notable levels of NDMA in ranitidine tablets, but not detecting it in testing of famotidine and several similar drugs. In an emailed statement, David Light, founder and CEO of Valisure, said the structure of Dr. Mohyuddin’s research limited its ability to detect cancer correlations because of the large and generalized study population.

“When comparing millions of people on medications which are both over-the-counter and prescription, any epidemiological impact will very likely be eclipsed by the sheer variations of exposure and a wide variety of confounding factors,” Mr. Light wrote.

There are a “vast number of variables that aren’t controlled for in such a massive and broadly defined cohort,” he added.

More focused and controlled studies will be needed to best evaluate NDMA and ranitidine, according to Mr. Light. “We are also investigating this issue with researchers at Memorial Sloan Kettering Cancer Center and plan to publish results soon,” he said.

Dr. Mohyuddin and Dr. Patel did not disclose financial conflicts in connection with ranitidine.

SOURCE: Mohyuddin N et al. DDW 2020. Abstract Tu1360.

A study using deidentified medical data for 65 million people found those who had used the recalled H₂ blocker, ranitidine, were not more likely than those who had taken the similar famotidine to develop cancer. People who had taken either of these drugs had a higher risk for cancer, but they also were more likely to have risk factors for this disease such as obesity or a history of smoking, an investigator said.

The findings from Nabeeha Mohyuddin, MD, an internal medicine resident at Allegheny Health Network in Pittsburgh, appear to be at odds with warnings from the U.S. Food and Drug Administration. The FDA in April called on manufacturers to pull all versions of ranitidine because of a problem with probable contamination by human carcinogen n-nitrosodimethylamine (NDMA). The FDA and the European Medicines Agency last year announced investigations of this NDMA contamination.

Dr. Mohyuddin and colleagues used IBM’s Explorys database, which includes data collected from EMRs from more than 40 U.S. health systems, to see if ranitidine use appeared connected to cancer diagnoses. Dr. Mohyuddin presented the findings in an abstract released as part of the annual Digestive Disease Week^®, which was canceled because of COVID-19. The researchers identified records for 1.62 million users of ranitidine, 3.37 million users of famotidine, and 59.63 million people who did not use either H₂ blocker.

The incidence of cancer was respectively 14.69%, 21.24%, and 5.38% for the ranitidine group, the famotidine group, and the group representing the general population without use of either H₂ blocker. Among subjects without risk factors including smoking, obesity, alcohol use, family history, cirrhosis, and gastroesophageal reflux disease, ranitidine users did not have an increased risk of cancer, compared with famotidine users (odds ratio, 0.77; 95% confidence interval, 0.76-0.77; P < .001), Dr. Mohyuddin said in an email interview after her presentation.

“The results need to be interpreted with caution given that this is a retrospective study and it’s the first of its kind,” she said. “Further studies will be definitely needed on this to definitively answer the question, ‘does ranitidine have an increased risk of cancer?’ ”

People in the study who used H₂ blockers tended to be older and were more likely to have other risk factors for cancers, according to the abstract:

• Of the ranitidine group, about 33.6% were older than 65, 74.4% were smokers, and 8.9% had a body mass index above 30 kg/m².
• Of the famotidine group, about 38.3% were older than 65, 76.9% were smokers and 10.8% had a BMI above 30.
• Of the general population, about 23.9% were older than 65, 27.5% were smokers, and 1.69% were obese.

The Explorys database accounts for only 15%-19% of the entire U.S. population and that could be an explanation for why the percentage for obesity in the population seems spuriously low, Dr. Mohyuddin said in an email exchange.

Additionally, it pulls in patients through diagnosis codes, and if a different code for obesity was entered, those patients may not have been accounted for, she said.

Dhyanesh A. Patel, MD, assistant professor of medicine at Vanderbilt University, Nashville, Tenn., said it was a surprise to see the rate of cancer reported for famotidine users in the abstract presented at DDW.

“But these databases are so prone to multiple biases that it’s really hard to tease out. You’d really have to do prospective studies where you follow these patients and then control for variables to know the true risk,” Dr. Patel said in an interview.

In many cases, for example, patients may start on H₂ blockers after reporting symptoms that sound like indigestion, Patel said.

“And then the next week, they actually get an upper endoscopy and they get diagnosed with gastric cancer,” Patel said. “In the database, it may seem to be that ranitidine was started and the patient was diagnosed with gastric cancer. So now the gastric cancer gets associated with ranitidine.”

Physicians have had many options to use instead of ranitidine since the FDA last year announced its concerns about the drug, Dr. Patel said.

“Patients can easily switch over to a similar histamine blocker or you can use one of the proton pump inhibitors that haven’t been found to have this impurity in them,” he said. “Both of those are good options for patients.”
 

Further research

Manufacturers have been withdrawing their ranitidine products from the market since the reports of contamination surfaced last year. Several drugmakers, including Pfizer and Perrigo, have reported facing lawsuits connected with claims of cancer or increased cancer risk from ranitidine. In February 2020, a federal judicial panel opted to use a procedure, known as multidistrict litigation (MDL), to streamline the handling of these many cases. They were put before the U.S. District Court for the Southern District of Florida, labeled as In Re: Zantac (Ranitidine) NDMA Litigation.

A spokesman for FDA declined MDedge’s request for comment on Dr. Mohyuddin’s DDW presentation. He said the agency tends not to offer its views on work done by scientists outside of the FDA.

Valisure, an online pharmacy that runs quality checks on the medicines it dispenses, in September 2019 petitioned the FDA for a withdrawal of ranitidine due to concerns about NDMA. In its petition, Valisure reported finding notable levels of NDMA in ranitidine tablets, but not detecting it in testing of famotidine and several similar drugs. In an emailed statement, David Light, founder and CEO of Valisure, said the structure of Dr. Mohyuddin’s research limited its ability to detect cancer correlations because of the large and generalized study population.

“When comparing millions of people on medications which are both over-the-counter and prescription, any epidemiological impact will very likely be eclipsed by the sheer variations of exposure and a wide variety of confounding factors,” Mr. Light wrote.

There are a “vast number of variables that aren’t controlled for in such a massive and broadly defined cohort,” he added.

More focused and controlled studies will be needed to best evaluate NDMA and ranitidine, according to Mr. Light. “We are also investigating this issue with researchers at Memorial Sloan Kettering Cancer Center and plan to publish results soon,” he said.

Dr. Mohyuddin and Dr. Patel did not disclose financial conflicts in connection with ranitidine.

SOURCE: Mohyuddin N et al. DDW 2020. Abstract Tu1360.

Children whose parents are coping with illness and injuries were more likely to need medical treatment and prescriptions for gastrointestinal conditions, for which previous research already has indicated a brain-gut connection, an investigator said.

In a self-controlled case series using records from the Military Health System Data Repository, pediatric visits for disorders linked to gut-brain interactions were found to have increased 9% (incidence rate ratio, 1.09; 95% CI, 1.07-1.10) following a parent’s illness or injury, reported lead author Patrick Short, MD, of the Uniformed Services University of the Health Sciences, Bethesda, Md., said in an interview. The Military Health System Data Repository receives records from the Department of Defense’s global network of more than 260 medical facilities as well as outside health care organizations where military families are seen.

A secondary analysis done for this study found children of brain injured parents had 4% more postinjury visits for abdominal pain and 23% increased odds of antispasmodic prescription, compared with children whose parents had other physical injuries, Dr. Short said. He presented his research in an abstract released as part of the annual Digestive Disease Week, which was canceled because of COVID-19. The study focused on children aged 3-16 years with a parent who served in the military and was ill or injured between 2004 and 2014. Excluded from this research were records for children with diagnosed systemic or organic gastrointestinal disease, such as celiac disease.

The study used ICD-9 codes to identify outpatient visits for irritable bowel syndrome, abdominal pain, constipation, and fecal incontinence in the 2 years before and after parental injury or diagnosis of illness. Outpatient pharmacy records showed which of the children studied took laxatives and antispasmodics.

Parental injury or illness was defined by the placement of the children’s mothers and fathers on the injured, ill, or wounded file in the data repository. The data file generally covers people with conditions that severely limit their ability to do their usual jobs. These include traumatic brain injury, PTSD, amputation, shrapnel injury, and illnesses such as cancer.

There was a 7% increase in visits for constipation but fecal incontinence did not significantly change following parental illness or injury, Dr. Short said. But the odds of being prescribed an antispasmodic increased 23% following parents’ injuries and serious illnesses, while the odds for laxative prescription decreased by 5%.

The study highlights the potential physical impact of stress on children when families experience a crisis, Dr. Short said in an interview. Children may feel anxious about their parent’s health, while at the same time experiencing unavoidable disruption in family life because of an injury or illness.

“It impacts the day-to-day regimens and routines and decreases the family support,” Dr. Short said. “As humans we are limited in what we have to offer. When we are trying to take care of things on our own, it limits what we can give to people around us.”

The findings of this study should serve to remind physicians to alert parents that their children could experience worsening of GI conditions because of the stress of an ill or injured parent. They then can focus on securing help ahead of the time for the child, such as therapy, he said.

The next step in advancing on the research he prepared for DDW could be testing through prospective studies how well preventive measures such as family counseling work, Dr. Short said.

Dr. Short’s research adds to the growing body of evidence about the brain-gut connection, said Kara Gross Margolis, MD, a spokesperson for the American Gastroenterological Association. An associate professor of pediatrics at Columbia University Medical Center, New York, Dr. Margolis has published research on the brain-gut axis. Her lab focuses on the effects of neurotransmitters and inflammation on enteric nervous system development and function.

Physicians should take a broad view when treating children for functional GI illnesses. Behavioral therapy and antidepressants, for example, have been shown to help children with conditions such as irritable bowel syndrome and other functional gastrointestinal diseases, said Dr. Margolis.

“In a number of these cases, we not only have to treat the gut. We have to treat the brain as well,” Dr. Margolis said.

“When mental health issues are involved that impact the parents of these kids, You have to look at a family as an entire unit,” she added. “You not only treat the child for those symptoms, but you really have to look at how their parents can also be cared for so that their impact on their children will be positive as well.”

Research in the vein explored by Dr. Short will be important to remember as society works through the legacy of the COVID-19 pandemic, Dr. Margolis said. “We have huge numbers of families undergoing tremendous stress due to loss of jobs, health care, medical issues, and parental injury potentially from coronavirus.”

No outside funding was reported, and the study was covered through Uniformed Services University budget.

SOURCE: Short P et al. DDW 2020, Abstract 815.

Children whose parents are coping with illness and injuries were more likely to need medical treatment and prescriptions for gastrointestinal conditions, for which previous research already has indicated a brain-gut connection, an investigator said.

In a self-controlled case series using records from the Military Health System Data Repository, pediatric visits for disorders linked to gut-brain interactions were found to have increased 9% (incidence rate ratio, 1.09; 95% CI, 1.07-1.10) following a parent’s illness or injury, reported lead author Patrick Short, MD, of the Uniformed Services University of the Health Sciences, Bethesda, Md., said in an interview. The Military Health System Data Repository receives records from the Department of Defense’s global network of more than 260 medical facilities as well as outside health care organizations where military families are seen.

A secondary analysis done for this study found children of brain injured parents had 4% more postinjury visits for abdominal pain and 23% increased odds of antispasmodic prescription, compared with children whose parents had other physical injuries, Dr. Short said. He presented his research in an abstract released as part of the annual Digestive Disease Week, which was canceled because of COVID-19. The study focused on children aged 3-16 years with a parent who served in the military and was ill or injured between 2004 and 2014. Excluded from this research were records for children with diagnosed systemic or organic gastrointestinal disease, such as celiac disease.

The study used ICD-9 codes to identify outpatient visits for irritable bowel syndrome, abdominal pain, constipation, and fecal incontinence in the 2 years before and after parental injury or diagnosis of illness. Outpatient pharmacy records showed which of the children studied took laxatives and antispasmodics.

Parental injury or illness was defined by the placement of the children’s mothers and fathers on the injured, ill, or wounded file in the data repository. The data file generally covers people with conditions that severely limit their ability to do their usual jobs. These include traumatic brain injury, PTSD, amputation, shrapnel injury, and illnesses such as cancer.

There was a 7% increase in visits for constipation but fecal incontinence did not significantly change following parental illness or injury, Dr. Short said. But the odds of being prescribed an antispasmodic increased 23% following parents’ injuries and serious illnesses, while the odds for laxative prescription decreased by 5%.

The study highlights the potential physical impact of stress on children when families experience a crisis, Dr. Short said in an interview. Children may feel anxious about their parent’s health, while at the same time experiencing unavoidable disruption in family life because of an injury or illness.

“It impacts the day-to-day regimens and routines and decreases the family support,” Dr. Short said. “As humans we are limited in what we have to offer. When we are trying to take care of things on our own, it limits what we can give to people around us.”

The findings of this study should serve to remind physicians to alert parents that their children could experience worsening of GI conditions because of the stress of an ill or injured parent. They then can focus on securing help ahead of the time for the child, such as therapy, he said.

The next step in advancing on the research he prepared for DDW could be testing through prospective studies how well preventive measures such as family counseling work, Dr. Short said.

Dr. Short’s research adds to the growing body of evidence about the brain-gut connection, said Kara Gross Margolis, MD, a spokesperson for the American Gastroenterological Association. An associate professor of pediatrics at Columbia University Medical Center, New York, Dr. Margolis has published research on the brain-gut axis. Her lab focuses on the effects of neurotransmitters and inflammation on enteric nervous system development and function.

Physicians should take a broad view when treating children for functional GI illnesses. Behavioral therapy and antidepressants, for example, have been shown to help children with conditions such as irritable bowel syndrome and other functional gastrointestinal diseases, said Dr. Margolis.

“In a number of these cases, we not only have to treat the gut. We have to treat the brain as well,” Dr. Margolis said.

“When mental health issues are involved that impact the parents of these kids, You have to look at a family as an entire unit,” she added. “You not only treat the child for those symptoms, but you really have to look at how their parents can also be cared for so that their impact on their children will be positive as well.”

Research in the vein explored by Dr. Short will be important to remember as society works through the legacy of the COVID-19 pandemic, Dr. Margolis said. “We have huge numbers of families undergoing tremendous stress due to loss of jobs, health care, medical issues, and parental injury potentially from coronavirus.”

No outside funding was reported, and the study was covered through Uniformed Services University budget.

SOURCE: Short P et al. DDW 2020, Abstract 815.

Children whose parents are coping with illness and injuries were more likely to need medical treatment and prescriptions for gastrointestinal conditions, for which previous research already has indicated a brain-gut connection, an investigator said.

In a self-controlled case series using records from the Military Health System Data Repository, pediatric visits for disorders linked to gut-brain interactions were found to have increased 9% (incidence rate ratio, 1.09; 95% CI, 1.07-1.10) following a parent’s illness or injury, reported lead author Patrick Short, MD, of the Uniformed Services University of the Health Sciences, Bethesda, Md., said in an interview. The Military Health System Data Repository receives records from the Department of Defense’s global network of more than 260 medical facilities as well as outside health care organizations where military families are seen.

A secondary analysis done for this study found children of brain injured parents had 4% more postinjury visits for abdominal pain and 23% increased odds of antispasmodic prescription, compared with children whose parents had other physical injuries, Dr. Short said. He presented his research in an abstract released as part of the annual Digestive Disease Week, which was canceled because of COVID-19. The study focused on children aged 3-16 years with a parent who served in the military and was ill or injured between 2004 and 2014. Excluded from this research were records for children with diagnosed systemic or organic gastrointestinal disease, such as celiac disease.

The study used ICD-9 codes to identify outpatient visits for irritable bowel syndrome, abdominal pain, constipation, and fecal incontinence in the 2 years before and after parental injury or diagnosis of illness. Outpatient pharmacy records showed which of the children studied took laxatives and antispasmodics.

Parental injury or illness was defined by the placement of the children’s mothers and fathers on the injured, ill, or wounded file in the data repository. The data file generally covers people with conditions that severely limit their ability to do their usual jobs. These include traumatic brain injury, PTSD, amputation, shrapnel injury, and illnesses such as cancer.

There was a 7% increase in visits for constipation but fecal incontinence did not significantly change following parental illness or injury, Dr. Short said. But the odds of being prescribed an antispasmodic increased 23% following parents’ injuries and serious illnesses, while the odds for laxative prescription decreased by 5%.

The study highlights the potential physical impact of stress on children when families experience a crisis, Dr. Short said in an interview. Children may feel anxious about their parent’s health, while at the same time experiencing unavoidable disruption in family life because of an injury or illness.

“It impacts the day-to-day regimens and routines and decreases the family support,” Dr. Short said. “As humans we are limited in what we have to offer. When we are trying to take care of things on our own, it limits what we can give to people around us.”

The findings of this study should serve to remind physicians to alert parents that their children could experience worsening of GI conditions because of the stress of an ill or injured parent. They then can focus on securing help ahead of the time for the child, such as therapy, he said.

The next step in advancing on the research he prepared for DDW could be testing through prospective studies how well preventive measures such as family counseling work, Dr. Short said.

Dr. Short’s research adds to the growing body of evidence about the brain-gut connection, said Kara Gross Margolis, MD, a spokesperson for the American Gastroenterological Association. An associate professor of pediatrics at Columbia University Medical Center, New York, Dr. Margolis has published research on the brain-gut axis. Her lab focuses on the effects of neurotransmitters and inflammation on enteric nervous system development and function.

Physicians should take a broad view when treating children for functional GI illnesses. Behavioral therapy and antidepressants, for example, have been shown to help children with conditions such as irritable bowel syndrome and other functional gastrointestinal diseases, said Dr. Margolis.

“In a number of these cases, we not only have to treat the gut. We have to treat the brain as well,” Dr. Margolis said.

“When mental health issues are involved that impact the parents of these kids, You have to look at a family as an entire unit,” she added. “You not only treat the child for those symptoms, but you really have to look at how their parents can also be cared for so that their impact on their children will be positive as well.”

Research in the vein explored by Dr. Short will be important to remember as society works through the legacy of the COVID-19 pandemic, Dr. Margolis said. “We have huge numbers of families undergoing tremendous stress due to loss of jobs, health care, medical issues, and parental injury potentially from coronavirus.”

No outside funding was reported, and the study was covered through Uniformed Services University budget.

SOURCE: Short P et al. DDW 2020, Abstract 815.

A new advocacy organization is launching on April 28 to give “every physician” a voice in decisions that affect their professional lives. But this group doesn’t intend to use the top-down approach to decision making seen in many medical societies.

Paul Teirstein, MD, chief of cardiology for Scripps Clinic in La Jolla, Calif., and founder of the new organization United Physicians, said in an interviewit is a nonprofit group that will operate through online participation.

He said the intention is to use online voting and discussions among physicians across specialties to advocate for more and better legislation on pressing issues for their profession and, as noted on the group’s website, “protect the physician-patient relationship.”

Projects would need the support of a two-thirds majority of United Physicians’ members to proceed with any proposals. Meetings will be held publicly online, Dr. Teirstein explained.

There is a need for a broad-based organization that will respond to the voice of practicing physicians rather than dictate legislative priorities from management ranks, he said.

Dr. Teirstein said he learned how challenging it is to bring physicians together on issues in 2014 in his battles against changes in maintenance of certification rules. The result of his efforts was the National Board of Physicians and Surgeons (NBPAS), set up to provide a means of certification different from the one offered by the American Board of Internal Medicine.

Dr. Teirstein has argued that the approach of ABIM unfairly burdened physicians with a stepped-up schedule of testing and relied on an outdated approach to the practice of medicine.

Physicians busy with their practices feel they lack a unified voice in contesting the growing administrative burden and unproductive federal and state policies, Dr. Teirstein said.

He cited the limited enrollment in the largest physician groups as evidence of how disenfranchised many clinicians feel. There are about 1 million professional active physicians in the United States, according to the nonprofit Kaiser Family Foundation. Yet, even the largest physician group, the American Medical Association, has about 250,000 members, according to its 2018 annual report

“Clearly, most physicians believe they have little voice when it comes to health care decisions,” Dr. Teirstein said. “Our physician associations are governed from the top down. The leaders set the agenda. There may be delegates, but does leadership really listen to the delegates? Do the delegates really listen to the physician community?”

On its website, AMA describes itself as “physicians’ powerful ally in patient care” that works with more than 190 state and specialty medical societies. In recent months, James L. Madara, MD, the group’s chief executive officer, has urged governors to remove obstacles for physicians who want to fill workforce gaps in COVID-19 hot spots, among other actions.

In its annual report, the AMA, which declined to comment for this article, said its membership rose by 3.4% in 2018, double the growth rate of the previous year, thanks to a membership drive.

“The campaign celebrates the powerful work of our physician members and showcases how their individual efforts – along with the AMA – are moving medicine forward,” wrote Dr. Madara and other organization leaders in the report.

What Dr. Teirstein proposes is an inversion of the structure used by other medical societies, in which he says leaders and delegates dictate priorities.

United Physicians will use meetings and votes held by members online to decide which projects to pursue. Fees would be kept nominal, likely about $10 a year, depending on the number of members. Fees would be subject to change on the basis of expenses. The AMA has a sliding fee schedule that tops out with annual dues for physicians in regular practice of $420.

“There are no delegates, no representatives, and no board of directors. We want every physician to join and every physician to vote on every issue,” Dr. Teirstein said.

He stressed that he sees United Physicians as being complementary to the AMA.

“We do not compete with other organizations. Ideally, other organizations will use the platform,” Dr. Teirstein said. “If the AMA is considering a new policy, it can use the United Physicians platform to measure physician support. For example, through online discussions, petitions, and voting, it might learn a proposed policy needs a few tweaks to be accepted by most physicians.”

No compensation

Dr. Teirstein is among physician leaders who in recent years have sought to rally their colleagues to fight back against growing administrative burdens.

In a 2015 article in JAMA that was written with Medscape’s editor in chief, Eric Topol, MD, Dr. Teirstein criticized the ABIM’s drive to have physicians complete tests every 2 years and participate in continuous certification instead of recertifying once a decade, as had been the practice.

Dr. Teirstein formed the NBPAS as an alternative path for certification, with Dr. Topol serving on the board for that organization. Dr. Topol also will serve as a member of the advisory board for Teirstein’s United Physicians.

Dr. Topol wrote an article that appeared in the New Yorker last August that argued for physicians to move beyond the confines of medical societies and seek a path for broad-based activism. He said he intended to challenge medical societies, which, for all the good they do, can sometimes lose focus on that core relationship in favor of the bottom line.

Dr. Topol said in an interview that his colleague’s new project is a “good idea for a democratized platform at a time when physician solidarity is needed more than ever.”

Dr. Teirstein plans to run United Physicians on a volunteer basis. This builds on the approach he has used for NBPAS. He and the directors of the NBPAS will receive no compensation, he said, as was confirmed by the NBPAS.

In contrast, Dr. Madara made about $2.5 million in total compensation for 2018, according to the organization’s Internal Revenue Service filing. Physicians who served as trustees and officials for the AMA that year received annual compensation that ranged from around $60,000 to $291,980, depending on their duties.

“Having volunteer leadership mitigates conflict of interest. It also ensures leadership has a ‘day job’ that keeps them in touch with issues impacting practicing physicians,” Dr. Teirstein said

Start-up costs for United Physicians will be supported by NBPAS, but it will function as a completely independent organization, he added.

In introducing the group, Dr. Teirstein outlined suggestions for proposals it might pursue. These include making hospitals secure adequate supplies of personal protective equipment ahead of health crises such as the COVID-19 pandemic.

His outline also includes suggestions for issues that likely will persist beyond the response to the pandemic.

Dr. Teirstein proposed a project for persuading insurance companies to provide online calendar appointments for peer-to-peer patient preauthorization. Failure of the insurer’s representative to attend would trigger approval of authorization under this proposal. He also suggested a lobbying effort for specific reimbursement for peer-to-peer, patient preauthorization phone calls.

Dr. Teirstein said he hopes most of the proposals will come from physicians who join United Physicians. Still, it is unclear whether United Physicians will succeed. An initial challenge could be in sorting through a barrage of competing ideas submitted to United Physicians.

But Dr. Teirstein appears hopeful about the changes for this experiment in online advocacy. He intends for United Physicians to be a pathway for clinicians to translate their complaints about policies into calls for action, with only a short investment of their time.

“Most of us have wonderful, engrossing jobs. It’s hard to beat helping a patient, and most of us get to do it every day,” Dr. .Teirstein said. “Will we take the 30 seconds required to sign up and become a United Physicians member? Will we spend a little time each week reviewing the issues and voting? I think it’s an experiment worth watching.”

A version of this article originally appeared on Medscape.com.

A new advocacy organization is launching on April 28 to give “every physician” a voice in decisions that affect their professional lives. But this group doesn’t intend to use the top-down approach to decision making seen in many medical societies.

Paul Teirstein, MD, chief of cardiology for Scripps Clinic in La Jolla, Calif., and founder of the new organization United Physicians, said in an interviewit is a nonprofit group that will operate through online participation.

He said the intention is to use online voting and discussions among physicians across specialties to advocate for more and better legislation on pressing issues for their profession and, as noted on the group’s website, “protect the physician-patient relationship.”

Projects would need the support of a two-thirds majority of United Physicians’ members to proceed with any proposals. Meetings will be held publicly online, Dr. Teirstein explained.

There is a need for a broad-based organization that will respond to the voice of practicing physicians rather than dictate legislative priorities from management ranks, he said.

Dr. Teirstein said he learned how challenging it is to bring physicians together on issues in 2014 in his battles against changes in maintenance of certification rules. The result of his efforts was the National Board of Physicians and Surgeons (NBPAS), set up to provide a means of certification different from the one offered by the American Board of Internal Medicine.

Dr. Teirstein has argued that the approach of ABIM unfairly burdened physicians with a stepped-up schedule of testing and relied on an outdated approach to the practice of medicine.

Physicians busy with their practices feel they lack a unified voice in contesting the growing administrative burden and unproductive federal and state policies, Dr. Teirstein said.

He cited the limited enrollment in the largest physician groups as evidence of how disenfranchised many clinicians feel. There are about 1 million professional active physicians in the United States, according to the nonprofit Kaiser Family Foundation. Yet, even the largest physician group, the American Medical Association, has about 250,000 members, according to its 2018 annual report

“Clearly, most physicians believe they have little voice when it comes to health care decisions,” Dr. Teirstein said. “Our physician associations are governed from the top down. The leaders set the agenda. There may be delegates, but does leadership really listen to the delegates? Do the delegates really listen to the physician community?”

On its website, AMA describes itself as “physicians’ powerful ally in patient care” that works with more than 190 state and specialty medical societies. In recent months, James L. Madara, MD, the group’s chief executive officer, has urged governors to remove obstacles for physicians who want to fill workforce gaps in COVID-19 hot spots, among other actions.

In its annual report, the AMA, which declined to comment for this article, said its membership rose by 3.4% in 2018, double the growth rate of the previous year, thanks to a membership drive.

“The campaign celebrates the powerful work of our physician members and showcases how their individual efforts – along with the AMA – are moving medicine forward,” wrote Dr. Madara and other organization leaders in the report.

What Dr. Teirstein proposes is an inversion of the structure used by other medical societies, in which he says leaders and delegates dictate priorities.

United Physicians will use meetings and votes held by members online to decide which projects to pursue. Fees would be kept nominal, likely about $10 a year, depending on the number of members. Fees would be subject to change on the basis of expenses. The AMA has a sliding fee schedule that tops out with annual dues for physicians in regular practice of $420.

“There are no delegates, no representatives, and no board of directors. We want every physician to join and every physician to vote on every issue,” Dr. Teirstein said.

He stressed that he sees United Physicians as being complementary to the AMA.

“We do not compete with other organizations. Ideally, other organizations will use the platform,” Dr. Teirstein said. “If the AMA is considering a new policy, it can use the United Physicians platform to measure physician support. For example, through online discussions, petitions, and voting, it might learn a proposed policy needs a few tweaks to be accepted by most physicians.”

No compensation

Dr. Teirstein is among physician leaders who in recent years have sought to rally their colleagues to fight back against growing administrative burdens.

In a 2015 article in JAMA that was written with Medscape’s editor in chief, Eric Topol, MD, Dr. Teirstein criticized the ABIM’s drive to have physicians complete tests every 2 years and participate in continuous certification instead of recertifying once a decade, as had been the practice.

Dr. Teirstein formed the NBPAS as an alternative path for certification, with Dr. Topol serving on the board for that organization. Dr. Topol also will serve as a member of the advisory board for Teirstein’s United Physicians.

Dr. Topol wrote an article that appeared in the New Yorker last August that argued for physicians to move beyond the confines of medical societies and seek a path for broad-based activism. He said he intended to challenge medical societies, which, for all the good they do, can sometimes lose focus on that core relationship in favor of the bottom line.

Dr. Topol said in an interview that his colleague’s new project is a “good idea for a democratized platform at a time when physician solidarity is needed more than ever.”

Dr. Teirstein plans to run United Physicians on a volunteer basis. This builds on the approach he has used for NBPAS. He and the directors of the NBPAS will receive no compensation, he said, as was confirmed by the NBPAS.

In contrast, Dr. Madara made about $2.5 million in total compensation for 2018, according to the organization’s Internal Revenue Service filing. Physicians who served as trustees and officials for the AMA that year received annual compensation that ranged from around $60,000 to $291,980, depending on their duties.

“Having volunteer leadership mitigates conflict of interest. It also ensures leadership has a ‘day job’ that keeps them in touch with issues impacting practicing physicians,” Dr. Teirstein said

Start-up costs for United Physicians will be supported by NBPAS, but it will function as a completely independent organization, he added.

In introducing the group, Dr. Teirstein outlined suggestions for proposals it might pursue. These include making hospitals secure adequate supplies of personal protective equipment ahead of health crises such as the COVID-19 pandemic.

His outline also includes suggestions for issues that likely will persist beyond the response to the pandemic.

Dr. Teirstein proposed a project for persuading insurance companies to provide online calendar appointments for peer-to-peer patient preauthorization. Failure of the insurer’s representative to attend would trigger approval of authorization under this proposal. He also suggested a lobbying effort for specific reimbursement for peer-to-peer, patient preauthorization phone calls.

Dr. Teirstein said he hopes most of the proposals will come from physicians who join United Physicians. Still, it is unclear whether United Physicians will succeed. An initial challenge could be in sorting through a barrage of competing ideas submitted to United Physicians.

But Dr. Teirstein appears hopeful about the changes for this experiment in online advocacy. He intends for United Physicians to be a pathway for clinicians to translate their complaints about policies into calls for action, with only a short investment of their time.

“Most of us have wonderful, engrossing jobs. It’s hard to beat helping a patient, and most of us get to do it every day,” Dr. .Teirstein said. “Will we take the 30 seconds required to sign up and become a United Physicians member? Will we spend a little time each week reviewing the issues and voting? I think it’s an experiment worth watching.”

A version of this article originally appeared on Medscape.com.

A new advocacy organization is launching on April 28 to give “every physician” a voice in decisions that affect their professional lives. But this group doesn’t intend to use the top-down approach to decision making seen in many medical societies.

Paul Teirstein, MD, chief of cardiology for Scripps Clinic in La Jolla, Calif., and founder of the new organization United Physicians, said in an interviewit is a nonprofit group that will operate through online participation.

He said the intention is to use online voting and discussions among physicians across specialties to advocate for more and better legislation on pressing issues for their profession and, as noted on the group’s website, “protect the physician-patient relationship.”

Projects would need the support of a two-thirds majority of United Physicians’ members to proceed with any proposals. Meetings will be held publicly online, Dr. Teirstein explained.

There is a need for a broad-based organization that will respond to the voice of practicing physicians rather than dictate legislative priorities from management ranks, he said.

Dr. Teirstein said he learned how challenging it is to bring physicians together on issues in 2014 in his battles against changes in maintenance of certification rules. The result of his efforts was the National Board of Physicians and Surgeons (NBPAS), set up to provide a means of certification different from the one offered by the American Board of Internal Medicine.

Dr. Teirstein has argued that the approach of ABIM unfairly burdened physicians with a stepped-up schedule of testing and relied on an outdated approach to the practice of medicine.

Physicians busy with their practices feel they lack a unified voice in contesting the growing administrative burden and unproductive federal and state policies, Dr. Teirstein said.

He cited the limited enrollment in the largest physician groups as evidence of how disenfranchised many clinicians feel. There are about 1 million professional active physicians in the United States, according to the nonprofit Kaiser Family Foundation. Yet, even the largest physician group, the American Medical Association, has about 250,000 members, according to its 2018 annual report

“Clearly, most physicians believe they have little voice when it comes to health care decisions,” Dr. Teirstein said. “Our physician associations are governed from the top down. The leaders set the agenda. There may be delegates, but does leadership really listen to the delegates? Do the delegates really listen to the physician community?”

On its website, AMA describes itself as “physicians’ powerful ally in patient care” that works with more than 190 state and specialty medical societies. In recent months, James L. Madara, MD, the group’s chief executive officer, has urged governors to remove obstacles for physicians who want to fill workforce gaps in COVID-19 hot spots, among other actions.

In its annual report, the AMA, which declined to comment for this article, said its membership rose by 3.4% in 2018, double the growth rate of the previous year, thanks to a membership drive.

“The campaign celebrates the powerful work of our physician members and showcases how their individual efforts – along with the AMA – are moving medicine forward,” wrote Dr. Madara and other organization leaders in the report.

What Dr. Teirstein proposes is an inversion of the structure used by other medical societies, in which he says leaders and delegates dictate priorities.

United Physicians will use meetings and votes held by members online to decide which projects to pursue. Fees would be kept nominal, likely about $10 a year, depending on the number of members. Fees would be subject to change on the basis of expenses. The AMA has a sliding fee schedule that tops out with annual dues for physicians in regular practice of $420.

“There are no delegates, no representatives, and no board of directors. We want every physician to join and every physician to vote on every issue,” Dr. Teirstein said.

He stressed that he sees United Physicians as being complementary to the AMA.

“We do not compete with other organizations. Ideally, other organizations will use the platform,” Dr. Teirstein said. “If the AMA is considering a new policy, it can use the United Physicians platform to measure physician support. For example, through online discussions, petitions, and voting, it might learn a proposed policy needs a few tweaks to be accepted by most physicians.”

No compensation

Dr. Teirstein is among physician leaders who in recent years have sought to rally their colleagues to fight back against growing administrative burdens.

In a 2015 article in JAMA that was written with Medscape’s editor in chief, Eric Topol, MD, Dr. Teirstein criticized the ABIM’s drive to have physicians complete tests every 2 years and participate in continuous certification instead of recertifying once a decade, as had been the practice.

Dr. Teirstein formed the NBPAS as an alternative path for certification, with Dr. Topol serving on the board for that organization. Dr. Topol also will serve as a member of the advisory board for Teirstein’s United Physicians.

Dr. Topol wrote an article that appeared in the New Yorker last August that argued for physicians to move beyond the confines of medical societies and seek a path for broad-based activism. He said he intended to challenge medical societies, which, for all the good they do, can sometimes lose focus on that core relationship in favor of the bottom line.

Dr. Topol said in an interview that his colleague’s new project is a “good idea for a democratized platform at a time when physician solidarity is needed more than ever.”

Dr. Teirstein plans to run United Physicians on a volunteer basis. This builds on the approach he has used for NBPAS. He and the directors of the NBPAS will receive no compensation, he said, as was confirmed by the NBPAS.

In contrast, Dr. Madara made about $2.5 million in total compensation for 2018, according to the organization’s Internal Revenue Service filing. Physicians who served as trustees and officials for the AMA that year received annual compensation that ranged from around $60,000 to $291,980, depending on their duties.

“Having volunteer leadership mitigates conflict of interest. It also ensures leadership has a ‘day job’ that keeps them in touch with issues impacting practicing physicians,” Dr. Teirstein said

Start-up costs for United Physicians will be supported by NBPAS, but it will function as a completely independent organization, he added.

In introducing the group, Dr. Teirstein outlined suggestions for proposals it might pursue. These include making hospitals secure adequate supplies of personal protective equipment ahead of health crises such as the COVID-19 pandemic.

His outline also includes suggestions for issues that likely will persist beyond the response to the pandemic.

Dr. Teirstein proposed a project for persuading insurance companies to provide online calendar appointments for peer-to-peer patient preauthorization. Failure of the insurer’s representative to attend would trigger approval of authorization under this proposal. He also suggested a lobbying effort for specific reimbursement for peer-to-peer, patient preauthorization phone calls.

Dr. Teirstein said he hopes most of the proposals will come from physicians who join United Physicians. Still, it is unclear whether United Physicians will succeed. An initial challenge could be in sorting through a barrage of competing ideas submitted to United Physicians.

But Dr. Teirstein appears hopeful about the changes for this experiment in online advocacy. He intends for United Physicians to be a pathway for clinicians to translate their complaints about policies into calls for action, with only a short investment of their time.

“Most of us have wonderful, engrossing jobs. It’s hard to beat helping a patient, and most of us get to do it every day,” Dr. .Teirstein said. “Will we take the 30 seconds required to sign up and become a United Physicians member? Will we spend a little time each week reviewing the issues and voting? I think it’s an experiment worth watching.”

A version of this article originally appeared on Medscape.com.

The Centers for Medicare & Medicaid Services will suspend its Medicare advance payment program for clinicians and is reevaluating how much to pay to hospitals going forward through particular COVID-19 relief initiatives. CMS announced the changes on April 26. Physicians and others who use the accelerated and advance Medicare payments program repay these advances, and they are typically given 1 year or less to repay the funding.

CMS said in a news release it will not accept new applications for the advanced Medicare payment, and it will be reevaluating all pending and new applications “in light of historical direct payments made available through the Department of Health & Human Services’ (HHS) Provider Relief Fund.”

The advance Medicare payment program predates COVID-19, although it previously was used on a much smaller scale. In the past 5 years, CMS approved about 100 total requests for advanced Medicare payment, with most being tied to natural disasters such as hurricanes.

CMS said it has approved, since March, more than 21,000 applications for advanced Medicare payment, totaling $59.6 billion, for hospitals and other organizations that bill its Part A program. In addition, CMS approved almost 24,000 applications for its Part B program, advancing $40.4 billion for physicians, other clinicians, and medical equipment suppliers.

CMS noted that Congress also has provided $175 billion in aid for the medical community that clinicians and medical organizations would not need to repay. The Coronavirus Aid, Relief, and Economic Security (CARES) Act enacted in March included $100 billion, and the Paycheck Protection Program and Health Care Enhancement Act, enacted March 24, includes another $75 billion.

A version of this article was originally published on Medscape.com.

The Centers for Medicare & Medicaid Services will suspend its Medicare advance payment program for clinicians and is reevaluating how much to pay to hospitals going forward through particular COVID-19 relief initiatives. CMS announced the changes on April 26. Physicians and others who use the accelerated and advance Medicare payments program repay these advances, and they are typically given 1 year or less to repay the funding.

CMS said in a news release it will not accept new applications for the advanced Medicare payment, and it will be reevaluating all pending and new applications “in light of historical direct payments made available through the Department of Health & Human Services’ (HHS) Provider Relief Fund.”

The advance Medicare payment program predates COVID-19, although it previously was used on a much smaller scale. In the past 5 years, CMS approved about 100 total requests for advanced Medicare payment, with most being tied to natural disasters such as hurricanes.

CMS said it has approved, since March, more than 21,000 applications for advanced Medicare payment, totaling $59.6 billion, for hospitals and other organizations that bill its Part A program. In addition, CMS approved almost 24,000 applications for its Part B program, advancing $40.4 billion for physicians, other clinicians, and medical equipment suppliers.

CMS noted that Congress also has provided $175 billion in aid for the medical community that clinicians and medical organizations would not need to repay. The Coronavirus Aid, Relief, and Economic Security (CARES) Act enacted in March included $100 billion, and the Paycheck Protection Program and Health Care Enhancement Act, enacted March 24, includes another $75 billion.

A version of this article was originally published on Medscape.com.

The Centers for Medicare & Medicaid Services will suspend its Medicare advance payment program for clinicians and is reevaluating how much to pay to hospitals going forward through particular COVID-19 relief initiatives. CMS announced the changes on April 26. Physicians and others who use the accelerated and advance Medicare payments program repay these advances, and they are typically given 1 year or less to repay the funding.

CMS said in a news release it will not accept new applications for the advanced Medicare payment, and it will be reevaluating all pending and new applications “in light of historical direct payments made available through the Department of Health & Human Services’ (HHS) Provider Relief Fund.”

The advance Medicare payment program predates COVID-19, although it previously was used on a much smaller scale. In the past 5 years, CMS approved about 100 total requests for advanced Medicare payment, with most being tied to natural disasters such as hurricanes.

CMS said it has approved, since March, more than 21,000 applications for advanced Medicare payment, totaling $59.6 billion, for hospitals and other organizations that bill its Part A program. In addition, CMS approved almost 24,000 applications for its Part B program, advancing $40.4 billion for physicians, other clinicians, and medical equipment suppliers.

CMS noted that Congress also has provided $175 billion in aid for the medical community that clinicians and medical organizations would not need to repay. The Coronavirus Aid, Relief, and Economic Security (CARES) Act enacted in March included $100 billion, and the Paycheck Protection Program and Health Care Enhancement Act, enacted March 24, includes another $75 billion.

A version of this article was originally published on Medscape.com.

The Trump administration on Feb. 10 argued for cutting spending for a federal agency at the forefront of the efforts to combat the coronavirus, while also seeking to slow spending in certain parts of the Medicare and Medicaid programs.

President Donald Trump presented his fiscal 2021 request to Congress for refilling the coffers of federal agencies. In any administration, an annual budget serves only as a political blueprint, as the White House document itself makes no changes in federal spending.

In Mr. Trump’s case, several of his requests for agencies within the Department of Health & Human Services run counter to recent budget trends. Republicans and Democrats in Congress have worked together in recent years to increase budgets for major federal health agencies.

But Mr. Trump asked Congress to cut annual budget authority for the National Institute of Allergy and Infectious Diseases by $430 million to $5.446 billion for fiscal 2021. In contrast, Congress has raised the annual budget for NIAID, a key agency in combating the coronavirus, from $5.545 billion in fiscal 2019 to $5.876 billion in fiscal 2020, which began in October, according to an HHS summary of Mr. Trump’s request.

For the Centers for Disease Control and Prevention, which is central to the battle against the coronavirus, Mr. Trump proposed a drop in discretionary funding to $5.627 billion. In contrast, Congress raised the CDC budget from $6.544 billion in fiscal 2019 to $6.917 in fiscal 2020.

Mr. Trump also wants to cut $559 million from the budget of the National Cancer Institute, dropping it to $5.881 billion in fiscal 2021. In contrast, Congress raised NCI’s budget from $6.121 billion in fiscal 2019 to $6.440 billion in fiscal 2020.

Mr. Trump requested a $2.6 billion reduction in the National Institutes of Health’s total discretionary budget, seeking to drop it to $37.70 billion. In contrast, Congress raised NIH’s budget from $37.887 in fiscal 2019 to $40.304 billion in fiscal 2020.

Mr. Trump’s budget proposal also includes an estimate of $152 billion in savings over a decade for Medicaid through the implementation of what the administration calls “community engagement” requirements.

The Trump administration has been at odds with Democrats for years about whether work requirements should be attached to Medicaid. “Well-designed community engagement incentives have great potential to improve health and well-being while empowering beneficiaries to rise out of poverty,” HHS said in a budget document.

Yet researchers last year reported that Arkansas’ attempt to attach work requirements to Medicaid caused almost 17,000 adults to lose this health care coverage within the first 6 months, and there was no significant difference in employment.

The researchers say this loss of coverage was partly a result of bureaucratic obstacles and confusion about the new rules. In June 2018, Arkansas became the first state to implement work requirements for Medicaid, Benjamin D. Sommers, MD, PhD, of the Harvard T.H. Chan School of Public Health, Boston, and colleagues wrote in the New England Journal of Medicine (2019 Sep 12;381[11]:1073-82). 

Budget ‘would thwart’ progress

A few medical groups on Monday quickly criticized Mr. Trump’s proposals.

“In a time where our nation continues to face significant public health challenges — including 2019 novel coronavirus, climate change, gun violence, and costly chronic diseases such as heart disease and cancer – the administration should be investing more resources in better health, not cutting federal health budgets,” said Georges C. Benjamin, MD, executive director of the American Public Health Association, in a statement.

David J. Skorton, MD, chief executive and president of the Association of American Medical Colleges (AAMC) also urged increased investment in fighting disease.

“We must continue the bipartisan budget trajectory set forth by Congress over the last several years, not reverse course,” Dr. Skorton said in a statement.

Mr. Trump’s proposed cuts in medical research “would thwart scientific progress on strategies to prevent, diagnose, treat, and cure medical conditions that affect countless patients nationwide,” he said.

In total, the new 2021 appropriations for HHS would fall by $9.46 billion to $85.667 billion under Mr. Trump’s proposal. Appropriations, also called discretionary budget authority, represents the operating budgets for federal agencies. These are decided through annual spending bills.

Congress has separate sets of laws for handling payments the federal government makes through Medicare and Medicaid. These are known as mandatory spending.

‘Untenable cuts’

AAMC’s Dr. Skorton also objected to what he termed Mr. Trump’s bid “to reduce and consolidate Medicare, Medicaid, and children’s hospital graduate medical education into a single grant program.”

This would force teaching hospitals to absorb $52 billion in “untenable cuts,” he said.

“The proposal ignores the intent of the Medicare GME program, which is to ensure an adequate physician workforce to care for Medicare beneficiaries and support the critical patient care missions of America’s teaching hospitals,” Dr. Skorton said.

The budget also seeks cuts to Medicaid, which come in addition to the administration’s “recent proposals to scale back Medicaid coverage,” Dr. Skorton said.

“More than 26% of all Medicaid hospitalizations occur at AAMC-member teaching hospitals, even though these institutions represent only 5% of all hospitals,” Dr. Skorton said. “Each of the administration’s proposals on their own would be devastating for patients – and combined, they would be disastrous.”

Rick Pollack, the chief executive and president of the American Hospital Association, described Mr. Trump’s fiscal 2021 proposal as another bid to undermine medical care in the United States.

“Every year, we adapt to a constantly changing environment, but every year, the administration aims to gut our nation’s health care infrastructure,” Mr. Pollack said in a statement.

In it, he noted that about one in five people in America depend on Medicaid, with children accounting for a large proportion of those covered by the state-federal program.

“The budget’s proposal on Medicaid financing and service delivery would cut hundreds of billions of dollars from the Medicaid program annually,” Mr. Pollack said.

He also objected to “hundreds of billions of proposed reductions to Medicare” endorsed by Mr. Trump.

Medical malpractice overhaul

The Trump administration also offered many suggestions for changing federal laws to reduce health care spending. Among these was a proposed overhaul of the approach to medical malpractice cases.

The president’s budget proposal estimates $40 billion in savings over a decade from steps to limit medical liability, according to a report from the Office of Management and Budget (OMB).

“The current medical liability system does not work for patients or providers, nor does it promote high-quality, evidence-based care,” OMB said. “Providers practice with a threat of potentially frivolous lawsuits, and injured patients often do not receive just compensation for their injuries.”

Mr. Trump’s fiscal 2021 budget calls for a cap on noneconomic damage awards of $250,000, which would increase with inflation over time, and a 3-year statute of limitations. Under this plan, courts could also modify attorney’s fee arrangements. HHS could provide guidance to states on how to create expert panels and administrative health care tribunals to review medical liability.

These steps would lead to lower health care spending, with clinicians dropping “defensive medicine practices,” OMB said. That would benefit the Medicare and Medicaid programs as well as lowering costs of health insurance in general.

Mr. Trump’s fiscal 2021 budget also includes a series of proposals for Medicare that it estimates would, in aggregate, save $755.5 billion over a decade.

Site-neutral policy

A large chunk of the estimated Medicare savings in Mr. Trump’s fiscal 2021 health budget would come from lowering payments to hospitals for services provided in their outpatient and physician offices.

In the fiscal 2021 proposal, HHS noted that “Medicare generally pays on-campus hospital outpatient departments substantially more than physician offices for the same services.”

Mr. Trump’s budget proposal seeks a more expansive shift to what’s called a “site-neutral” payment for services delivered in hospital outpatient programs or physician offices. This would bring these payments more in line with those made to independent physician practices.

“This proposal would eliminate the often significant disparity between what Medicare pays in these different settings for the same services,” HHS said in the budget summary.

HHS estimated this change in policy would generate $117.2 billion in savings over a decade. Combined with saving from medical malpractice reforms, the Trump administration estimates these two moves combined could save about $164 billion over a decade.

The site-neutral policy has been a legal battleground, with hospital and physician groups winning a round last year. 

Another Medicare proposal included in Mr. Trump’s fiscal 2021 budget homes in on this issue for cases where a hospital owns a physician office. Medicare now pays most off-campus hospital outpatient departments higher rates than the program’s physician fee schedule dictates for the same services.

Switching to a site-neutral policy for these hospital-owned physician offices would result in $47.2 billion in savings over a decade, HHS said in the budget document.
 

This article first appeared on Medscape.com.

The Trump administration on Feb. 10 argued for cutting spending for a federal agency at the forefront of the efforts to combat the coronavirus, while also seeking to slow spending in certain parts of the Medicare and Medicaid programs.

President Donald Trump presented his fiscal 2021 request to Congress for refilling the coffers of federal agencies. In any administration, an annual budget serves only as a political blueprint, as the White House document itself makes no changes in federal spending.

In Mr. Trump’s case, several of his requests for agencies within the Department of Health & Human Services run counter to recent budget trends. Republicans and Democrats in Congress have worked together in recent years to increase budgets for major federal health agencies.

But Mr. Trump asked Congress to cut annual budget authority for the National Institute of Allergy and Infectious Diseases by $430 million to $5.446 billion for fiscal 2021. In contrast, Congress has raised the annual budget for NIAID, a key agency in combating the coronavirus, from $5.545 billion in fiscal 2019 to $5.876 billion in fiscal 2020, which began in October, according to an HHS summary of Mr. Trump’s request.

For the Centers for Disease Control and Prevention, which is central to the battle against the coronavirus, Mr. Trump proposed a drop in discretionary funding to $5.627 billion. In contrast, Congress raised the CDC budget from $6.544 billion in fiscal 2019 to $6.917 in fiscal 2020.

Mr. Trump also wants to cut $559 million from the budget of the National Cancer Institute, dropping it to $5.881 billion in fiscal 2021. In contrast, Congress raised NCI’s budget from $6.121 billion in fiscal 2019 to $6.440 billion in fiscal 2020.

Mr. Trump requested a $2.6 billion reduction in the National Institutes of Health’s total discretionary budget, seeking to drop it to $37.70 billion. In contrast, Congress raised NIH’s budget from $37.887 in fiscal 2019 to $40.304 billion in fiscal 2020.

Mr. Trump’s budget proposal also includes an estimate of $152 billion in savings over a decade for Medicaid through the implementation of what the administration calls “community engagement” requirements.

The Trump administration has been at odds with Democrats for years about whether work requirements should be attached to Medicaid. “Well-designed community engagement incentives have great potential to improve health and well-being while empowering beneficiaries to rise out of poverty,” HHS said in a budget document.

Yet researchers last year reported that Arkansas’ attempt to attach work requirements to Medicaid caused almost 17,000 adults to lose this health care coverage within the first 6 months, and there was no significant difference in employment.

The researchers say this loss of coverage was partly a result of bureaucratic obstacles and confusion about the new rules. In June 2018, Arkansas became the first state to implement work requirements for Medicaid, Benjamin D. Sommers, MD, PhD, of the Harvard T.H. Chan School of Public Health, Boston, and colleagues wrote in the New England Journal of Medicine (2019 Sep 12;381[11]:1073-82). 

Budget ‘would thwart’ progress

A few medical groups on Monday quickly criticized Mr. Trump’s proposals.

“In a time where our nation continues to face significant public health challenges — including 2019 novel coronavirus, climate change, gun violence, and costly chronic diseases such as heart disease and cancer – the administration should be investing more resources in better health, not cutting federal health budgets,” said Georges C. Benjamin, MD, executive director of the American Public Health Association, in a statement.

David J. Skorton, MD, chief executive and president of the Association of American Medical Colleges (AAMC) also urged increased investment in fighting disease.

“We must continue the bipartisan budget trajectory set forth by Congress over the last several years, not reverse course,” Dr. Skorton said in a statement.

Mr. Trump’s proposed cuts in medical research “would thwart scientific progress on strategies to prevent, diagnose, treat, and cure medical conditions that affect countless patients nationwide,” he said.

In total, the new 2021 appropriations for HHS would fall by $9.46 billion to $85.667 billion under Mr. Trump’s proposal. Appropriations, also called discretionary budget authority, represents the operating budgets for federal agencies. These are decided through annual spending bills.

Congress has separate sets of laws for handling payments the federal government makes through Medicare and Medicaid. These are known as mandatory spending.

‘Untenable cuts’

AAMC’s Dr. Skorton also objected to what he termed Mr. Trump’s bid “to reduce and consolidate Medicare, Medicaid, and children’s hospital graduate medical education into a single grant program.”

This would force teaching hospitals to absorb $52 billion in “untenable cuts,” he said.

“The proposal ignores the intent of the Medicare GME program, which is to ensure an adequate physician workforce to care for Medicare beneficiaries and support the critical patient care missions of America’s teaching hospitals,” Dr. Skorton said.

The budget also seeks cuts to Medicaid, which come in addition to the administration’s “recent proposals to scale back Medicaid coverage,” Dr. Skorton said.

“More than 26% of all Medicaid hospitalizations occur at AAMC-member teaching hospitals, even though these institutions represent only 5% of all hospitals,” Dr. Skorton said. “Each of the administration’s proposals on their own would be devastating for patients – and combined, they would be disastrous.”

Rick Pollack, the chief executive and president of the American Hospital Association, described Mr. Trump’s fiscal 2021 proposal as another bid to undermine medical care in the United States.

“Every year, we adapt to a constantly changing environment, but every year, the administration aims to gut our nation’s health care infrastructure,” Mr. Pollack said in a statement.

In it, he noted that about one in five people in America depend on Medicaid, with children accounting for a large proportion of those covered by the state-federal program.

“The budget’s proposal on Medicaid financing and service delivery would cut hundreds of billions of dollars from the Medicaid program annually,” Mr. Pollack said.

He also objected to “hundreds of billions of proposed reductions to Medicare” endorsed by Mr. Trump.

Medical malpractice overhaul

The Trump administration also offered many suggestions for changing federal laws to reduce health care spending. Among these was a proposed overhaul of the approach to medical malpractice cases.

The president’s budget proposal estimates $40 billion in savings over a decade from steps to limit medical liability, according to a report from the Office of Management and Budget (OMB).

“The current medical liability system does not work for patients or providers, nor does it promote high-quality, evidence-based care,” OMB said. “Providers practice with a threat of potentially frivolous lawsuits, and injured patients often do not receive just compensation for their injuries.”

Mr. Trump’s fiscal 2021 budget calls for a cap on noneconomic damage awards of $250,000, which would increase with inflation over time, and a 3-year statute of limitations. Under this plan, courts could also modify attorney’s fee arrangements. HHS could provide guidance to states on how to create expert panels and administrative health care tribunals to review medical liability.

These steps would lead to lower health care spending, with clinicians dropping “defensive medicine practices,” OMB said. That would benefit the Medicare and Medicaid programs as well as lowering costs of health insurance in general.

Mr. Trump’s fiscal 2021 budget also includes a series of proposals for Medicare that it estimates would, in aggregate, save $755.5 billion over a decade.

Site-neutral policy

A large chunk of the estimated Medicare savings in Mr. Trump’s fiscal 2021 health budget would come from lowering payments to hospitals for services provided in their outpatient and physician offices.

In the fiscal 2021 proposal, HHS noted that “Medicare generally pays on-campus hospital outpatient departments substantially more than physician offices for the same services.”

Mr. Trump’s budget proposal seeks a more expansive shift to what’s called a “site-neutral” payment for services delivered in hospital outpatient programs or physician offices. This would bring these payments more in line with those made to independent physician practices.

“This proposal would eliminate the often significant disparity between what Medicare pays in these different settings for the same services,” HHS said in the budget summary.

HHS estimated this change in policy would generate $117.2 billion in savings over a decade. Combined with saving from medical malpractice reforms, the Trump administration estimates these two moves combined could save about $164 billion over a decade.

The site-neutral policy has been a legal battleground, with hospital and physician groups winning a round last year. 

Another Medicare proposal included in Mr. Trump’s fiscal 2021 budget homes in on this issue for cases where a hospital owns a physician office. Medicare now pays most off-campus hospital outpatient departments higher rates than the program’s physician fee schedule dictates for the same services.

Switching to a site-neutral policy for these hospital-owned physician offices would result in $47.2 billion in savings over a decade, HHS said in the budget document.
 

This article first appeared on Medscape.com.

The Trump administration on Feb. 10 argued for cutting spending for a federal agency at the forefront of the efforts to combat the coronavirus, while also seeking to slow spending in certain parts of the Medicare and Medicaid programs.

President Donald Trump presented his fiscal 2021 request to Congress for refilling the coffers of federal agencies. In any administration, an annual budget serves only as a political blueprint, as the White House document itself makes no changes in federal spending.

In Mr. Trump’s case, several of his requests for agencies within the Department of Health & Human Services run counter to recent budget trends. Republicans and Democrats in Congress have worked together in recent years to increase budgets for major federal health agencies.

But Mr. Trump asked Congress to cut annual budget authority for the National Institute of Allergy and Infectious Diseases by $430 million to $5.446 billion for fiscal 2021. In contrast, Congress has raised the annual budget for NIAID, a key agency in combating the coronavirus, from $5.545 billion in fiscal 2019 to $5.876 billion in fiscal 2020, which began in October, according to an HHS summary of Mr. Trump’s request.

For the Centers for Disease Control and Prevention, which is central to the battle against the coronavirus, Mr. Trump proposed a drop in discretionary funding to $5.627 billion. In contrast, Congress raised the CDC budget from $6.544 billion in fiscal 2019 to $6.917 in fiscal 2020.

Mr. Trump also wants to cut $559 million from the budget of the National Cancer Institute, dropping it to $5.881 billion in fiscal 2021. In contrast, Congress raised NCI’s budget from $6.121 billion in fiscal 2019 to $6.440 billion in fiscal 2020.

Mr. Trump requested a $2.6 billion reduction in the National Institutes of Health’s total discretionary budget, seeking to drop it to $37.70 billion. In contrast, Congress raised NIH’s budget from $37.887 in fiscal 2019 to $40.304 billion in fiscal 2020.

Mr. Trump’s budget proposal also includes an estimate of $152 billion in savings over a decade for Medicaid through the implementation of what the administration calls “community engagement” requirements.

The Trump administration has been at odds with Democrats for years about whether work requirements should be attached to Medicaid. “Well-designed community engagement incentives have great potential to improve health and well-being while empowering beneficiaries to rise out of poverty,” HHS said in a budget document.

Yet researchers last year reported that Arkansas’ attempt to attach work requirements to Medicaid caused almost 17,000 adults to lose this health care coverage within the first 6 months, and there was no significant difference in employment.

The researchers say this loss of coverage was partly a result of bureaucratic obstacles and confusion about the new rules. In June 2018, Arkansas became the first state to implement work requirements for Medicaid, Benjamin D. Sommers, MD, PhD, of the Harvard T.H. Chan School of Public Health, Boston, and colleagues wrote in the New England Journal of Medicine (2019 Sep 12;381[11]:1073-82). 

Budget ‘would thwart’ progress

A few medical groups on Monday quickly criticized Mr. Trump’s proposals.

“In a time where our nation continues to face significant public health challenges — including 2019 novel coronavirus, climate change, gun violence, and costly chronic diseases such as heart disease and cancer – the administration should be investing more resources in better health, not cutting federal health budgets,” said Georges C. Benjamin, MD, executive director of the American Public Health Association, in a statement.

David J. Skorton, MD, chief executive and president of the Association of American Medical Colleges (AAMC) also urged increased investment in fighting disease.

“We must continue the bipartisan budget trajectory set forth by Congress over the last several years, not reverse course,” Dr. Skorton said in a statement.

Mr. Trump’s proposed cuts in medical research “would thwart scientific progress on strategies to prevent, diagnose, treat, and cure medical conditions that affect countless patients nationwide,” he said.

In total, the new 2021 appropriations for HHS would fall by $9.46 billion to $85.667 billion under Mr. Trump’s proposal. Appropriations, also called discretionary budget authority, represents the operating budgets for federal agencies. These are decided through annual spending bills.

Congress has separate sets of laws for handling payments the federal government makes through Medicare and Medicaid. These are known as mandatory spending.

‘Untenable cuts’

AAMC’s Dr. Skorton also objected to what he termed Mr. Trump’s bid “to reduce and consolidate Medicare, Medicaid, and children’s hospital graduate medical education into a single grant program.”

This would force teaching hospitals to absorb $52 billion in “untenable cuts,” he said.

“The proposal ignores the intent of the Medicare GME program, which is to ensure an adequate physician workforce to care for Medicare beneficiaries and support the critical patient care missions of America’s teaching hospitals,” Dr. Skorton said.

The budget also seeks cuts to Medicaid, which come in addition to the administration’s “recent proposals to scale back Medicaid coverage,” Dr. Skorton said.

“More than 26% of all Medicaid hospitalizations occur at AAMC-member teaching hospitals, even though these institutions represent only 5% of all hospitals,” Dr. Skorton said. “Each of the administration’s proposals on their own would be devastating for patients – and combined, they would be disastrous.”

Rick Pollack, the chief executive and president of the American Hospital Association, described Mr. Trump’s fiscal 2021 proposal as another bid to undermine medical care in the United States.

“Every year, we adapt to a constantly changing environment, but every year, the administration aims to gut our nation’s health care infrastructure,” Mr. Pollack said in a statement.

In it, he noted that about one in five people in America depend on Medicaid, with children accounting for a large proportion of those covered by the state-federal program.

“The budget’s proposal on Medicaid financing and service delivery would cut hundreds of billions of dollars from the Medicaid program annually,” Mr. Pollack said.

He also objected to “hundreds of billions of proposed reductions to Medicare” endorsed by Mr. Trump.

Medical malpractice overhaul

The Trump administration also offered many suggestions for changing federal laws to reduce health care spending. Among these was a proposed overhaul of the approach to medical malpractice cases.

The president’s budget proposal estimates $40 billion in savings over a decade from steps to limit medical liability, according to a report from the Office of Management and Budget (OMB).

“The current medical liability system does not work for patients or providers, nor does it promote high-quality, evidence-based care,” OMB said. “Providers practice with a threat of potentially frivolous lawsuits, and injured patients often do not receive just compensation for their injuries.”

Mr. Trump’s fiscal 2021 budget calls for a cap on noneconomic damage awards of $250,000, which would increase with inflation over time, and a 3-year statute of limitations. Under this plan, courts could also modify attorney’s fee arrangements. HHS could provide guidance to states on how to create expert panels and administrative health care tribunals to review medical liability.

These steps would lead to lower health care spending, with clinicians dropping “defensive medicine practices,” OMB said. That would benefit the Medicare and Medicaid programs as well as lowering costs of health insurance in general.

Mr. Trump’s fiscal 2021 budget also includes a series of proposals for Medicare that it estimates would, in aggregate, save $755.5 billion over a decade.

Site-neutral policy

A large chunk of the estimated Medicare savings in Mr. Trump’s fiscal 2021 health budget would come from lowering payments to hospitals for services provided in their outpatient and physician offices.

In the fiscal 2021 proposal, HHS noted that “Medicare generally pays on-campus hospital outpatient departments substantially more than physician offices for the same services.”

Mr. Trump’s budget proposal seeks a more expansive shift to what’s called a “site-neutral” payment for services delivered in hospital outpatient programs or physician offices. This would bring these payments more in line with those made to independent physician practices.

“This proposal would eliminate the often significant disparity between what Medicare pays in these different settings for the same services,” HHS said in the budget summary.

HHS estimated this change in policy would generate $117.2 billion in savings over a decade. Combined with saving from medical malpractice reforms, the Trump administration estimates these two moves combined could save about $164 billion over a decade.

The site-neutral policy has been a legal battleground, with hospital and physician groups winning a round last year. 

Another Medicare proposal included in Mr. Trump’s fiscal 2021 budget homes in on this issue for cases where a hospital owns a physician office. Medicare now pays most off-campus hospital outpatient departments higher rates than the program’s physician fee schedule dictates for the same services.

Switching to a site-neutral policy for these hospital-owned physician offices would result in $47.2 billion in savings over a decade, HHS said in the budget document.
 

This article first appeared on Medscape.com.

A newly approved triple-combination modulator to treat cystic fibrosis (CF) has raised expectations of a treatment turning point among patients and specialists. If the early results are sustained, elexacaftor/ivacaftor/tezacaftor (Trikafta) could prove to be the rare case of a much-touted new medicine that meets high expectations.

Courtesy Susan Millard, MD

“CF even in infants causes inflammation, so we know that lung damage can start early and progress,” said Susan Millard, MD, FCCP, of Helen DeVos Children’s Hospital in Grand Rapids, Mich., and the local clinical research director for the pediatric pulmonary and sleep medicine section. “This oral drug therapy is actually treating the underlying problem, as opposed to many of the therapies we have that take hours to nebulize and only work locally in the airways.”

Dr. Millard is the recent past pediatric editor for Chest Physician and has been a local principal investigator at Helen DeVos Children’s Hospital for many Vertex-sponsored clinical studies.

The pivotal studies

The Food and Drug Administration approval of Trikafta rested on two pivotal phase 3, placebo-controlled studies, one in patients with two copies of the most common CF mutations, F508del, and the second in patients with one copy of F508del and a second mutation that was called a “minimal-function” mutation. The findings have ignited the hopes of many people with CF and their physicians. The drug was approved in October 2019 for patients aged 12 years and older who have at least one F508del mutation of the cystic fibrosis transmembrane conductance regulator gene. About 90% of patients in the United States have at least one copy of F508del. In the study looking at patients with one copy of F508del, the mean predicted forced expiratory volume in 1 second increased 13.8% in patients taking the drug versus placebo (N Engl J Med. 2019 Oct 31. doi: 10.1056/NEJMoa1908639). The number of pulmonary exacerbations decreased by 63% in the Trikafta group, compared with placebo. Pulmonary exacerbations were described as a change in specific symptoms that required treatment with a new oral, intravenous, or inhaled antibiotic. Serious adverse drug reactions that occurred more frequently in patients receiving Trikafta, compared with placebo, were rash and influenza events.

In the study that included patients with two copies of F508del, on average, the lung function increased 10% versus patients on ivacaftor/tezacaftor at 4 weeks. In addition, there was a 45.1 mmol/L on average decrease in the sweat chloride level in the Trikafta group, compared with ivacaftor/tezacaftor.
 

A hopeful start

Robert Giusti, MD, a pediatric pulmonologist at New York University Langone Health, is also hopeful. “This could be the kind of treatment that will make a revolution in terms of [cystic fibrosis] care if it can be started very early in life shortly after diagnosis. We anticipate that patients will be disease free for a longer period of time.”

The Cystic Fibrosis Foundation’s (CFF) “venture philanthropy” initiative played an important role in the development of the drug by Vertex Pharmaceuticals. The CFF has invested many millions of dollars in research by drug companies since the 1980s and was an early backer of Vertex. According to a statement on the CFF website, the Foundation sold its royalty rights for treatments developed by Vertex for $3.3 billion in 2014. The drug has a list price of about $311,000 a year. Payment issues may arise in the future, but for now, Vertex has stated that insurers and some Medicaid programs have begun paying claims for Trikafta

Specialists who treat CF now are watching to see how well patients tolerate this highly anticipated drug – and how well it meets expectations. The Therapeutic Development Network, the clinical research division of the CFF, is enrolling patients taking Trikafta in an observational study to follow for long-term follow-up.
 

Meeting expectations

“[Long-term efficacy is] something that we’re always concerned about. When the drug comes to market, is it going to be as effective as we thought it might be?” said Ryan Thomas, MD, director of the Cystic Fibrosis Center at Michigan State University, East Lansing. The MSU Cystic Fibrosis Center receives funding from the Cystic Fibrosis Foundation.

A different disease

Trikafta is the latest in a series of improvements of CF treatment in recent decades, recalled Dr. Giusti, who has been in this field for about 3 decades. “It used to be that I attended many funerals for children with CF. Now with patients living longer and healthier lives I am invited to attend their weddings and even their children’s baptisms and bris ceremonies. It is a very different disease than it used to be.”

The promise of Trikafta leaves behind the minority of patients for whom the drug won’t work. This is for the 10% of patients that have rare mutations. That can lead to difficult conversations with parents about why this new option is not a choice for their child, Dr. Millard said. “It just crushes you, but the Cystic Fibrosis Foundation is committing a lot of new research in that direction. Their mantra is ‘until it is done.’ ”
 

Realistic expectations

William (Randy) Hunt, MD, FAAP, FACP, assistant professor of medicine in the Division of Pulmonary, Allergy, Critical Care and Sleep, Emory University School of Medicine, Atlanta, agrees that Trikafta is an exciting development in CF treatment. He noted,  “Starting this medication early in life may very well significantly attenuate the disease, but it is not a cure. For individuals who already have significant disease, we may not see the same level of improvements in lung function as what we saw in the studies. The studies generally excluded individuals with ppFEV1 < 40%.  Nevertheless, I remain optimistic and have been prescribing it to nearly everyone that qualifies after a discussion.” 

Dr. Hunt added, “Patients are asking if they can stop their current chronic CF therapies once they start Trikafta. The answer is “no, at least not right now.” While all the relatively short-term data around Trikafta are very promising, we do not yet know how sustained the long-term benefits will be.  Still, safely removing therapeutic burden from our patient population is a real interest. There are plans underway by the CFF and other institutions to systematically research whether discontinuing chronic CF therapies is safe in the setting of Trikafta.”

He concluded that 10% of individuals with CF mutations still do not respond to the modulators currently available. “We will not leave that population behind, but treating these remaining mutations is going to take continued efforts and likely modulators that are therapeutically differently from the mechanism of actions of those that are currently available,” he said.
 

Therese Borden contributed to this article.

1/2/2020 - This story was updated.

A newly approved triple-combination modulator to treat cystic fibrosis (CF) has raised expectations of a treatment turning point among patients and specialists. If the early results are sustained, elexacaftor/ivacaftor/tezacaftor (Trikafta) could prove to be the rare case of a much-touted new medicine that meets high expectations.

Courtesy Susan Millard, MD

“CF even in infants causes inflammation, so we know that lung damage can start early and progress,” said Susan Millard, MD, FCCP, of Helen DeVos Children’s Hospital in Grand Rapids, Mich., and the local clinical research director for the pediatric pulmonary and sleep medicine section. “This oral drug therapy is actually treating the underlying problem, as opposed to many of the therapies we have that take hours to nebulize and only work locally in the airways.”

Dr. Millard is the recent past pediatric editor for Chest Physician and has been a local principal investigator at Helen DeVos Children’s Hospital for many Vertex-sponsored clinical studies.

The pivotal studies

The Food and Drug Administration approval of Trikafta rested on two pivotal phase 3, placebo-controlled studies, one in patients with two copies of the most common CF mutations, F508del, and the second in patients with one copy of F508del and a second mutation that was called a “minimal-function” mutation. The findings have ignited the hopes of many people with CF and their physicians. The drug was approved in October 2019 for patients aged 12 years and older who have at least one F508del mutation of the cystic fibrosis transmembrane conductance regulator gene. About 90% of patients in the United States have at least one copy of F508del. In the study looking at patients with one copy of F508del, the mean predicted forced expiratory volume in 1 second increased 13.8% in patients taking the drug versus placebo (N Engl J Med. 2019 Oct 31. doi: 10.1056/NEJMoa1908639). The number of pulmonary exacerbations decreased by 63% in the Trikafta group, compared with placebo. Pulmonary exacerbations were described as a change in specific symptoms that required treatment with a new oral, intravenous, or inhaled antibiotic. Serious adverse drug reactions that occurred more frequently in patients receiving Trikafta, compared with placebo, were rash and influenza events.

In the study that included patients with two copies of F508del, on average, the lung function increased 10% versus patients on ivacaftor/tezacaftor at 4 weeks. In addition, there was a 45.1 mmol/L on average decrease in the sweat chloride level in the Trikafta group, compared with ivacaftor/tezacaftor.
 

A hopeful start

Robert Giusti, MD, a pediatric pulmonologist at New York University Langone Health, is also hopeful. “This could be the kind of treatment that will make a revolution in terms of [cystic fibrosis] care if it can be started very early in life shortly after diagnosis. We anticipate that patients will be disease free for a longer period of time.”

The Cystic Fibrosis Foundation’s (CFF) “venture philanthropy” initiative played an important role in the development of the drug by Vertex Pharmaceuticals. The CFF has invested many millions of dollars in research by drug companies since the 1980s and was an early backer of Vertex. According to a statement on the CFF website, the Foundation sold its royalty rights for treatments developed by Vertex for $3.3 billion in 2014. The drug has a list price of about $311,000 a year. Payment issues may arise in the future, but for now, Vertex has stated that insurers and some Medicaid programs have begun paying claims for Trikafta

Specialists who treat CF now are watching to see how well patients tolerate this highly anticipated drug – and how well it meets expectations. The Therapeutic Development Network, the clinical research division of the CFF, is enrolling patients taking Trikafta in an observational study to follow for long-term follow-up.
 

Meeting expectations

“[Long-term efficacy is] something that we’re always concerned about. When the drug comes to market, is it going to be as effective as we thought it might be?” said Ryan Thomas, MD, director of the Cystic Fibrosis Center at Michigan State University, East Lansing. The MSU Cystic Fibrosis Center receives funding from the Cystic Fibrosis Foundation.

A different disease

Trikafta is the latest in a series of improvements of CF treatment in recent decades, recalled Dr. Giusti, who has been in this field for about 3 decades. “It used to be that I attended many funerals for children with CF. Now with patients living longer and healthier lives I am invited to attend their weddings and even their children’s baptisms and bris ceremonies. It is a very different disease than it used to be.”

The promise of Trikafta leaves behind the minority of patients for whom the drug won’t work. This is for the 10% of patients that have rare mutations. That can lead to difficult conversations with parents about why this new option is not a choice for their child, Dr. Millard said. “It just crushes you, but the Cystic Fibrosis Foundation is committing a lot of new research in that direction. Their mantra is ‘until it is done.’ ”
 

Realistic expectations

William (Randy) Hunt, MD, FAAP, FACP, assistant professor of medicine in the Division of Pulmonary, Allergy, Critical Care and Sleep, Emory University School of Medicine, Atlanta, agrees that Trikafta is an exciting development in CF treatment. He noted,  “Starting this medication early in life may very well significantly attenuate the disease, but it is not a cure. For individuals who already have significant disease, we may not see the same level of improvements in lung function as what we saw in the studies. The studies generally excluded individuals with ppFEV1 < 40%.  Nevertheless, I remain optimistic and have been prescribing it to nearly everyone that qualifies after a discussion.” 

Dr. Hunt added, “Patients are asking if they can stop their current chronic CF therapies once they start Trikafta. The answer is “no, at least not right now.” While all the relatively short-term data around Trikafta are very promising, we do not yet know how sustained the long-term benefits will be.  Still, safely removing therapeutic burden from our patient population is a real interest. There are plans underway by the CFF and other institutions to systematically research whether discontinuing chronic CF therapies is safe in the setting of Trikafta.”

He concluded that 10% of individuals with CF mutations still do not respond to the modulators currently available. “We will not leave that population behind, but treating these remaining mutations is going to take continued efforts and likely modulators that are therapeutically differently from the mechanism of actions of those that are currently available,” he said.
 

Therese Borden contributed to this article.

1/2/2020 - This story was updated.

A newly approved triple-combination modulator to treat cystic fibrosis (CF) has raised expectations of a treatment turning point among patients and specialists. If the early results are sustained, elexacaftor/ivacaftor/tezacaftor (Trikafta) could prove to be the rare case of a much-touted new medicine that meets high expectations.

Courtesy Susan Millard, MD

“CF even in infants causes inflammation, so we know that lung damage can start early and progress,” said Susan Millard, MD, FCCP, of Helen DeVos Children’s Hospital in Grand Rapids, Mich., and the local clinical research director for the pediatric pulmonary and sleep medicine section. “This oral drug therapy is actually treating the underlying problem, as opposed to many of the therapies we have that take hours to nebulize and only work locally in the airways.”

Dr. Millard is the recent past pediatric editor for Chest Physician and has been a local principal investigator at Helen DeVos Children’s Hospital for many Vertex-sponsored clinical studies.

The pivotal studies

The Food and Drug Administration approval of Trikafta rested on two pivotal phase 3, placebo-controlled studies, one in patients with two copies of the most common CF mutations, F508del, and the second in patients with one copy of F508del and a second mutation that was called a “minimal-function” mutation. The findings have ignited the hopes of many people with CF and their physicians. The drug was approved in October 2019 for patients aged 12 years and older who have at least one F508del mutation of the cystic fibrosis transmembrane conductance regulator gene. About 90% of patients in the United States have at least one copy of F508del. In the study looking at patients with one copy of F508del, the mean predicted forced expiratory volume in 1 second increased 13.8% in patients taking the drug versus placebo (N Engl J Med. 2019 Oct 31. doi: 10.1056/NEJMoa1908639). The number of pulmonary exacerbations decreased by 63% in the Trikafta group, compared with placebo. Pulmonary exacerbations were described as a change in specific symptoms that required treatment with a new oral, intravenous, or inhaled antibiotic. Serious adverse drug reactions that occurred more frequently in patients receiving Trikafta, compared with placebo, were rash and influenza events.

In the study that included patients with two copies of F508del, on average, the lung function increased 10% versus patients on ivacaftor/tezacaftor at 4 weeks. In addition, there was a 45.1 mmol/L on average decrease in the sweat chloride level in the Trikafta group, compared with ivacaftor/tezacaftor.
 

A hopeful start

Robert Giusti, MD, a pediatric pulmonologist at New York University Langone Health, is also hopeful. “This could be the kind of treatment that will make a revolution in terms of [cystic fibrosis] care if it can be started very early in life shortly after diagnosis. We anticipate that patients will be disease free for a longer period of time.”

The Cystic Fibrosis Foundation’s (CFF) “venture philanthropy” initiative played an important role in the development of the drug by Vertex Pharmaceuticals. The CFF has invested many millions of dollars in research by drug companies since the 1980s and was an early backer of Vertex. According to a statement on the CFF website, the Foundation sold its royalty rights for treatments developed by Vertex for $3.3 billion in 2014. The drug has a list price of about $311,000 a year. Payment issues may arise in the future, but for now, Vertex has stated that insurers and some Medicaid programs have begun paying claims for Trikafta

Specialists who treat CF now are watching to see how well patients tolerate this highly anticipated drug – and how well it meets expectations. The Therapeutic Development Network, the clinical research division of the CFF, is enrolling patients taking Trikafta in an observational study to follow for long-term follow-up.
 

Meeting expectations

“[Long-term efficacy is] something that we’re always concerned about. When the drug comes to market, is it going to be as effective as we thought it might be?” said Ryan Thomas, MD, director of the Cystic Fibrosis Center at Michigan State University, East Lansing. The MSU Cystic Fibrosis Center receives funding from the Cystic Fibrosis Foundation.

A different disease

Trikafta is the latest in a series of improvements of CF treatment in recent decades, recalled Dr. Giusti, who has been in this field for about 3 decades. “It used to be that I attended many funerals for children with CF. Now with patients living longer and healthier lives I am invited to attend their weddings and even their children’s baptisms and bris ceremonies. It is a very different disease than it used to be.”

The promise of Trikafta leaves behind the minority of patients for whom the drug won’t work. This is for the 10% of patients that have rare mutations. That can lead to difficult conversations with parents about why this new option is not a choice for their child, Dr. Millard said. “It just crushes you, but the Cystic Fibrosis Foundation is committing a lot of new research in that direction. Their mantra is ‘until it is done.’ ”
 

Realistic expectations

William (Randy) Hunt, MD, FAAP, FACP, assistant professor of medicine in the Division of Pulmonary, Allergy, Critical Care and Sleep, Emory University School of Medicine, Atlanta, agrees that Trikafta is an exciting development in CF treatment. He noted,  “Starting this medication early in life may very well significantly attenuate the disease, but it is not a cure. For individuals who already have significant disease, we may not see the same level of improvements in lung function as what we saw in the studies. The studies generally excluded individuals with ppFEV1 < 40%.  Nevertheless, I remain optimistic and have been prescribing it to nearly everyone that qualifies after a discussion.” 

Dr. Hunt added, “Patients are asking if they can stop their current chronic CF therapies once they start Trikafta. The answer is “no, at least not right now.” While all the relatively short-term data around Trikafta are very promising, we do not yet know how sustained the long-term benefits will be.  Still, safely removing therapeutic burden from our patient population is a real interest. There are plans underway by the CFF and other institutions to systematically research whether discontinuing chronic CF therapies is safe in the setting of Trikafta.”

He concluded that 10% of individuals with CF mutations still do not respond to the modulators currently available. “We will not leave that population behind, but treating these remaining mutations is going to take continued efforts and likely modulators that are therapeutically differently from the mechanism of actions of those that are currently available,” he said.
 

Therese Borden contributed to this article.

1/2/2020 - This story was updated.

Allowing people aged 50-64 to buy into Medicare might result in higher premiums for people who purchase their health insurance on the individual market, a finding that runs counter to many people’s expectations, said the authors of a newly released report.

In the report, Christine Eibner, PhD, of the RAND Corporation and colleagues estimated that the premium for so-called bronze market plans might increase by 2%-10%, depending on the design of a Medicare buy-in program. (The bronze plans are ones with fewer benefits sold on exchanges created through the implementation of the Affordable Care Act of 2010 [ACA].)

A perception has been that younger adults have been in effect subsidizing the cost of older ones in the marketplace plans. Instead, it appears that younger adults who enroll in the individual market tend to be relatively unhealthy and thus expensive to cover.

“When older adults leave the market, insurers are left with a smaller pool of younger, less healthy, and relatively expensive people given their age, leading to higher premiums,” Dr. Eibner and colleagues said in the report.

This result was unexpected, as there has been discussion of using a Medicare buy-in to reduce the cost of premiums for others in the marketplace, Dr. Eibner and colleagues said. But this result is consistent with other recent findings, including research presented by the consulting firm Milliman at a Society of Actuaries meeting in June. The Blue Cross Blue Shield Association estimates that losing a large group of customers could raise premiums by about 10% for the remaining pool of insured people, the New York Times has reported.

A buy-in would be less expensive than most ACA plans and would not have the kinds of limits on spending used in private insurance. That might make it attractive to middle-aged Americans. Dr. Eibner and colleagues estimated the annual premium for a Medicare buy-in at $9,747 in 2022. For a 50-year-old, a bronze-level ACA plan would cost $9,208 and a gold-level one, $12,277. For a 60-year-old, the annual premium for a bronze-level plan might be $13,512 and $18,016 for a gold-level plan.

Total out-of-pocket health spending, including premiums, would fall, on average, by 16%-35% for those who moved from ACA-compliant individual market coverage to a buy-in. The lower spending reflects that buy-in enrollees would have access to Medicare payment rates, which are substantially lower than private rates, and lower administrative costs.

There may be growing interest in allowing people aged 50-64 to buy into Medicare if enthusiasm wanes for bids to create a giant national health program, Dr. Eibner said in an interview.

“If there is concern that Medicare for all is going too far, I think this option is something that could become more prominent,” she said.

Many Democratic lawmakers already are focused on a Medicare buy-in approach. Sen. Debbie Stabenow (D-Mich.) has 20 Democratic cosponsors for her bill, which would allow for a Medicare buy-in at age 50. Sen. Bernie Sanders (I-Vt.) has 14 Democratic cosponsors for the current version of his well-known “Medicare-for-all” bill. That’s two fewer than he had for the Medicare-for-all bill he offered in the 115th session of Congress (Jan. 3, 2017–Jan. 3, 2019).

Among the supporters of Sen. Stabenow’s bill are several 2020 presidential contenders: Sen. Cory Booker (D-N.J.), Sen. Amy Klobuchar (D-Minn.), and Sen. Kamala Harris (D-Calif). As of Saturday, Sen. Elizabeth Warren (D-Mass.) backed Sanders’ bill, but not Stabenow’s. But Sen. Warren also has spoken recently of a Medicare expansion for people at age 50 as a step on the path toward universal coverage. Former Vice President Joseph R. Biden and Mayor Pete Buttigieg of South Bend, Ind., have said they would like to offer Americans the option to buy into Medicare or a public plan.

The idea of lowering the Medicare age has been considered for many years by Democrats. It was seen as a way to help older Americans afford medical care before the enactment of the ACA. Before that law took effect, consumers were not guaranteed access to a health plan, causing many older Americans to go without coverage.

But Dr. Eibner and colleagues found that a Medicare buy-in would have little to no effect on total health insurance enrollment. A Medicare buy-in might increase enrollment by 400,000 to 1.6 million for those over age 50, while decreasing enrollment by 100,000 to 800,000 for those under age 50 because of rising premiums.

“It’s not doing a lot to get people covered,” Dr. Eibner said in the interview.

In the report, Dr. Eibner and colleagues estimated that between 2.8 million and 7.0 million people would choose to enroll, depending on the approach used to design a Medicare buy-in. They considered numerous potential options for the design of a Medicare expansion, including various levels of federal subsidy for people using the buy-in. Dr. Eibner and colleagues also considered whether insurers would respond by trying to selectively market to healthier individuals, increasing their chance of enrolling.

The envisioned Medicare buy-in would have no effect on the Medicare Trust Fund, which pools money available through previously collected dedicated taxes, Dr. Eibner and colleagues said. In creating their model, they drew upon data from the Survey of Income and Program Participation, the Medical Expenditure Panel Survey, and the Kaiser Family Foundation and Health Research and Educational Trust Employer Health Benefits Survey.

Dr. Eibner and colleagues noted “several important limitations” for their work. It does not look at how the buy-in might affect clinicians and hospitals. Lower Medicare payment rates might cause some physicians to turn away patients covered by a Medicare buy-in.

“On the other hand, even if some providers decided not to participate in the buy-in, the buy-in might offer enrollees a broader network than what is available on the individual market,” Dr. Eibner and colleagues wrote. “Furthermore, it is not clear that providers would be legally allowed to opt out of the buy-in while still accepting payment for current Medicare beneficiaries.”

The Rand report was developed with the support of funding by the AARP.

SOURCE: Eibner C et al. RAND Corporation. Research Report. 2019. doi: 10.7249/RR4246.

Allowing people aged 50-64 to buy into Medicare might result in higher premiums for people who purchase their health insurance on the individual market, a finding that runs counter to many people’s expectations, said the authors of a newly released report.

In the report, Christine Eibner, PhD, of the RAND Corporation and colleagues estimated that the premium for so-called bronze market plans might increase by 2%-10%, depending on the design of a Medicare buy-in program. (The bronze plans are ones with fewer benefits sold on exchanges created through the implementation of the Affordable Care Act of 2010 [ACA].)

A perception has been that younger adults have been in effect subsidizing the cost of older ones in the marketplace plans. Instead, it appears that younger adults who enroll in the individual market tend to be relatively unhealthy and thus expensive to cover.

“When older adults leave the market, insurers are left with a smaller pool of younger, less healthy, and relatively expensive people given their age, leading to higher premiums,” Dr. Eibner and colleagues said in the report.

This result was unexpected, as there has been discussion of using a Medicare buy-in to reduce the cost of premiums for others in the marketplace, Dr. Eibner and colleagues said. But this result is consistent with other recent findings, including research presented by the consulting firm Milliman at a Society of Actuaries meeting in June. The Blue Cross Blue Shield Association estimates that losing a large group of customers could raise premiums by about 10% for the remaining pool of insured people, the New York Times has reported.

A buy-in would be less expensive than most ACA plans and would not have the kinds of limits on spending used in private insurance. That might make it attractive to middle-aged Americans. Dr. Eibner and colleagues estimated the annual premium for a Medicare buy-in at $9,747 in 2022. For a 50-year-old, a bronze-level ACA plan would cost $9,208 and a gold-level one, $12,277. For a 60-year-old, the annual premium for a bronze-level plan might be $13,512 and $18,016 for a gold-level plan.

Total out-of-pocket health spending, including premiums, would fall, on average, by 16%-35% for those who moved from ACA-compliant individual market coverage to a buy-in. The lower spending reflects that buy-in enrollees would have access to Medicare payment rates, which are substantially lower than private rates, and lower administrative costs.

There may be growing interest in allowing people aged 50-64 to buy into Medicare if enthusiasm wanes for bids to create a giant national health program, Dr. Eibner said in an interview.

“If there is concern that Medicare for all is going too far, I think this option is something that could become more prominent,” she said.

Many Democratic lawmakers already are focused on a Medicare buy-in approach. Sen. Debbie Stabenow (D-Mich.) has 20 Democratic cosponsors for her bill, which would allow for a Medicare buy-in at age 50. Sen. Bernie Sanders (I-Vt.) has 14 Democratic cosponsors for the current version of his well-known “Medicare-for-all” bill. That’s two fewer than he had for the Medicare-for-all bill he offered in the 115th session of Congress (Jan. 3, 2017–Jan. 3, 2019).

Among the supporters of Sen. Stabenow’s bill are several 2020 presidential contenders: Sen. Cory Booker (D-N.J.), Sen. Amy Klobuchar (D-Minn.), and Sen. Kamala Harris (D-Calif). As of Saturday, Sen. Elizabeth Warren (D-Mass.) backed Sanders’ bill, but not Stabenow’s. But Sen. Warren also has spoken recently of a Medicare expansion for people at age 50 as a step on the path toward universal coverage. Former Vice President Joseph R. Biden and Mayor Pete Buttigieg of South Bend, Ind., have said they would like to offer Americans the option to buy into Medicare or a public plan.

The idea of lowering the Medicare age has been considered for many years by Democrats. It was seen as a way to help older Americans afford medical care before the enactment of the ACA. Before that law took effect, consumers were not guaranteed access to a health plan, causing many older Americans to go without coverage.

But Dr. Eibner and colleagues found that a Medicare buy-in would have little to no effect on total health insurance enrollment. A Medicare buy-in might increase enrollment by 400,000 to 1.6 million for those over age 50, while decreasing enrollment by 100,000 to 800,000 for those under age 50 because of rising premiums.

“It’s not doing a lot to get people covered,” Dr. Eibner said in the interview.

In the report, Dr. Eibner and colleagues estimated that between 2.8 million and 7.0 million people would choose to enroll, depending on the approach used to design a Medicare buy-in. They considered numerous potential options for the design of a Medicare expansion, including various levels of federal subsidy for people using the buy-in. Dr. Eibner and colleagues also considered whether insurers would respond by trying to selectively market to healthier individuals, increasing their chance of enrolling.

The envisioned Medicare buy-in would have no effect on the Medicare Trust Fund, which pools money available through previously collected dedicated taxes, Dr. Eibner and colleagues said. In creating their model, they drew upon data from the Survey of Income and Program Participation, the Medical Expenditure Panel Survey, and the Kaiser Family Foundation and Health Research and Educational Trust Employer Health Benefits Survey.

Dr. Eibner and colleagues noted “several important limitations” for their work. It does not look at how the buy-in might affect clinicians and hospitals. Lower Medicare payment rates might cause some physicians to turn away patients covered by a Medicare buy-in.

“On the other hand, even if some providers decided not to participate in the buy-in, the buy-in might offer enrollees a broader network than what is available on the individual market,” Dr. Eibner and colleagues wrote. “Furthermore, it is not clear that providers would be legally allowed to opt out of the buy-in while still accepting payment for current Medicare beneficiaries.”

The Rand report was developed with the support of funding by the AARP.

SOURCE: Eibner C et al. RAND Corporation. Research Report. 2019. doi: 10.7249/RR4246.

Allowing people aged 50-64 to buy into Medicare might result in higher premiums for people who purchase their health insurance on the individual market, a finding that runs counter to many people’s expectations, said the authors of a newly released report.

In the report, Christine Eibner, PhD, of the RAND Corporation and colleagues estimated that the premium for so-called bronze market plans might increase by 2%-10%, depending on the design of a Medicare buy-in program. (The bronze plans are ones with fewer benefits sold on exchanges created through the implementation of the Affordable Care Act of 2010 [ACA].)

A perception has been that younger adults have been in effect subsidizing the cost of older ones in the marketplace plans. Instead, it appears that younger adults who enroll in the individual market tend to be relatively unhealthy and thus expensive to cover.

“When older adults leave the market, insurers are left with a smaller pool of younger, less healthy, and relatively expensive people given their age, leading to higher premiums,” Dr. Eibner and colleagues said in the report.

This result was unexpected, as there has been discussion of using a Medicare buy-in to reduce the cost of premiums for others in the marketplace, Dr. Eibner and colleagues said. But this result is consistent with other recent findings, including research presented by the consulting firm Milliman at a Society of Actuaries meeting in June. The Blue Cross Blue Shield Association estimates that losing a large group of customers could raise premiums by about 10% for the remaining pool of insured people, the New York Times has reported.

A buy-in would be less expensive than most ACA plans and would not have the kinds of limits on spending used in private insurance. That might make it attractive to middle-aged Americans. Dr. Eibner and colleagues estimated the annual premium for a Medicare buy-in at $9,747 in 2022. For a 50-year-old, a bronze-level ACA plan would cost $9,208 and a gold-level one, $12,277. For a 60-year-old, the annual premium for a bronze-level plan might be $13,512 and $18,016 for a gold-level plan.

Total out-of-pocket health spending, including premiums, would fall, on average, by 16%-35% for those who moved from ACA-compliant individual market coverage to a buy-in. The lower spending reflects that buy-in enrollees would have access to Medicare payment rates, which are substantially lower than private rates, and lower administrative costs.

There may be growing interest in allowing people aged 50-64 to buy into Medicare if enthusiasm wanes for bids to create a giant national health program, Dr. Eibner said in an interview.

“If there is concern that Medicare for all is going too far, I think this option is something that could become more prominent,” she said.

Many Democratic lawmakers already are focused on a Medicare buy-in approach. Sen. Debbie Stabenow (D-Mich.) has 20 Democratic cosponsors for her bill, which would allow for a Medicare buy-in at age 50. Sen. Bernie Sanders (I-Vt.) has 14 Democratic cosponsors for the current version of his well-known “Medicare-for-all” bill. That’s two fewer than he had for the Medicare-for-all bill he offered in the 115th session of Congress (Jan. 3, 2017–Jan. 3, 2019).

Among the supporters of Sen. Stabenow’s bill are several 2020 presidential contenders: Sen. Cory Booker (D-N.J.), Sen. Amy Klobuchar (D-Minn.), and Sen. Kamala Harris (D-Calif). As of Saturday, Sen. Elizabeth Warren (D-Mass.) backed Sanders’ bill, but not Stabenow’s. But Sen. Warren also has spoken recently of a Medicare expansion for people at age 50 as a step on the path toward universal coverage. Former Vice President Joseph R. Biden and Mayor Pete Buttigieg of South Bend, Ind., have said they would like to offer Americans the option to buy into Medicare or a public plan.

The idea of lowering the Medicare age has been considered for many years by Democrats. It was seen as a way to help older Americans afford medical care before the enactment of the ACA. Before that law took effect, consumers were not guaranteed access to a health plan, causing many older Americans to go without coverage.

But Dr. Eibner and colleagues found that a Medicare buy-in would have little to no effect on total health insurance enrollment. A Medicare buy-in might increase enrollment by 400,000 to 1.6 million for those over age 50, while decreasing enrollment by 100,000 to 800,000 for those under age 50 because of rising premiums.

“It’s not doing a lot to get people covered,” Dr. Eibner said in the interview.

In the report, Dr. Eibner and colleagues estimated that between 2.8 million and 7.0 million people would choose to enroll, depending on the approach used to design a Medicare buy-in. They considered numerous potential options for the design of a Medicare expansion, including various levels of federal subsidy for people using the buy-in. Dr. Eibner and colleagues also considered whether insurers would respond by trying to selectively market to healthier individuals, increasing their chance of enrolling.

The envisioned Medicare buy-in would have no effect on the Medicare Trust Fund, which pools money available through previously collected dedicated taxes, Dr. Eibner and colleagues said. In creating their model, they drew upon data from the Survey of Income and Program Participation, the Medical Expenditure Panel Survey, and the Kaiser Family Foundation and Health Research and Educational Trust Employer Health Benefits Survey.

Dr. Eibner and colleagues noted “several important limitations” for their work. It does not look at how the buy-in might affect clinicians and hospitals. Lower Medicare payment rates might cause some physicians to turn away patients covered by a Medicare buy-in.

“On the other hand, even if some providers decided not to participate in the buy-in, the buy-in might offer enrollees a broader network than what is available on the individual market,” Dr. Eibner and colleagues wrote. “Furthermore, it is not clear that providers would be legally allowed to opt out of the buy-in while still accepting payment for current Medicare beneficiaries.”

The Rand report was developed with the support of funding by the AARP.

SOURCE: Eibner C et al. RAND Corporation. Research Report. 2019. doi: 10.7249/RR4246.