Liz Szabo

The Food and Drug Administration (FDA) plans to scrutinize the safety and efficacy of lab-developed tests — those designed, manufactured, and used in a single laboratory — far more thoroughly in the future.

Under a rule finalized in April, the FDA will treat facilities that develop and use lab tests as manufacturers and regulate tests as medical devices. That means that most lab tests will need an FDA review before going on sale.

The FDA will also impose new quality standards, requiring test manufacturers to report adverse events and create a registry of lab tests under the new rule, which will be phased in over 4 years.

FDA officials have been concerned for years about the reliability of commercial lab tests, which have ballooned into a multibillion-dollar industry.

Consumer groups have long urged the FDA to regulate lab tests more strictly, arguing that the lack of scrutiny allows doctors and patients to be exploited by bad actors such as Theranos, which falsely claimed that its tests could diagnose multiple diseases with a single drop of blood.

“When it comes to some of these tests that doctors are recommending for patients, many doctors are just crossing their fingers and relying on the representation of the company because nobody is checking” to verify a manufacturer’s claims, said Joshua Sharfstein, MD, vice dean for public health practice and community engagement at the Johns Hopkins Bloomberg School of Public Health, Baltimore, Maryland.
 

Nearly 12,000 Labs Making Medical Tests

Although the FDA estimates there are nearly 12,000 labs manufacturing medical tests, agency officials said they don’t know how many tests are being marketed. The FDA already requires that home test kits marketed directly to consumers, such as those used to detect COVID-19, get clearance from the agency before being sold.

“There’s plenty of time for industry to get its act together to develop the data that it might need to make a premarket application,” said Peter Lurie, MD, PhD, a former associate commissioner at the FDA. In 2015, Dr. Lurie led a report outlining some of the dangers of unregulated lab tests.

For the average physician who orders lab tests, nothing is going to immediately change because of the final rule, said Dr. Lurie, now president of the Center for Science in the Public Interest, a nonprofit consumer watchdog.

“Tomorrow, this will look just the same as it does today,” Dr. Lurie said. “For the next 3 years, the companies will be scurrying behind the scenes to comply with the early stages of implementation. But most of that will be invisible to the average practitioner.”

Dr. Lurie predicted the FDA will focus its scrutiny on tests that pose the greatest potential risk to patients, such as ones used to diagnose serious diseases or guide treatment for life-threatening conditions. “The least significant tests will likely get very limited, if any, scrutiny,” said Dr. Lurie, adding that the FDA will likely issue guidance about how it plans to define low- and high-risk tests. “My suspicion is that it will be probably a small minority of products that are subject to full premarket approval.”
 

Lab Industry Groups Push Back

But imposing new rules with the potential to affect an industry’s bottom line is no easy task.

The American Clinical Laboratory Association, which represents the lab industry, said in a statement that the FDA rule will “limit access to scores of critical tests, increase healthcare costs, and undermine innovation in new diagnostics.” Another industry group, the Association for Molecular Pathology, has warned of “significant and harmful disruption to laboratory medicine.”

The two associations have filed separate lawsuits, charging that the FDA overstepped the authority granted by Congress. In their lawsuits, groups claim that lab tests are professional services, not manufactured products. The groups noted that the Centers for Medicare & Medicaid Services (CMS) already inspects lab facilities. CMS does not assess the tests’ quality or reliability.

A recent Supreme Court decision could make those lawsuits more likely to succeed, said David Simon, JD, LLM, PhD, an assistant professor of law at the Northeastern University School of Law, Boston, Massachusetts.

In the case of Loper Bright Enterprises v. Raimondo, decided in June, justices overturned a long-standing precedent known as Chevron deference, which required courts to defer to federal agencies when interpreting ambiguous laws. That means that courts no longer have to accept the FDA’s definition of a device, Dr. Simon said.

“Because judges may have more active roles in defining agency authority, federal agencies may have correspondingly less robust roles in policymaking,” Dr. Simon wrote in an editorial coauthored with Michael J. Young, MD, MPhil, of Harvard Medical School, Boston.

The Supreme Court ruling could pressure Congress to more clearly define FDA’s ruling in regulating lab tests, Dr. Simon and Dr. Young wrote.

Members of Congress first introduced a bill to clarify the FDA’s role in regulating lab tests, called the VALID Act, in 2020. The bill stalled and, despite efforts to revive it, still hasn’t passed.

FDA officials have said they remain “open to working with Congress,” noting that any future legislation about lab-developed tests would supersede their current policy.

In an interview, Dr. Simon noted the FDA significantly narrowed the scope of the final rule in response to comments from critics who objected to an earlier version of the policy proposed in 2023. The final rule carves out several categories of tests that won’t need to apply for “premarket review.”

Notably, a “grandfather clause” will allow some lab tests already on the market to continue being sold without undergoing FDA’s premarket review process. In explaining the exemption, FDA officials said they did not want doctors and patients to lose access to tests on which they rely. But Dr. Lurie noted that because the FDA views all these tests as under its jurisdiction, the agency could opt to take a closer look “at a very old device that is causing a problem today.”

The FDA also will exempt tests approved by New York State’s Clinical Laboratory Evaluation Program, which conducts its own stringent reviews. And the FDA will continue to allow hospitals to develop tests for patients within their healthcare system without going through the FDA approval process, if no FDA-approved tests are available.

Hospital-based tests play a critical role in treating infectious diseases, said Amesh Adalja, MD, an infectious diseases specialist and senior scholar at the Johns Hopkins Center for Health Security. For example, a large research hospital treating a patient with cytomegalovirus may need to develop its own test to determine whether the infection is resistant to antiviral drugs, Dr. Adalja said.

“With novel infectious disease outbreaks, researchers are able to move quickly to make diagnostic tests months and months before commercial laboratories are able to get through regulatory processes,” Dr. Adalja said.

To help scientists respond quickly to emergencies, the FDA published special guidance for labs that develop unauthorized lab tests for disease outbreaks.

Medical groups such as the American Hospital Association and Infectious Diseases Society of America remain concerned about the burden of complying with new regulations.

“Many vital tests developed in hospitals and health systems may be subjected to unnecessary and costly paperwork,” said Stacey Hughes, executive vice president of the American Hospital Association, in a statement.

Other groups, such as the American Society of Clinical Oncology, praised the new FDA policy. In comments submitted to the FDA in 2023, the cancer group said it “emphatically supports” requiring lab tests to undergo FDA review.

“We appreciate FDA action to modernize oversight of these tests and are hopeful this rule will increase focus on the need to balance rapid diagnostic innovation with patient safety and access” Everett Vokes, MD, the group’s board chair, said in a statement released after the FDA’s final rule was published.

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

The Food and Drug Administration (FDA) plans to scrutinize the safety and efficacy of lab-developed tests — those designed, manufactured, and used in a single laboratory — far more thoroughly in the future.

Under a rule finalized in April, the FDA will treat facilities that develop and use lab tests as manufacturers and regulate tests as medical devices. That means that most lab tests will need an FDA review before going on sale.

The FDA will also impose new quality standards, requiring test manufacturers to report adverse events and create a registry of lab tests under the new rule, which will be phased in over 4 years.

FDA officials have been concerned for years about the reliability of commercial lab tests, which have ballooned into a multibillion-dollar industry.

Consumer groups have long urged the FDA to regulate lab tests more strictly, arguing that the lack of scrutiny allows doctors and patients to be exploited by bad actors such as Theranos, which falsely claimed that its tests could diagnose multiple diseases with a single drop of blood.

“When it comes to some of these tests that doctors are recommending for patients, many doctors are just crossing their fingers and relying on the representation of the company because nobody is checking” to verify a manufacturer’s claims, said Joshua Sharfstein, MD, vice dean for public health practice and community engagement at the Johns Hopkins Bloomberg School of Public Health, Baltimore, Maryland.
 

Nearly 12,000 Labs Making Medical Tests

Although the FDA estimates there are nearly 12,000 labs manufacturing medical tests, agency officials said they don’t know how many tests are being marketed. The FDA already requires that home test kits marketed directly to consumers, such as those used to detect COVID-19, get clearance from the agency before being sold.

“There’s plenty of time for industry to get its act together to develop the data that it might need to make a premarket application,” said Peter Lurie, MD, PhD, a former associate commissioner at the FDA. In 2015, Dr. Lurie led a report outlining some of the dangers of unregulated lab tests.

For the average physician who orders lab tests, nothing is going to immediately change because of the final rule, said Dr. Lurie, now president of the Center for Science in the Public Interest, a nonprofit consumer watchdog.

“Tomorrow, this will look just the same as it does today,” Dr. Lurie said. “For the next 3 years, the companies will be scurrying behind the scenes to comply with the early stages of implementation. But most of that will be invisible to the average practitioner.”

Dr. Lurie predicted the FDA will focus its scrutiny on tests that pose the greatest potential risk to patients, such as ones used to diagnose serious diseases or guide treatment for life-threatening conditions. “The least significant tests will likely get very limited, if any, scrutiny,” said Dr. Lurie, adding that the FDA will likely issue guidance about how it plans to define low- and high-risk tests. “My suspicion is that it will be probably a small minority of products that are subject to full premarket approval.”
 

Lab Industry Groups Push Back

But imposing new rules with the potential to affect an industry’s bottom line is no easy task.

The American Clinical Laboratory Association, which represents the lab industry, said in a statement that the FDA rule will “limit access to scores of critical tests, increase healthcare costs, and undermine innovation in new diagnostics.” Another industry group, the Association for Molecular Pathology, has warned of “significant and harmful disruption to laboratory medicine.”

The two associations have filed separate lawsuits, charging that the FDA overstepped the authority granted by Congress. In their lawsuits, groups claim that lab tests are professional services, not manufactured products. The groups noted that the Centers for Medicare & Medicaid Services (CMS) already inspects lab facilities. CMS does not assess the tests’ quality or reliability.

A recent Supreme Court decision could make those lawsuits more likely to succeed, said David Simon, JD, LLM, PhD, an assistant professor of law at the Northeastern University School of Law, Boston, Massachusetts.

In the case of Loper Bright Enterprises v. Raimondo, decided in June, justices overturned a long-standing precedent known as Chevron deference, which required courts to defer to federal agencies when interpreting ambiguous laws. That means that courts no longer have to accept the FDA’s definition of a device, Dr. Simon said.

“Because judges may have more active roles in defining agency authority, federal agencies may have correspondingly less robust roles in policymaking,” Dr. Simon wrote in an editorial coauthored with Michael J. Young, MD, MPhil, of Harvard Medical School, Boston.

The Supreme Court ruling could pressure Congress to more clearly define FDA’s ruling in regulating lab tests, Dr. Simon and Dr. Young wrote.

Members of Congress first introduced a bill to clarify the FDA’s role in regulating lab tests, called the VALID Act, in 2020. The bill stalled and, despite efforts to revive it, still hasn’t passed.

FDA officials have said they remain “open to working with Congress,” noting that any future legislation about lab-developed tests would supersede their current policy.

In an interview, Dr. Simon noted the FDA significantly narrowed the scope of the final rule in response to comments from critics who objected to an earlier version of the policy proposed in 2023. The final rule carves out several categories of tests that won’t need to apply for “premarket review.”

Notably, a “grandfather clause” will allow some lab tests already on the market to continue being sold without undergoing FDA’s premarket review process. In explaining the exemption, FDA officials said they did not want doctors and patients to lose access to tests on which they rely. But Dr. Lurie noted that because the FDA views all these tests as under its jurisdiction, the agency could opt to take a closer look “at a very old device that is causing a problem today.”

The FDA also will exempt tests approved by New York State’s Clinical Laboratory Evaluation Program, which conducts its own stringent reviews. And the FDA will continue to allow hospitals to develop tests for patients within their healthcare system without going through the FDA approval process, if no FDA-approved tests are available.

Hospital-based tests play a critical role in treating infectious diseases, said Amesh Adalja, MD, an infectious diseases specialist and senior scholar at the Johns Hopkins Center for Health Security. For example, a large research hospital treating a patient with cytomegalovirus may need to develop its own test to determine whether the infection is resistant to antiviral drugs, Dr. Adalja said.

“With novel infectious disease outbreaks, researchers are able to move quickly to make diagnostic tests months and months before commercial laboratories are able to get through regulatory processes,” Dr. Adalja said.

To help scientists respond quickly to emergencies, the FDA published special guidance for labs that develop unauthorized lab tests for disease outbreaks.

Medical groups such as the American Hospital Association and Infectious Diseases Society of America remain concerned about the burden of complying with new regulations.

“Many vital tests developed in hospitals and health systems may be subjected to unnecessary and costly paperwork,” said Stacey Hughes, executive vice president of the American Hospital Association, in a statement.

Other groups, such as the American Society of Clinical Oncology, praised the new FDA policy. In comments submitted to the FDA in 2023, the cancer group said it “emphatically supports” requiring lab tests to undergo FDA review.

“We appreciate FDA action to modernize oversight of these tests and are hopeful this rule will increase focus on the need to balance rapid diagnostic innovation with patient safety and access” Everett Vokes, MD, the group’s board chair, said in a statement released after the FDA’s final rule was published.

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

The Food and Drug Administration (FDA) plans to scrutinize the safety and efficacy of lab-developed tests — those designed, manufactured, and used in a single laboratory — far more thoroughly in the future.

Under a rule finalized in April, the FDA will treat facilities that develop and use lab tests as manufacturers and regulate tests as medical devices. That means that most lab tests will need an FDA review before going on sale.

The FDA will also impose new quality standards, requiring test manufacturers to report adverse events and create a registry of lab tests under the new rule, which will be phased in over 4 years.

FDA officials have been concerned for years about the reliability of commercial lab tests, which have ballooned into a multibillion-dollar industry.

Consumer groups have long urged the FDA to regulate lab tests more strictly, arguing that the lack of scrutiny allows doctors and patients to be exploited by bad actors such as Theranos, which falsely claimed that its tests could diagnose multiple diseases with a single drop of blood.

“When it comes to some of these tests that doctors are recommending for patients, many doctors are just crossing their fingers and relying on the representation of the company because nobody is checking” to verify a manufacturer’s claims, said Joshua Sharfstein, MD, vice dean for public health practice and community engagement at the Johns Hopkins Bloomberg School of Public Health, Baltimore, Maryland.
 

Nearly 12,000 Labs Making Medical Tests

Although the FDA estimates there are nearly 12,000 labs manufacturing medical tests, agency officials said they don’t know how many tests are being marketed. The FDA already requires that home test kits marketed directly to consumers, such as those used to detect COVID-19, get clearance from the agency before being sold.

“There’s plenty of time for industry to get its act together to develop the data that it might need to make a premarket application,” said Peter Lurie, MD, PhD, a former associate commissioner at the FDA. In 2015, Dr. Lurie led a report outlining some of the dangers of unregulated lab tests.

For the average physician who orders lab tests, nothing is going to immediately change because of the final rule, said Dr. Lurie, now president of the Center for Science in the Public Interest, a nonprofit consumer watchdog.

“Tomorrow, this will look just the same as it does today,” Dr. Lurie said. “For the next 3 years, the companies will be scurrying behind the scenes to comply with the early stages of implementation. But most of that will be invisible to the average practitioner.”

Dr. Lurie predicted the FDA will focus its scrutiny on tests that pose the greatest potential risk to patients, such as ones used to diagnose serious diseases or guide treatment for life-threatening conditions. “The least significant tests will likely get very limited, if any, scrutiny,” said Dr. Lurie, adding that the FDA will likely issue guidance about how it plans to define low- and high-risk tests. “My suspicion is that it will be probably a small minority of products that are subject to full premarket approval.”
 

Lab Industry Groups Push Back

But imposing new rules with the potential to affect an industry’s bottom line is no easy task.

The American Clinical Laboratory Association, which represents the lab industry, said in a statement that the FDA rule will “limit access to scores of critical tests, increase healthcare costs, and undermine innovation in new diagnostics.” Another industry group, the Association for Molecular Pathology, has warned of “significant and harmful disruption to laboratory medicine.”

The two associations have filed separate lawsuits, charging that the FDA overstepped the authority granted by Congress. In their lawsuits, groups claim that lab tests are professional services, not manufactured products. The groups noted that the Centers for Medicare & Medicaid Services (CMS) already inspects lab facilities. CMS does not assess the tests’ quality or reliability.

A recent Supreme Court decision could make those lawsuits more likely to succeed, said David Simon, JD, LLM, PhD, an assistant professor of law at the Northeastern University School of Law, Boston, Massachusetts.

In the case of Loper Bright Enterprises v. Raimondo, decided in June, justices overturned a long-standing precedent known as Chevron deference, which required courts to defer to federal agencies when interpreting ambiguous laws. That means that courts no longer have to accept the FDA’s definition of a device, Dr. Simon said.

“Because judges may have more active roles in defining agency authority, federal agencies may have correspondingly less robust roles in policymaking,” Dr. Simon wrote in an editorial coauthored with Michael J. Young, MD, MPhil, of Harvard Medical School, Boston.

The Supreme Court ruling could pressure Congress to more clearly define FDA’s ruling in regulating lab tests, Dr. Simon and Dr. Young wrote.

Members of Congress first introduced a bill to clarify the FDA’s role in regulating lab tests, called the VALID Act, in 2020. The bill stalled and, despite efforts to revive it, still hasn’t passed.

FDA officials have said they remain “open to working with Congress,” noting that any future legislation about lab-developed tests would supersede their current policy.

In an interview, Dr. Simon noted the FDA significantly narrowed the scope of the final rule in response to comments from critics who objected to an earlier version of the policy proposed in 2023. The final rule carves out several categories of tests that won’t need to apply for “premarket review.”

Notably, a “grandfather clause” will allow some lab tests already on the market to continue being sold without undergoing FDA’s premarket review process. In explaining the exemption, FDA officials said they did not want doctors and patients to lose access to tests on which they rely. But Dr. Lurie noted that because the FDA views all these tests as under its jurisdiction, the agency could opt to take a closer look “at a very old device that is causing a problem today.”

The FDA also will exempt tests approved by New York State’s Clinical Laboratory Evaluation Program, which conducts its own stringent reviews. And the FDA will continue to allow hospitals to develop tests for patients within their healthcare system without going through the FDA approval process, if no FDA-approved tests are available.

Hospital-based tests play a critical role in treating infectious diseases, said Amesh Adalja, MD, an infectious diseases specialist and senior scholar at the Johns Hopkins Center for Health Security. For example, a large research hospital treating a patient with cytomegalovirus may need to develop its own test to determine whether the infection is resistant to antiviral drugs, Dr. Adalja said.

“With novel infectious disease outbreaks, researchers are able to move quickly to make diagnostic tests months and months before commercial laboratories are able to get through regulatory processes,” Dr. Adalja said.

To help scientists respond quickly to emergencies, the FDA published special guidance for labs that develop unauthorized lab tests for disease outbreaks.

Medical groups such as the American Hospital Association and Infectious Diseases Society of America remain concerned about the burden of complying with new regulations.

“Many vital tests developed in hospitals and health systems may be subjected to unnecessary and costly paperwork,” said Stacey Hughes, executive vice president of the American Hospital Association, in a statement.

Other groups, such as the American Society of Clinical Oncology, praised the new FDA policy. In comments submitted to the FDA in 2023, the cancer group said it “emphatically supports” requiring lab tests to undergo FDA review.

“We appreciate FDA action to modernize oversight of these tests and are hopeful this rule will increase focus on the need to balance rapid diagnostic innovation with patient safety and access” Everett Vokes, MD, the group’s board chair, said in a statement released after the FDA’s final rule was published.

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

The pandemic has created a hostile environment for pregnant people and their babies.

Stress levels among expectant mothers have soared. Pregnant women with COVID are 5 times as likely as uninfected pregnant people to require intensive care and 22 times as likely to die. Infected moms are four times as likely to have a stillborn child.

Yet some of the pandemic’s greatest threats to infants’ health may not be apparent for years or even decades.

That’s because babies of COVID-infected moms are 60% more likely to be born very prematurely, which increases the danger of infant mortality and long-term disabilities such as cerebral palsy, asthma, and hearing loss, as well as a child’s risk of adult disease, including depression, anxiety, heart disease, and kidney disease.

Studies have linked fever and infection during pregnancy to developmental and psychiatric conditions such as autism, depression, and schizophrenia.

“Some of these conditions do not show up until middle childhood or early adult life, but they have their origins in fetal life,” said Evdokia Anagnostou, MD, a child neurologist at Holland Bloorview Kids Rehabilitation Hospital and a pediatrics professor at the University of Toronto.

For fetuses exposed to COVID, the greatest danger is usually not the coronavirus itself, but the mother’s immune system.

Both severe COVID infections and the strain of the pandemic can expose fetuses to harmful inflammation, which can occur when a mother’s immune system is fighting a virus or when stress hormones send nonstop alarm signals.

Prenatal inflammation “changes the way the brain develops and, depending on the timing of the infection, it can change the way the heart or kidneys develop,” Dr. Anagnostou said.

Although health officials have strongly recommended COVID vaccines for pregnant people, only 35% are fully vaccinated.

At least 150,000 pregnant women have been diagnosed with COVID; more than 25,000 of them have been hospitalized, and 249 have died, according to the Centers for Disease Control and Prevention.

Although most babies will be fine, even a small increase in the percentage of children with special medical or educational needs could have a large effect on the population, given the huge number of COVID infections, Dr. Anagnostou said.

“If someone has a baby who is doing well, that is what they should focus on,” Dr. Anagnostou said. “But from a public health point of view, we need to follow women who experienced severe COVID and their babies to understand the impact.”

Learning from history

Researchers in the United States and other countries are already studying “the COVID generation” to see whether these children have more health issues than those conceived or born before 2020.

Previous crises have shown that the challenges fetuses face in the womb – such as maternal infections, hunger, stress, and hormone-disrupting chemicals – can leave a lasting imprint on their health, as well as that of their children and grandchildren, said Frederick Kaskel, MD, director of pediatric nephrology at the Children’s Hospital at Montefiore, New York.

People whose mothers were pregnant during surges in the 1918 influenza pandemic, for example, had poorer health throughout their lives, compared with Americans born at other times, said John McCarthy, who is a medical student at Albert Einstein College of Medicine, New York, and cowrote a recent review in JAMA Pediatrics with Dr. Kaskel.

Researchers don’t know exactly which moms were infected with pandemic flu, Mr. McCarthy said. But women who were pregnant during major surges – when infection was widespread – had children with higher rates of heart disease or diabetes. These children were also less successful in school, less economically productive, and more likely to live with a disability.

Because organ systems develop during different periods of pregnancy, fetuses exposed during the first trimester may face different risks than those exposed toward the end of pregnancy, Mr. McCarthy said. For example, people born in the fall of 1918 were 50% more likely than others to develop kidney disease; that may reflect an exposure to the pandemic in the third trimester, while the kidneys were still developing.

Nearly 2 years into the COVID pandemic, researchers have begun to publish preliminary observations of infants exposed to COVID infections and stress before birth.

Although Dr. Anagnostou noted that it’s too early to reach definitive conclusions, “there is evidence that babies born to moms with severe COVID infections have changes to their immune system,” she said. “It’s enough to make us worry a little bit.”

Damaging a fetal security system

The good news about the coronavirus is that it seldom crosses the placenta, the organ tasked with protecting a developing fetus from infections and providing it with oxygen. So moms with COVID rarely give the virus to their children before birth.

That’s important, because some viruses that directly infect the fetus – such as Zika – can cause devastating birth defects, said Karin Nielsen-Saines, MD, a specialist in pediatric infectious diseases at University of California, Los Angeles.

But studies also suggest that inflammation from a mother’s COVID infection can injure the placenta, said Jeffery Goldstein, MD, an assistant professor of pathology at Northwestern University, Chicago. In a study published in American Journal of Clinical Pathology , Dr. Goldstein and his coauthors found that placentas from COVID-infected moms had more abnormal blood vessels than placentas from patients without COVID, making it harder for them to deliver sufficient oxygen to the fetus.

Placental damage can also lead to preeclampsia, a serious complication of pregnancy that can cause a mother’s blood pressure to spike.

Preeclampsia occurs when blood vessels in the placenta don’t develop or function properly, forcing the mother’s heart to work harder to get blood to the fetus, which may not receive enough oxygen and nutrients. Preeclampsia also predisposes women to heart attacks and strokes later in life.

Rewiring the immune system

In some cases, COVID also appears to rewire a baby’s immune response, Dr. Nielsen-Saines said.

In an October study in the journal Cell Reports Medicine, Dr. Nielsen-Saines and her coauthors found that infants born to people with severe COVID infections had a different mix of immune cells and proteins than other babies. None of the newborns tested positive for the coronavirus.

The immune changes are concerning, Dr. Nielsen-Saines said, because this pattern of immune cells and proteins has previously been found in infants with respiratory problems and in some cases poor neurodevelopment.

Notably, all the babies in her study appear healthy, said Dr. Nielsen-Saines, who plans to follow them for 3 years to see whether these early signals translate into developmental delays, such as problems talking, walking, or interacting with others.

“How big of a difference does any of this make in the baby?” asked Dr. Anagnostou. “We won’t know for a few years. All we can do is try to be as prepared as possible.”

Increasing the risk for boys

Boys could face higher risks from COVID, even before birth.

Males are generally more vulnerable than females as fetuses and newborns; they’re more likely to be born prematurely and to die as infants. Preterm boys also have a higher risk of disability and death.

But coronavirus infection poses special dangers, said Sabra Klein, PhD, a professor of molecular microbiology and immunology at the Johns Hopkins Bloomberg School of Public Health, Baltimore.

That’s because boys are disproportionately affected by conditions linked to maternal infections. Boys are four times as likely as girls to be diagnosed with autism or attention-deficit/hyperactivity disorder, for example, while men are 75% more likely than women to develop schizophrenia.

Scientists don’t fully understand why boys appear more fragile in the womb, although testosterone – which can dampen immune response – may play a role, said Kristina Adams Waldorf, MD, a professor of obstetrics and gynecology at the University of Washington.

Men generally mount weaker immune responses than women and more often develop severe COVID infections. Recent research suggests boys with COVID are more likely than girls to become seriously ill or develop a rare inflammatory condition called multisystem inflammatory syndrome.

New research on COVID could help illuminate this vulnerability.

In a study published in October, researchers found that the sex of a fetus influences the way its placenta responds to COVID, as well as how its mother’s immune system responds.

Pregnant people infected with COVID made fewer antibodies against the coronavirus if they were carrying male fetuses than if they were carrying females. Mothers also transferred fewer antibodies to boys than to girls, said Andrea Edlow, MD, senior author of the study and a maternal-fetal medicine specialist at Massachusetts General Hospital, Boston.

When examining the placentas of male fetuses after delivery, researchers found changes that could leave boys less protected against damaging inflammation.

The sex of a fetus can influence its mother’s response to other illnesses, as well.

For example, research shows that pregnant women with asthma have worse symptoms if they’re carrying a female. Women carrying males are slightly more likely to develop gestational diabetes.

Dr. Edlow said her findings raise questions about the “cross talk” between mother and baby. “The mom’s immune system is sensing there is a male fetus,” Dr. Edlow said. “And the fetus is actively communicating with the mom’s immune system.”

Boosting toxic stress

Rates of depression and stress among pregnant women have increased dramatically during the pandemic.

That’s concerning because chronic stress can lead to inflammation, affecting the babies of both infected and uninfected women, Dr. Anagnostou said.

Studies consistently show that infants born to mothers who experience significant stress during pregnancy have higher rates of short- and long-term health damage – including heart defects and obesity – than babies born to women with less stress.

“We know that inflammation directly influences the way a baby’s brain develops,” said Elinor Sullivan, PhD, an associate professor in psychiatry at Oregon Health & Science University, Portland.

Lockdowns, travel restrictions and physical distancing left many pregnant women without the support of family and friends. The stress of losing a loved one, a job, or a home further heightens the risks to moms and babies, said Dr. Sullivan, who is following children born during the pandemic for 5 years.

In research that has not yet been published, Dr. Sullivan found that babies of women who were pregnant during the pandemic showed more sadness and negative emotions in the first year of life, compared with infants of women who were pregnant before the pandemic.

The findings show the importance of helping and protecting pregnant people before and after delivery, said Dr. Sullivan, who conducted a separate study that found women who received more social support were less depressed.

Italian researchers are also studying the effect of maternal stress on infants’ behavior, as well as the way their genes are regulated.

Although stress-related inflammation doesn’t alter the structure of a baby’s genes, it can influence whether they’re turned on and off, said Livio Provenzi, PhD, a psychologist at the C. Mondino National Institute of Neurology Foundation in Pavia, Italy.

In Dr. Provenzi’s study of 163 mother-baby pairs he found differences in how genes that regulate the stress response were activated. Genes that help people respond to stress were more likely to be turned off in babies whose moms reported the most stress during pregnancy. The same moms also reported that their babies cried more and were fussier when they were 3 months old.

Researchers usually prefer to make in-person observations of babies as they interact with their mothers, Dr. Provenzi said. But because of the pandemic, Dr. Provenzi asked mothers to fill out questionnaires about infant behavior. He plans to observe mothers and babies in person when the children are 12 months old.

While vaccinating pregnant people is the best way to protect them and their fetuses from the virus, Dr. Anagnostou said, society needs to do more to preserve expectant mothers’ mental health.

“We can’t escape the fact that we’ve lived through 2 years of a pandemic,” Dr. Anagnostou said. “But we can think about opportunities for reducing the risk.”

KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.

The pandemic has created a hostile environment for pregnant people and their babies.

Stress levels among expectant mothers have soared. Pregnant women with COVID are 5 times as likely as uninfected pregnant people to require intensive care and 22 times as likely to die. Infected moms are four times as likely to have a stillborn child.

Yet some of the pandemic’s greatest threats to infants’ health may not be apparent for years or even decades.

That’s because babies of COVID-infected moms are 60% more likely to be born very prematurely, which increases the danger of infant mortality and long-term disabilities such as cerebral palsy, asthma, and hearing loss, as well as a child’s risk of adult disease, including depression, anxiety, heart disease, and kidney disease.

Studies have linked fever and infection during pregnancy to developmental and psychiatric conditions such as autism, depression, and schizophrenia.

“Some of these conditions do not show up until middle childhood or early adult life, but they have their origins in fetal life,” said Evdokia Anagnostou, MD, a child neurologist at Holland Bloorview Kids Rehabilitation Hospital and a pediatrics professor at the University of Toronto.

For fetuses exposed to COVID, the greatest danger is usually not the coronavirus itself, but the mother’s immune system.

Both severe COVID infections and the strain of the pandemic can expose fetuses to harmful inflammation, which can occur when a mother’s immune system is fighting a virus or when stress hormones send nonstop alarm signals.

Prenatal inflammation “changes the way the brain develops and, depending on the timing of the infection, it can change the way the heart or kidneys develop,” Dr. Anagnostou said.

Although health officials have strongly recommended COVID vaccines for pregnant people, only 35% are fully vaccinated.

At least 150,000 pregnant women have been diagnosed with COVID; more than 25,000 of them have been hospitalized, and 249 have died, according to the Centers for Disease Control and Prevention.

Although most babies will be fine, even a small increase in the percentage of children with special medical or educational needs could have a large effect on the population, given the huge number of COVID infections, Dr. Anagnostou said.

“If someone has a baby who is doing well, that is what they should focus on,” Dr. Anagnostou said. “But from a public health point of view, we need to follow women who experienced severe COVID and their babies to understand the impact.”

Learning from history

Researchers in the United States and other countries are already studying “the COVID generation” to see whether these children have more health issues than those conceived or born before 2020.

Previous crises have shown that the challenges fetuses face in the womb – such as maternal infections, hunger, stress, and hormone-disrupting chemicals – can leave a lasting imprint on their health, as well as that of their children and grandchildren, said Frederick Kaskel, MD, director of pediatric nephrology at the Children’s Hospital at Montefiore, New York.

People whose mothers were pregnant during surges in the 1918 influenza pandemic, for example, had poorer health throughout their lives, compared with Americans born at other times, said John McCarthy, who is a medical student at Albert Einstein College of Medicine, New York, and cowrote a recent review in JAMA Pediatrics with Dr. Kaskel.

Researchers don’t know exactly which moms were infected with pandemic flu, Mr. McCarthy said. But women who were pregnant during major surges – when infection was widespread – had children with higher rates of heart disease or diabetes. These children were also less successful in school, less economically productive, and more likely to live with a disability.

Because organ systems develop during different periods of pregnancy, fetuses exposed during the first trimester may face different risks than those exposed toward the end of pregnancy, Mr. McCarthy said. For example, people born in the fall of 1918 were 50% more likely than others to develop kidney disease; that may reflect an exposure to the pandemic in the third trimester, while the kidneys were still developing.

Nearly 2 years into the COVID pandemic, researchers have begun to publish preliminary observations of infants exposed to COVID infections and stress before birth.

Although Dr. Anagnostou noted that it’s too early to reach definitive conclusions, “there is evidence that babies born to moms with severe COVID infections have changes to their immune system,” she said. “It’s enough to make us worry a little bit.”

Damaging a fetal security system

The good news about the coronavirus is that it seldom crosses the placenta, the organ tasked with protecting a developing fetus from infections and providing it with oxygen. So moms with COVID rarely give the virus to their children before birth.

That’s important, because some viruses that directly infect the fetus – such as Zika – can cause devastating birth defects, said Karin Nielsen-Saines, MD, a specialist in pediatric infectious diseases at University of California, Los Angeles.

But studies also suggest that inflammation from a mother’s COVID infection can injure the placenta, said Jeffery Goldstein, MD, an assistant professor of pathology at Northwestern University, Chicago. In a study published in American Journal of Clinical Pathology , Dr. Goldstein and his coauthors found that placentas from COVID-infected moms had more abnormal blood vessels than placentas from patients without COVID, making it harder for them to deliver sufficient oxygen to the fetus.

Placental damage can also lead to preeclampsia, a serious complication of pregnancy that can cause a mother’s blood pressure to spike.

Preeclampsia occurs when blood vessels in the placenta don’t develop or function properly, forcing the mother’s heart to work harder to get blood to the fetus, which may not receive enough oxygen and nutrients. Preeclampsia also predisposes women to heart attacks and strokes later in life.

Rewiring the immune system

In some cases, COVID also appears to rewire a baby’s immune response, Dr. Nielsen-Saines said.

In an October study in the journal Cell Reports Medicine, Dr. Nielsen-Saines and her coauthors found that infants born to people with severe COVID infections had a different mix of immune cells and proteins than other babies. None of the newborns tested positive for the coronavirus.

The immune changes are concerning, Dr. Nielsen-Saines said, because this pattern of immune cells and proteins has previously been found in infants with respiratory problems and in some cases poor neurodevelopment.

Notably, all the babies in her study appear healthy, said Dr. Nielsen-Saines, who plans to follow them for 3 years to see whether these early signals translate into developmental delays, such as problems talking, walking, or interacting with others.

“How big of a difference does any of this make in the baby?” asked Dr. Anagnostou. “We won’t know for a few years. All we can do is try to be as prepared as possible.”

Increasing the risk for boys

Boys could face higher risks from COVID, even before birth.

Males are generally more vulnerable than females as fetuses and newborns; they’re more likely to be born prematurely and to die as infants. Preterm boys also have a higher risk of disability and death.

But coronavirus infection poses special dangers, said Sabra Klein, PhD, a professor of molecular microbiology and immunology at the Johns Hopkins Bloomberg School of Public Health, Baltimore.

That’s because boys are disproportionately affected by conditions linked to maternal infections. Boys are four times as likely as girls to be diagnosed with autism or attention-deficit/hyperactivity disorder, for example, while men are 75% more likely than women to develop schizophrenia.

Scientists don’t fully understand why boys appear more fragile in the womb, although testosterone – which can dampen immune response – may play a role, said Kristina Adams Waldorf, MD, a professor of obstetrics and gynecology at the University of Washington.

Men generally mount weaker immune responses than women and more often develop severe COVID infections. Recent research suggests boys with COVID are more likely than girls to become seriously ill or develop a rare inflammatory condition called multisystem inflammatory syndrome.

New research on COVID could help illuminate this vulnerability.

In a study published in October, researchers found that the sex of a fetus influences the way its placenta responds to COVID, as well as how its mother’s immune system responds.

Pregnant people infected with COVID made fewer antibodies against the coronavirus if they were carrying male fetuses than if they were carrying females. Mothers also transferred fewer antibodies to boys than to girls, said Andrea Edlow, MD, senior author of the study and a maternal-fetal medicine specialist at Massachusetts General Hospital, Boston.

When examining the placentas of male fetuses after delivery, researchers found changes that could leave boys less protected against damaging inflammation.

The sex of a fetus can influence its mother’s response to other illnesses, as well.

For example, research shows that pregnant women with asthma have worse symptoms if they’re carrying a female. Women carrying males are slightly more likely to develop gestational diabetes.

Dr. Edlow said her findings raise questions about the “cross talk” between mother and baby. “The mom’s immune system is sensing there is a male fetus,” Dr. Edlow said. “And the fetus is actively communicating with the mom’s immune system.”

Boosting toxic stress

Rates of depression and stress among pregnant women have increased dramatically during the pandemic.

That’s concerning because chronic stress can lead to inflammation, affecting the babies of both infected and uninfected women, Dr. Anagnostou said.

Studies consistently show that infants born to mothers who experience significant stress during pregnancy have higher rates of short- and long-term health damage – including heart defects and obesity – than babies born to women with less stress.

“We know that inflammation directly influences the way a baby’s brain develops,” said Elinor Sullivan, PhD, an associate professor in psychiatry at Oregon Health & Science University, Portland.

Lockdowns, travel restrictions and physical distancing left many pregnant women without the support of family and friends. The stress of losing a loved one, a job, or a home further heightens the risks to moms and babies, said Dr. Sullivan, who is following children born during the pandemic for 5 years.

In research that has not yet been published, Dr. Sullivan found that babies of women who were pregnant during the pandemic showed more sadness and negative emotions in the first year of life, compared with infants of women who were pregnant before the pandemic.

The findings show the importance of helping and protecting pregnant people before and after delivery, said Dr. Sullivan, who conducted a separate study that found women who received more social support were less depressed.

Italian researchers are also studying the effect of maternal stress on infants’ behavior, as well as the way their genes are regulated.

Although stress-related inflammation doesn’t alter the structure of a baby’s genes, it can influence whether they’re turned on and off, said Livio Provenzi, PhD, a psychologist at the C. Mondino National Institute of Neurology Foundation in Pavia, Italy.

In Dr. Provenzi’s study of 163 mother-baby pairs he found differences in how genes that regulate the stress response were activated. Genes that help people respond to stress were more likely to be turned off in babies whose moms reported the most stress during pregnancy. The same moms also reported that their babies cried more and were fussier when they were 3 months old.

Researchers usually prefer to make in-person observations of babies as they interact with their mothers, Dr. Provenzi said. But because of the pandemic, Dr. Provenzi asked mothers to fill out questionnaires about infant behavior. He plans to observe mothers and babies in person when the children are 12 months old.

While vaccinating pregnant people is the best way to protect them and their fetuses from the virus, Dr. Anagnostou said, society needs to do more to preserve expectant mothers’ mental health.

“We can’t escape the fact that we’ve lived through 2 years of a pandemic,” Dr. Anagnostou said. “But we can think about opportunities for reducing the risk.”

KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.

The pandemic has created a hostile environment for pregnant people and their babies.

Stress levels among expectant mothers have soared. Pregnant women with COVID are 5 times as likely as uninfected pregnant people to require intensive care and 22 times as likely to die. Infected moms are four times as likely to have a stillborn child.

Yet some of the pandemic’s greatest threats to infants’ health may not be apparent for years or even decades.

That’s because babies of COVID-infected moms are 60% more likely to be born very prematurely, which increases the danger of infant mortality and long-term disabilities such as cerebral palsy, asthma, and hearing loss, as well as a child’s risk of adult disease, including depression, anxiety, heart disease, and kidney disease.

Studies have linked fever and infection during pregnancy to developmental and psychiatric conditions such as autism, depression, and schizophrenia.

“Some of these conditions do not show up until middle childhood or early adult life, but they have their origins in fetal life,” said Evdokia Anagnostou, MD, a child neurologist at Holland Bloorview Kids Rehabilitation Hospital and a pediatrics professor at the University of Toronto.

For fetuses exposed to COVID, the greatest danger is usually not the coronavirus itself, but the mother’s immune system.

Both severe COVID infections and the strain of the pandemic can expose fetuses to harmful inflammation, which can occur when a mother’s immune system is fighting a virus or when stress hormones send nonstop alarm signals.

Prenatal inflammation “changes the way the brain develops and, depending on the timing of the infection, it can change the way the heart or kidneys develop,” Dr. Anagnostou said.

Although health officials have strongly recommended COVID vaccines for pregnant people, only 35% are fully vaccinated.

At least 150,000 pregnant women have been diagnosed with COVID; more than 25,000 of them have been hospitalized, and 249 have died, according to the Centers for Disease Control and Prevention.

Although most babies will be fine, even a small increase in the percentage of children with special medical or educational needs could have a large effect on the population, given the huge number of COVID infections, Dr. Anagnostou said.

“If someone has a baby who is doing well, that is what they should focus on,” Dr. Anagnostou said. “But from a public health point of view, we need to follow women who experienced severe COVID and their babies to understand the impact.”

Learning from history

Researchers in the United States and other countries are already studying “the COVID generation” to see whether these children have more health issues than those conceived or born before 2020.

Previous crises have shown that the challenges fetuses face in the womb – such as maternal infections, hunger, stress, and hormone-disrupting chemicals – can leave a lasting imprint on their health, as well as that of their children and grandchildren, said Frederick Kaskel, MD, director of pediatric nephrology at the Children’s Hospital at Montefiore, New York.

People whose mothers were pregnant during surges in the 1918 influenza pandemic, for example, had poorer health throughout their lives, compared with Americans born at other times, said John McCarthy, who is a medical student at Albert Einstein College of Medicine, New York, and cowrote a recent review in JAMA Pediatrics with Dr. Kaskel.

Researchers don’t know exactly which moms were infected with pandemic flu, Mr. McCarthy said. But women who were pregnant during major surges – when infection was widespread – had children with higher rates of heart disease or diabetes. These children were also less successful in school, less economically productive, and more likely to live with a disability.

Because organ systems develop during different periods of pregnancy, fetuses exposed during the first trimester may face different risks than those exposed toward the end of pregnancy, Mr. McCarthy said. For example, people born in the fall of 1918 were 50% more likely than others to develop kidney disease; that may reflect an exposure to the pandemic in the third trimester, while the kidneys were still developing.

Nearly 2 years into the COVID pandemic, researchers have begun to publish preliminary observations of infants exposed to COVID infections and stress before birth.

Although Dr. Anagnostou noted that it’s too early to reach definitive conclusions, “there is evidence that babies born to moms with severe COVID infections have changes to their immune system,” she said. “It’s enough to make us worry a little bit.”

Damaging a fetal security system

The good news about the coronavirus is that it seldom crosses the placenta, the organ tasked with protecting a developing fetus from infections and providing it with oxygen. So moms with COVID rarely give the virus to their children before birth.

That’s important, because some viruses that directly infect the fetus – such as Zika – can cause devastating birth defects, said Karin Nielsen-Saines, MD, a specialist in pediatric infectious diseases at University of California, Los Angeles.

But studies also suggest that inflammation from a mother’s COVID infection can injure the placenta, said Jeffery Goldstein, MD, an assistant professor of pathology at Northwestern University, Chicago. In a study published in American Journal of Clinical Pathology , Dr. Goldstein and his coauthors found that placentas from COVID-infected moms had more abnormal blood vessels than placentas from patients without COVID, making it harder for them to deliver sufficient oxygen to the fetus.

Placental damage can also lead to preeclampsia, a serious complication of pregnancy that can cause a mother’s blood pressure to spike.

Preeclampsia occurs when blood vessels in the placenta don’t develop or function properly, forcing the mother’s heart to work harder to get blood to the fetus, which may not receive enough oxygen and nutrients. Preeclampsia also predisposes women to heart attacks and strokes later in life.

Rewiring the immune system

In some cases, COVID also appears to rewire a baby’s immune response, Dr. Nielsen-Saines said.

In an October study in the journal Cell Reports Medicine, Dr. Nielsen-Saines and her coauthors found that infants born to people with severe COVID infections had a different mix of immune cells and proteins than other babies. None of the newborns tested positive for the coronavirus.

The immune changes are concerning, Dr. Nielsen-Saines said, because this pattern of immune cells and proteins has previously been found in infants with respiratory problems and in some cases poor neurodevelopment.

Notably, all the babies in her study appear healthy, said Dr. Nielsen-Saines, who plans to follow them for 3 years to see whether these early signals translate into developmental delays, such as problems talking, walking, or interacting with others.

“How big of a difference does any of this make in the baby?” asked Dr. Anagnostou. “We won’t know for a few years. All we can do is try to be as prepared as possible.”

Increasing the risk for boys

Boys could face higher risks from COVID, even before birth.

Males are generally more vulnerable than females as fetuses and newborns; they’re more likely to be born prematurely and to die as infants. Preterm boys also have a higher risk of disability and death.

But coronavirus infection poses special dangers, said Sabra Klein, PhD, a professor of molecular microbiology and immunology at the Johns Hopkins Bloomberg School of Public Health, Baltimore.

That’s because boys are disproportionately affected by conditions linked to maternal infections. Boys are four times as likely as girls to be diagnosed with autism or attention-deficit/hyperactivity disorder, for example, while men are 75% more likely than women to develop schizophrenia.

Scientists don’t fully understand why boys appear more fragile in the womb, although testosterone – which can dampen immune response – may play a role, said Kristina Adams Waldorf, MD, a professor of obstetrics and gynecology at the University of Washington.

Men generally mount weaker immune responses than women and more often develop severe COVID infections. Recent research suggests boys with COVID are more likely than girls to become seriously ill or develop a rare inflammatory condition called multisystem inflammatory syndrome.

New research on COVID could help illuminate this vulnerability.

In a study published in October, researchers found that the sex of a fetus influences the way its placenta responds to COVID, as well as how its mother’s immune system responds.

Pregnant people infected with COVID made fewer antibodies against the coronavirus if they were carrying male fetuses than if they were carrying females. Mothers also transferred fewer antibodies to boys than to girls, said Andrea Edlow, MD, senior author of the study and a maternal-fetal medicine specialist at Massachusetts General Hospital, Boston.

When examining the placentas of male fetuses after delivery, researchers found changes that could leave boys less protected against damaging inflammation.

The sex of a fetus can influence its mother’s response to other illnesses, as well.

For example, research shows that pregnant women with asthma have worse symptoms if they’re carrying a female. Women carrying males are slightly more likely to develop gestational diabetes.

Dr. Edlow said her findings raise questions about the “cross talk” between mother and baby. “The mom’s immune system is sensing there is a male fetus,” Dr. Edlow said. “And the fetus is actively communicating with the mom’s immune system.”

Boosting toxic stress

Rates of depression and stress among pregnant women have increased dramatically during the pandemic.

That’s concerning because chronic stress can lead to inflammation, affecting the babies of both infected and uninfected women, Dr. Anagnostou said.

Studies consistently show that infants born to mothers who experience significant stress during pregnancy have higher rates of short- and long-term health damage – including heart defects and obesity – than babies born to women with less stress.

“We know that inflammation directly influences the way a baby’s brain develops,” said Elinor Sullivan, PhD, an associate professor in psychiatry at Oregon Health & Science University, Portland.

Lockdowns, travel restrictions and physical distancing left many pregnant women without the support of family and friends. The stress of losing a loved one, a job, or a home further heightens the risks to moms and babies, said Dr. Sullivan, who is following children born during the pandemic for 5 years.

In research that has not yet been published, Dr. Sullivan found that babies of women who were pregnant during the pandemic showed more sadness and negative emotions in the first year of life, compared with infants of women who were pregnant before the pandemic.

The findings show the importance of helping and protecting pregnant people before and after delivery, said Dr. Sullivan, who conducted a separate study that found women who received more social support were less depressed.

Italian researchers are also studying the effect of maternal stress on infants’ behavior, as well as the way their genes are regulated.

Although stress-related inflammation doesn’t alter the structure of a baby’s genes, it can influence whether they’re turned on and off, said Livio Provenzi, PhD, a psychologist at the C. Mondino National Institute of Neurology Foundation in Pavia, Italy.

In Dr. Provenzi’s study of 163 mother-baby pairs he found differences in how genes that regulate the stress response were activated. Genes that help people respond to stress were more likely to be turned off in babies whose moms reported the most stress during pregnancy. The same moms also reported that their babies cried more and were fussier when they were 3 months old.

Researchers usually prefer to make in-person observations of babies as they interact with their mothers, Dr. Provenzi said. But because of the pandemic, Dr. Provenzi asked mothers to fill out questionnaires about infant behavior. He plans to observe mothers and babies in person when the children are 12 months old.

While vaccinating pregnant people is the best way to protect them and their fetuses from the virus, Dr. Anagnostou said, society needs to do more to preserve expectant mothers’ mental health.

“We can’t escape the fact that we’ve lived through 2 years of a pandemic,” Dr. Anagnostou said. “But we can think about opportunities for reducing the risk.”

KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.

When Kristen Kilmer was diagnosed with incurable breast cancer at age 38, her first thought was of her 8-year-old daughter. Ms. Kilmer lost her own mother as a teenager and was determined to get more time with her only child.

adventtr/iStock/Getty Images Plus

Ms. Kilmer searched for experimental treatments, opting for an unproven approach in which researchers select drugs based on the genes in patients’ tumors. Doctors have selected her treatments for the past 3 years based on the unique, ever-changing DNA of her cancer cells. Now 41, Ms. Kilmer has responded better than anyone dared to hope. Her cancer has gone into hiding; her tumors are no longer visible on medical scans.

Researchers call the strategy “precision medicine.”

Ms. Kilmer’s insurance company calls it experimental. As a consequence, her insurer has covered only a fraction of her care, forcing Ms. Kilmer to make an agonizing choice: stop taking a drug that costs nearly $17,000 a month or pay out-of-pocket, burdening her family with tremendous debt.

“When you are looking at your daughter, you ask yourself, ‘Do I take a medication that might allow me to see her graduate high school?’ ” asked Ms. Kilmer of Spearfish, S.D. “Or do you stop taking it to avoid causing her financial harm?”

The high cost of cutting-edge tests and treatments is threatening to keep precision medicine – one of the most celebrated areas in cancer research – out of reach for many patients. Patients who pay for these new treatments on their own “could be in debt for decades,” said Scott Ramsey, MD, PhD, director of the Hutchinson Institute for Cancer Outcomes Research in Seattle.

Cancer care already is hugely expensive. A recent study in the American Journal of Medicine found that 42% of patients depleted 100% of their assets – an average loss of $92,000 – within 2 years of diagnosis.

Precision medicine involves running expensive tests called genomic sequencing, which scan the DNA of tumors to find mutations that might be susceptible to available drugs. Although the field is relatively new, hundreds of thousands of cancer patients have had their tumors sequenced to identify cancer-related mutations, according to testing companies.

Medicare, the government insurance plan for people 65 and older, announced in March that it will pay for genomic testing for people with advanced cancers – a decision that could add $2.5 billion to federal health care costs, according to a May analysis in Health Affairs.

Few private insurers cover the tests, leaving some patients with surprise medical bills.

Carrie Wyman, who also has advanced breast cancer, discovered that her insurance plan wouldn’t cover genomic sequencing only after she had received a $5,800 statement.

“I just assumed it would be covered,” said Ms. Wyman, 50, a resident of La Plata, Md., who has six children and stepchildren. “I was blindsided, to be honest with you.”
 

Looking for financial assistance

Yet paying for that initial test is just the beginning. As Ms. Kilmer learned, finding the money for ongoing treatment is far more challenging, said Gary H. Lyman, MD, who studies way to improve health care quality at Seattle’s Fred Hutchinson Cancer Research Center.

In some cases, genomic tests match patients to experimental drugs available only in clinical trials. Although these trials sometimes provide free medications, many cancer patients can’t afford to travel to participate in them. Ms. Kilmer drives 12 hours round-trip every month to participate in a clinical trial in Sioux Falls, S.D. The expenses add up quickly, she said.

Ms. Kilmer’s genomic tests identified a rearrangement in the PALB2 gene. Preliminary studies suggest that tumors with this genetic rearrangement could be susceptible to the drug olaparib (Lynparza), but those effects haven’t been definitively proven in large-scale studies. The Food and Drug Administration has approved Lynparza only for breast cancer patients with a BRCA mutation.

Legally, doctors can prescribe Lynparza “off label” to anyone with cancer. But insurance programs are reluctant to cover off-label treatments, unless they’re specifically recommended in expert guidelines.

Ms. Kilmer has spent much of the past 3 years battling insurance officials and begging drug companies for financial assistance. The drugmakers have been generous, allowing her to take a rotating cocktail of experimental drugs for free because of her modest income.

In September, however, AstraZeneca decided to end Ms. Kilmer’s financial aid. Ms. Kilmer appealed the drug company’s decision.

Paying thousands of dollars a month is not an option, Ms. Kilmer said. Her family already carries significant credit card debt from earlier cancer treatments. She estimates that she has spent about $80,600 out-of-pocket treating her illness, including $23,600 on her early breast cancer therapy and $57,000 treating metastatic disease.

Ms. Kilmer said she would rather stop taking Lynparza than financially burden her daughter and husband, a truck driver.

“It’s not worth it,” Ms. Kilmer said. “I will not put my family into that kind of debt.”
 

Uncertain benefits

Insurers say costs aren’t their only concern. Evidence is lacking that the precision medicine approach will work consistently, they argue.

America’s Health Insurance Plans, an industry group, said genetic sequencing remains unproven.

Cathryn Donaldson, the group’s spokeswoman, described recent scientific advances as “remarkable and noteworthy.” But she said insurers “need a more definitive answer” about whether the tests help the average patient live longer.

The South Dakota State Employee Health Plan – which runs Ms. Kilmer’s insurance plan – said it bases its coverage decisions on science and reviews “published, randomized data about the safety and efficacy of the requested drugs.”

Although genetic testing has become the standard of care for melanoma and a common type of lung cancer, no one knows if genomic sequencing will extend the lives of people with other types of cancer, said Carolyn J. Presley, MD, an assistant professor at the Ohio State University Comprehensive Cancer Center, Columbus.

Without insurance coverage, some cancer patients simply give up on treatment.

A study of more than 1,000 women with advanced breast cancer – presented at a September meeting of the American Society of Clinical Oncology – found that 54% had stopped or refused treatment because of costs. The women in the study may have been more vulnerable than most, because 30% were uninsured, about twice the national rate.

In an August study in JAMA, researchers found that relatively few of those who hoped to benefit from precision medicine actually ended up on a medication. Just 15% of those who underwent genomic sequencing ended up taking a targeted therapy, according to the study. The study didn’t ask participants why they failed to get a targeted drug, but Dr. Presley, the lead author, said it’s likely that some patients couldn’t afford them.

“We’re finding the mutations, but patients aren’t getting the drugs,” Dr. Presley said. Without insurance, she said, “you and I would not be able to afford these medications. It’s a huge barrier.”

Within hours of the publication of this story, AstraZeneca called Ms. Kilmer to notify her that it would continue to provide financial aid. Her medication arrived in the mail the next day.

“It’s a huge relief,” Ms. Kilmer said.
 

KHN’s coverage of these topics is supported by Laura and John Arnold Foundation and Gordon and Betty Moore Foundation. Kaiser Health News is a nonprofit national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation that is not affiliated with Kaiser Permanente.

When Kristen Kilmer was diagnosed with incurable breast cancer at age 38, her first thought was of her 8-year-old daughter. Ms. Kilmer lost her own mother as a teenager and was determined to get more time with her only child.

adventtr/iStock/Getty Images Plus

Ms. Kilmer searched for experimental treatments, opting for an unproven approach in which researchers select drugs based on the genes in patients’ tumors. Doctors have selected her treatments for the past 3 years based on the unique, ever-changing DNA of her cancer cells. Now 41, Ms. Kilmer has responded better than anyone dared to hope. Her cancer has gone into hiding; her tumors are no longer visible on medical scans.

Researchers call the strategy “precision medicine.”

Ms. Kilmer’s insurance company calls it experimental. As a consequence, her insurer has covered only a fraction of her care, forcing Ms. Kilmer to make an agonizing choice: stop taking a drug that costs nearly $17,000 a month or pay out-of-pocket, burdening her family with tremendous debt.

“When you are looking at your daughter, you ask yourself, ‘Do I take a medication that might allow me to see her graduate high school?’ ” asked Ms. Kilmer of Spearfish, S.D. “Or do you stop taking it to avoid causing her financial harm?”

The high cost of cutting-edge tests and treatments is threatening to keep precision medicine – one of the most celebrated areas in cancer research – out of reach for many patients. Patients who pay for these new treatments on their own “could be in debt for decades,” said Scott Ramsey, MD, PhD, director of the Hutchinson Institute for Cancer Outcomes Research in Seattle.

Cancer care already is hugely expensive. A recent study in the American Journal of Medicine found that 42% of patients depleted 100% of their assets – an average loss of $92,000 – within 2 years of diagnosis.

Precision medicine involves running expensive tests called genomic sequencing, which scan the DNA of tumors to find mutations that might be susceptible to available drugs. Although the field is relatively new, hundreds of thousands of cancer patients have had their tumors sequenced to identify cancer-related mutations, according to testing companies.

Medicare, the government insurance plan for people 65 and older, announced in March that it will pay for genomic testing for people with advanced cancers – a decision that could add $2.5 billion to federal health care costs, according to a May analysis in Health Affairs.

Few private insurers cover the tests, leaving some patients with surprise medical bills.

Carrie Wyman, who also has advanced breast cancer, discovered that her insurance plan wouldn’t cover genomic sequencing only after she had received a $5,800 statement.

“I just assumed it would be covered,” said Ms. Wyman, 50, a resident of La Plata, Md., who has six children and stepchildren. “I was blindsided, to be honest with you.”
 

Looking for financial assistance

Yet paying for that initial test is just the beginning. As Ms. Kilmer learned, finding the money for ongoing treatment is far more challenging, said Gary H. Lyman, MD, who studies way to improve health care quality at Seattle’s Fred Hutchinson Cancer Research Center.

In some cases, genomic tests match patients to experimental drugs available only in clinical trials. Although these trials sometimes provide free medications, many cancer patients can’t afford to travel to participate in them. Ms. Kilmer drives 12 hours round-trip every month to participate in a clinical trial in Sioux Falls, S.D. The expenses add up quickly, she said.

Ms. Kilmer’s genomic tests identified a rearrangement in the PALB2 gene. Preliminary studies suggest that tumors with this genetic rearrangement could be susceptible to the drug olaparib (Lynparza), but those effects haven’t been definitively proven in large-scale studies. The Food and Drug Administration has approved Lynparza only for breast cancer patients with a BRCA mutation.

Legally, doctors can prescribe Lynparza “off label” to anyone with cancer. But insurance programs are reluctant to cover off-label treatments, unless they’re specifically recommended in expert guidelines.

Ms. Kilmer has spent much of the past 3 years battling insurance officials and begging drug companies for financial assistance. The drugmakers have been generous, allowing her to take a rotating cocktail of experimental drugs for free because of her modest income.

In September, however, AstraZeneca decided to end Ms. Kilmer’s financial aid. Ms. Kilmer appealed the drug company’s decision.

Paying thousands of dollars a month is not an option, Ms. Kilmer said. Her family already carries significant credit card debt from earlier cancer treatments. She estimates that she has spent about $80,600 out-of-pocket treating her illness, including $23,600 on her early breast cancer therapy and $57,000 treating metastatic disease.

Ms. Kilmer said she would rather stop taking Lynparza than financially burden her daughter and husband, a truck driver.

“It’s not worth it,” Ms. Kilmer said. “I will not put my family into that kind of debt.”
 

Uncertain benefits

Insurers say costs aren’t their only concern. Evidence is lacking that the precision medicine approach will work consistently, they argue.

America’s Health Insurance Plans, an industry group, said genetic sequencing remains unproven.

Cathryn Donaldson, the group’s spokeswoman, described recent scientific advances as “remarkable and noteworthy.” But she said insurers “need a more definitive answer” about whether the tests help the average patient live longer.

The South Dakota State Employee Health Plan – which runs Ms. Kilmer’s insurance plan – said it bases its coverage decisions on science and reviews “published, randomized data about the safety and efficacy of the requested drugs.”

Although genetic testing has become the standard of care for melanoma and a common type of lung cancer, no one knows if genomic sequencing will extend the lives of people with other types of cancer, said Carolyn J. Presley, MD, an assistant professor at the Ohio State University Comprehensive Cancer Center, Columbus.

Without insurance coverage, some cancer patients simply give up on treatment.

A study of more than 1,000 women with advanced breast cancer – presented at a September meeting of the American Society of Clinical Oncology – found that 54% had stopped or refused treatment because of costs. The women in the study may have been more vulnerable than most, because 30% were uninsured, about twice the national rate.

In an August study in JAMA, researchers found that relatively few of those who hoped to benefit from precision medicine actually ended up on a medication. Just 15% of those who underwent genomic sequencing ended up taking a targeted therapy, according to the study. The study didn’t ask participants why they failed to get a targeted drug, but Dr. Presley, the lead author, said it’s likely that some patients couldn’t afford them.

“We’re finding the mutations, but patients aren’t getting the drugs,” Dr. Presley said. Without insurance, she said, “you and I would not be able to afford these medications. It’s a huge barrier.”

Within hours of the publication of this story, AstraZeneca called Ms. Kilmer to notify her that it would continue to provide financial aid. Her medication arrived in the mail the next day.

“It’s a huge relief,” Ms. Kilmer said.
 

KHN’s coverage of these topics is supported by Laura and John Arnold Foundation and Gordon and Betty Moore Foundation. Kaiser Health News is a nonprofit national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation that is not affiliated with Kaiser Permanente.

When Kristen Kilmer was diagnosed with incurable breast cancer at age 38, her first thought was of her 8-year-old daughter. Ms. Kilmer lost her own mother as a teenager and was determined to get more time with her only child.

adventtr/iStock/Getty Images Plus

Ms. Kilmer searched for experimental treatments, opting for an unproven approach in which researchers select drugs based on the genes in patients’ tumors. Doctors have selected her treatments for the past 3 years based on the unique, ever-changing DNA of her cancer cells. Now 41, Ms. Kilmer has responded better than anyone dared to hope. Her cancer has gone into hiding; her tumors are no longer visible on medical scans.

Researchers call the strategy “precision medicine.”

Ms. Kilmer’s insurance company calls it experimental. As a consequence, her insurer has covered only a fraction of her care, forcing Ms. Kilmer to make an agonizing choice: stop taking a drug that costs nearly $17,000 a month or pay out-of-pocket, burdening her family with tremendous debt.

“When you are looking at your daughter, you ask yourself, ‘Do I take a medication that might allow me to see her graduate high school?’ ” asked Ms. Kilmer of Spearfish, S.D. “Or do you stop taking it to avoid causing her financial harm?”

The high cost of cutting-edge tests and treatments is threatening to keep precision medicine – one of the most celebrated areas in cancer research – out of reach for many patients. Patients who pay for these new treatments on their own “could be in debt for decades,” said Scott Ramsey, MD, PhD, director of the Hutchinson Institute for Cancer Outcomes Research in Seattle.

Cancer care already is hugely expensive. A recent study in the American Journal of Medicine found that 42% of patients depleted 100% of their assets – an average loss of $92,000 – within 2 years of diagnosis.

Precision medicine involves running expensive tests called genomic sequencing, which scan the DNA of tumors to find mutations that might be susceptible to available drugs. Although the field is relatively new, hundreds of thousands of cancer patients have had their tumors sequenced to identify cancer-related mutations, according to testing companies.

Medicare, the government insurance plan for people 65 and older, announced in March that it will pay for genomic testing for people with advanced cancers – a decision that could add $2.5 billion to federal health care costs, according to a May analysis in Health Affairs.

Few private insurers cover the tests, leaving some patients with surprise medical bills.

Carrie Wyman, who also has advanced breast cancer, discovered that her insurance plan wouldn’t cover genomic sequencing only after she had received a $5,800 statement.

“I just assumed it would be covered,” said Ms. Wyman, 50, a resident of La Plata, Md., who has six children and stepchildren. “I was blindsided, to be honest with you.”
 

Looking for financial assistance

Yet paying for that initial test is just the beginning. As Ms. Kilmer learned, finding the money for ongoing treatment is far more challenging, said Gary H. Lyman, MD, who studies way to improve health care quality at Seattle’s Fred Hutchinson Cancer Research Center.

In some cases, genomic tests match patients to experimental drugs available only in clinical trials. Although these trials sometimes provide free medications, many cancer patients can’t afford to travel to participate in them. Ms. Kilmer drives 12 hours round-trip every month to participate in a clinical trial in Sioux Falls, S.D. The expenses add up quickly, she said.

Ms. Kilmer’s genomic tests identified a rearrangement in the PALB2 gene. Preliminary studies suggest that tumors with this genetic rearrangement could be susceptible to the drug olaparib (Lynparza), but those effects haven’t been definitively proven in large-scale studies. The Food and Drug Administration has approved Lynparza only for breast cancer patients with a BRCA mutation.

Legally, doctors can prescribe Lynparza “off label” to anyone with cancer. But insurance programs are reluctant to cover off-label treatments, unless they’re specifically recommended in expert guidelines.

Ms. Kilmer has spent much of the past 3 years battling insurance officials and begging drug companies for financial assistance. The drugmakers have been generous, allowing her to take a rotating cocktail of experimental drugs for free because of her modest income.

In September, however, AstraZeneca decided to end Ms. Kilmer’s financial aid. Ms. Kilmer appealed the drug company’s decision.

Paying thousands of dollars a month is not an option, Ms. Kilmer said. Her family already carries significant credit card debt from earlier cancer treatments. She estimates that she has spent about $80,600 out-of-pocket treating her illness, including $23,600 on her early breast cancer therapy and $57,000 treating metastatic disease.

Ms. Kilmer said she would rather stop taking Lynparza than financially burden her daughter and husband, a truck driver.

“It’s not worth it,” Ms. Kilmer said. “I will not put my family into that kind of debt.”
 

Uncertain benefits

Insurers say costs aren’t their only concern. Evidence is lacking that the precision medicine approach will work consistently, they argue.

America’s Health Insurance Plans, an industry group, said genetic sequencing remains unproven.

Cathryn Donaldson, the group’s spokeswoman, described recent scientific advances as “remarkable and noteworthy.” But she said insurers “need a more definitive answer” about whether the tests help the average patient live longer.

The South Dakota State Employee Health Plan – which runs Ms. Kilmer’s insurance plan – said it bases its coverage decisions on science and reviews “published, randomized data about the safety and efficacy of the requested drugs.”

Although genetic testing has become the standard of care for melanoma and a common type of lung cancer, no one knows if genomic sequencing will extend the lives of people with other types of cancer, said Carolyn J. Presley, MD, an assistant professor at the Ohio State University Comprehensive Cancer Center, Columbus.

Without insurance coverage, some cancer patients simply give up on treatment.

A study of more than 1,000 women with advanced breast cancer – presented at a September meeting of the American Society of Clinical Oncology – found that 54% had stopped or refused treatment because of costs. The women in the study may have been more vulnerable than most, because 30% were uninsured, about twice the national rate.

In an August study in JAMA, researchers found that relatively few of those who hoped to benefit from precision medicine actually ended up on a medication. Just 15% of those who underwent genomic sequencing ended up taking a targeted therapy, according to the study. The study didn’t ask participants why they failed to get a targeted drug, but Dr. Presley, the lead author, said it’s likely that some patients couldn’t afford them.

“We’re finding the mutations, but patients aren’t getting the drugs,” Dr. Presley said. Without insurance, she said, “you and I would not be able to afford these medications. It’s a huge barrier.”

Within hours of the publication of this story, AstraZeneca called Ms. Kilmer to notify her that it would continue to provide financial aid. Her medication arrived in the mail the next day.

“It’s a huge relief,” Ms. Kilmer said.
 

KHN’s coverage of these topics is supported by Laura and John Arnold Foundation and Gordon and Betty Moore Foundation. Kaiser Health News is a nonprofit national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation that is not affiliated with Kaiser Permanente.

At 87, Maxine Stanich cared more about improving the quality of her life than prolonging it.

She suffered from a long list of health problems, including heart failure and chronic lung disease that could leave her gasping for breath.

When her time came, she wanted to die a natural death, Stanich told her daughter, and signed a “do not resuscitate” directive, or DNR, ordering doctors not to revive her should her heart stop.

Yet a trip to a San Francisco emergency room for shortness of breath in 2008 led Stanich to get a defibrillator implanted in her chest – a medical device to keep her alive by delivering a powerful shock. At the time, Stanich didn’t fully grasp what she had agreed to, even though she signed a document granting permission for the procedure, said her daughter, Susan Giaquinto.

That clarity came only during a subsequent visit to a different hospital, when a surprised ER doctor saw a defibrillator protruding from the DNR patient’s thin chest. To Stanich’s horror, the ER doctor explained that the device would not allow her to slip away painlessly and that the jolt would be “so strong that it will knock her across the room,” said Giaquinto, who accompanied her mother on both hospital trips.

Surgery like this has become all too common among those near the end of life, experts say. Nearly one in three Medicare patients undergo an operation in the year before they die, even though the evidence shows that many are more likely to be harmed than to benefit from it.

The practice is driven by financial incentives that reward doctors for doing procedures, as well as a medical culture in which patients and doctors are reluctant to talk about how surgical interventions should be prescribed more judiciously, said Rita Redberg, M.D., a cardiologist who treated Stanich when she sought care at the second hospital.

“We have a culture that believes in very aggressive care,” said Dr. Redberg, who at the University of California–San Francisco specializes in heart disease in women. “We are often not considering the chance of benefit and chance of harm, and how that changes when you get older. We also fail to have conversations about what patients value most.”

While surgery is typically lifesaving for younger people, operating on frail, older patients rarely helps them live longer or returns the quality of life they once enjoyed, according to a 2016 paper in Annals of Surgery.

The cost of these surgeries – typically paid for by Medicare, the government health insurance program for people over 65 – involve more than money, said Amber Barnato, MD, professor at the Dartmouth Institute for Health Policy and Clinical Practice. Older patients who undergo surgery within a year of death spent 50 percent more time in the hospital than others, and nearly twice as many days in intensive care.

And while some robust octogenarians have many years ahead of them, studies show that surgery is also common among those who are far more frail.

Eighteen percent of Medicare patients have surgery in their final month of life and 8% in their final week, according to a 2011 study in The Lancet.

More than 12% of defibrillators were implanted in people older than 80, according to a 2015 study. Doctors implant about 158,000 of the devices each year, according to the American College of Cardiology. The total cost of the procedure runs about $60,000.

Procedures performed in the elderly range from major operations that require lengthy recoveries to relatively minor surgery performed in a doctor’s office, such as the removal of nonfatal skin cancers, that would likely never cause any problems.

Research led by Eleni Linos, MD, has shown that people with limited life expectancies are treated for nonfatal skin cancers as aggressively as younger patients. Among patients with a nonfatal skin cancer and a limited time to live, 70 percent underwent surgery, according to her 2013 study in JAMA Internal Medicine.

When less is more

Surgery poses serious risks for older people, who weather anesthesia poorly and whose skin takes longer to heal. Among seniors who undergo urgent or emergency abdominal surgery, 20% die within 30 days, studies show.

With diminished mental acuity and an old-fashioned respect for the medical profession, some aging patients are vulnerable to unwanted interventions. Stanich agreed to a pacemaker simply because her doctor suggested it, Giaquinto said. Many people of Stanich’s generation “thought doctors were God … They never questioned doctors – ever.”

Margaret Schwarze, MD, a surgeon and associate professor at the University of Wisconsin, said that older patients often don’t feel the financial pain of surgery because insurance pays most of the cost.

When a surgeon offers to “fix” the heart valve in a person with multiple diseases, for example, the patient may assume that surgery will fix all of her medical problems, Dr. Schwarze said. “With older patients with lots of chronic illnesses, we’re not really fixing anything.”

Even as a doctor, Dr. Redberg said, she struggles to prevent other doctors from performing too many procedures on her 92-year-old mother, Mae, who lives in New York City.

Dr. Redberg said doctors recently treated her mother for melanoma – the most serious type of skin cancer. After the cancer was removed from her leg, Dr. Redberg’s mother was urged by a doctor to undergo an additional surgery to cut away more tissue and nearby lymph nodes, which can harbor cancerous cells.

“Every time she went in, the dermatologist wanted to refer her to a surgeon,” Dr. Redberg said. And “Medicare would have been happy to pay for it.”

But her mother often has problems with wounds healing, she said, and recovery would likely have taken 3 months. When Dr. Redberg pressed a surgeon about the benefits, he said the procedure could reduce the chances of cancer coming back within 3-5 years.

Dr. Redberg said her mother laughed and said, “I’m not interested in doing something that will help me in 3-5 years. I doubt I’ll be here.”

Finding solutions

The momentum of hospital care can make people feel as if they’re on a moving train and can’t jump off.

The rush of medical decisions “doesn’t allow time to deliberate or consider the patients’ overall health or what their goals and values might be,” said Jacqueline Kruser, MD, an instructor in pulmonary and critical care medicine and medical social sciences at the Northwestern University.

Many hospitals and health systems are developing “decision aids,” easy-to-understand written materials and videos to help patients make more informed medical decisions, giving them time to develop more realistic expectations.

After Kaiser Permanente Washington introduced the tools relating to joint replacement, the number of patients choosing to have hip replacement surgery fell 26%, while knee replacements declined 38%, according to a study in Health Affairs. (Kaiser Permanente is not affiliated with Kaiser Health News, which is an editorially independent program of the Kaiser Family Foundation.)

In a paper published last year in JAMA Surgery and the Journal of Pain and Symptom Management, Dr. Schwarze, Dr. Kruser and colleagues suggested creating narratives to illustrate surgical risks, rather than relying on statistics.

Instead of telling patients that surgery carries a 20% risk of stroke, for example, doctors should lay out the best, worst and most likely outcomes.

In the best-case scenario, a patient might spend weeks in the hospital after surgery, living the rest of her life in a nursing home. In the worst case, the same patient dies after several weeks in intensive care. In the most likely scenario, the patient survives just 2-3 months after surgery.

Dr. Schwarze said, “If someone says they can’t tolerate the best-case scenario – which involves them being in a nursing home – then maybe we shouldn’t be doing this.”

Maxine Stanich was admitted to the hospital after going to the ER because she felt short of breath. She experienced an abnormal heart rhythm in the procedure room during a cardiac test – not an unusual event during a procedure in which a wire is threaded into the heart. Based on that, doctors decided to implant a pacemaker and defibrillator the next day.

Dr. Redberg was consulted when the patient objected to the device that was now embedded in her chest. She was “very alert. She was very clear about what she did and did not want done. She told me she didn’t want to be shocked,” Redberg said.

After Dr. Redberg deactivated the defibrillator, which can be reprogrammed remotely, Stanich was discharged, with home hospice service. With nothing more than her medicines, she survived another 2 years and 3 months, dying at home just after her 90th birthday in 2010.

Kaiser Health News is a nonprofit news service covering health issues. It is an editorially independent program of the Kaiser Family Foundation that is not affiliated with Kaiser Permanente. KHN’s coverage related to aging and improving care of older adults is supported in part by The John A. Hartford Foundation.

At 87, Maxine Stanich cared more about improving the quality of her life than prolonging it.

She suffered from a long list of health problems, including heart failure and chronic lung disease that could leave her gasping for breath.

When her time came, she wanted to die a natural death, Stanich told her daughter, and signed a “do not resuscitate” directive, or DNR, ordering doctors not to revive her should her heart stop.

Yet a trip to a San Francisco emergency room for shortness of breath in 2008 led Stanich to get a defibrillator implanted in her chest – a medical device to keep her alive by delivering a powerful shock. At the time, Stanich didn’t fully grasp what she had agreed to, even though she signed a document granting permission for the procedure, said her daughter, Susan Giaquinto.

That clarity came only during a subsequent visit to a different hospital, when a surprised ER doctor saw a defibrillator protruding from the DNR patient’s thin chest. To Stanich’s horror, the ER doctor explained that the device would not allow her to slip away painlessly and that the jolt would be “so strong that it will knock her across the room,” said Giaquinto, who accompanied her mother on both hospital trips.

Surgery like this has become all too common among those near the end of life, experts say. Nearly one in three Medicare patients undergo an operation in the year before they die, even though the evidence shows that many are more likely to be harmed than to benefit from it.

The practice is driven by financial incentives that reward doctors for doing procedures, as well as a medical culture in which patients and doctors are reluctant to talk about how surgical interventions should be prescribed more judiciously, said Rita Redberg, M.D., a cardiologist who treated Stanich when she sought care at the second hospital.

“We have a culture that believes in very aggressive care,” said Dr. Redberg, who at the University of California–San Francisco specializes in heart disease in women. “We are often not considering the chance of benefit and chance of harm, and how that changes when you get older. We also fail to have conversations about what patients value most.”

While surgery is typically lifesaving for younger people, operating on frail, older patients rarely helps them live longer or returns the quality of life they once enjoyed, according to a 2016 paper in Annals of Surgery.

The cost of these surgeries – typically paid for by Medicare, the government health insurance program for people over 65 – involve more than money, said Amber Barnato, MD, professor at the Dartmouth Institute for Health Policy and Clinical Practice. Older patients who undergo surgery within a year of death spent 50 percent more time in the hospital than others, and nearly twice as many days in intensive care.

And while some robust octogenarians have many years ahead of them, studies show that surgery is also common among those who are far more frail.

Eighteen percent of Medicare patients have surgery in their final month of life and 8% in their final week, according to a 2011 study in The Lancet.

More than 12% of defibrillators were implanted in people older than 80, according to a 2015 study. Doctors implant about 158,000 of the devices each year, according to the American College of Cardiology. The total cost of the procedure runs about $60,000.

Procedures performed in the elderly range from major operations that require lengthy recoveries to relatively minor surgery performed in a doctor’s office, such as the removal of nonfatal skin cancers, that would likely never cause any problems.

Research led by Eleni Linos, MD, has shown that people with limited life expectancies are treated for nonfatal skin cancers as aggressively as younger patients. Among patients with a nonfatal skin cancer and a limited time to live, 70 percent underwent surgery, according to her 2013 study in JAMA Internal Medicine.

When less is more

Surgery poses serious risks for older people, who weather anesthesia poorly and whose skin takes longer to heal. Among seniors who undergo urgent or emergency abdominal surgery, 20% die within 30 days, studies show.

With diminished mental acuity and an old-fashioned respect for the medical profession, some aging patients are vulnerable to unwanted interventions. Stanich agreed to a pacemaker simply because her doctor suggested it, Giaquinto said. Many people of Stanich’s generation “thought doctors were God … They never questioned doctors – ever.”

Margaret Schwarze, MD, a surgeon and associate professor at the University of Wisconsin, said that older patients often don’t feel the financial pain of surgery because insurance pays most of the cost.

When a surgeon offers to “fix” the heart valve in a person with multiple diseases, for example, the patient may assume that surgery will fix all of her medical problems, Dr. Schwarze said. “With older patients with lots of chronic illnesses, we’re not really fixing anything.”

Even as a doctor, Dr. Redberg said, she struggles to prevent other doctors from performing too many procedures on her 92-year-old mother, Mae, who lives in New York City.

Dr. Redberg said doctors recently treated her mother for melanoma – the most serious type of skin cancer. After the cancer was removed from her leg, Dr. Redberg’s mother was urged by a doctor to undergo an additional surgery to cut away more tissue and nearby lymph nodes, which can harbor cancerous cells.

“Every time she went in, the dermatologist wanted to refer her to a surgeon,” Dr. Redberg said. And “Medicare would have been happy to pay for it.”

But her mother often has problems with wounds healing, she said, and recovery would likely have taken 3 months. When Dr. Redberg pressed a surgeon about the benefits, he said the procedure could reduce the chances of cancer coming back within 3-5 years.

Dr. Redberg said her mother laughed and said, “I’m not interested in doing something that will help me in 3-5 years. I doubt I’ll be here.”

Finding solutions

The momentum of hospital care can make people feel as if they’re on a moving train and can’t jump off.

The rush of medical decisions “doesn’t allow time to deliberate or consider the patients’ overall health or what their goals and values might be,” said Jacqueline Kruser, MD, an instructor in pulmonary and critical care medicine and medical social sciences at the Northwestern University.

Many hospitals and health systems are developing “decision aids,” easy-to-understand written materials and videos to help patients make more informed medical decisions, giving them time to develop more realistic expectations.

After Kaiser Permanente Washington introduced the tools relating to joint replacement, the number of patients choosing to have hip replacement surgery fell 26%, while knee replacements declined 38%, according to a study in Health Affairs. (Kaiser Permanente is not affiliated with Kaiser Health News, which is an editorially independent program of the Kaiser Family Foundation.)

In a paper published last year in JAMA Surgery and the Journal of Pain and Symptom Management, Dr. Schwarze, Dr. Kruser and colleagues suggested creating narratives to illustrate surgical risks, rather than relying on statistics.

Instead of telling patients that surgery carries a 20% risk of stroke, for example, doctors should lay out the best, worst and most likely outcomes.

In the best-case scenario, a patient might spend weeks in the hospital after surgery, living the rest of her life in a nursing home. In the worst case, the same patient dies after several weeks in intensive care. In the most likely scenario, the patient survives just 2-3 months after surgery.

Dr. Schwarze said, “If someone says they can’t tolerate the best-case scenario – which involves them being in a nursing home – then maybe we shouldn’t be doing this.”

Maxine Stanich was admitted to the hospital after going to the ER because she felt short of breath. She experienced an abnormal heart rhythm in the procedure room during a cardiac test – not an unusual event during a procedure in which a wire is threaded into the heart. Based on that, doctors decided to implant a pacemaker and defibrillator the next day.

Dr. Redberg was consulted when the patient objected to the device that was now embedded in her chest. She was “very alert. She was very clear about what she did and did not want done. She told me she didn’t want to be shocked,” Redberg said.

After Dr. Redberg deactivated the defibrillator, which can be reprogrammed remotely, Stanich was discharged, with home hospice service. With nothing more than her medicines, she survived another 2 years and 3 months, dying at home just after her 90th birthday in 2010.

Kaiser Health News is a nonprofit news service covering health issues. It is an editorially independent program of the Kaiser Family Foundation that is not affiliated with Kaiser Permanente. KHN’s coverage related to aging and improving care of older adults is supported in part by The John A. Hartford Foundation.

At 87, Maxine Stanich cared more about improving the quality of her life than prolonging it.

She suffered from a long list of health problems, including heart failure and chronic lung disease that could leave her gasping for breath.

When her time came, she wanted to die a natural death, Stanich told her daughter, and signed a “do not resuscitate” directive, or DNR, ordering doctors not to revive her should her heart stop.

Yet a trip to a San Francisco emergency room for shortness of breath in 2008 led Stanich to get a defibrillator implanted in her chest – a medical device to keep her alive by delivering a powerful shock. At the time, Stanich didn’t fully grasp what she had agreed to, even though she signed a document granting permission for the procedure, said her daughter, Susan Giaquinto.

That clarity came only during a subsequent visit to a different hospital, when a surprised ER doctor saw a defibrillator protruding from the DNR patient’s thin chest. To Stanich’s horror, the ER doctor explained that the device would not allow her to slip away painlessly and that the jolt would be “so strong that it will knock her across the room,” said Giaquinto, who accompanied her mother on both hospital trips.

Surgery like this has become all too common among those near the end of life, experts say. Nearly one in three Medicare patients undergo an operation in the year before they die, even though the evidence shows that many are more likely to be harmed than to benefit from it.

The practice is driven by financial incentives that reward doctors for doing procedures, as well as a medical culture in which patients and doctors are reluctant to talk about how surgical interventions should be prescribed more judiciously, said Rita Redberg, M.D., a cardiologist who treated Stanich when she sought care at the second hospital.

“We have a culture that believes in very aggressive care,” said Dr. Redberg, who at the University of California–San Francisco specializes in heart disease in women. “We are often not considering the chance of benefit and chance of harm, and how that changes when you get older. We also fail to have conversations about what patients value most.”

While surgery is typically lifesaving for younger people, operating on frail, older patients rarely helps them live longer or returns the quality of life they once enjoyed, according to a 2016 paper in Annals of Surgery.

The cost of these surgeries – typically paid for by Medicare, the government health insurance program for people over 65 – involve more than money, said Amber Barnato, MD, professor at the Dartmouth Institute for Health Policy and Clinical Practice. Older patients who undergo surgery within a year of death spent 50 percent more time in the hospital than others, and nearly twice as many days in intensive care.

And while some robust octogenarians have many years ahead of them, studies show that surgery is also common among those who are far more frail.

Eighteen percent of Medicare patients have surgery in their final month of life and 8% in their final week, according to a 2011 study in The Lancet.

More than 12% of defibrillators were implanted in people older than 80, according to a 2015 study. Doctors implant about 158,000 of the devices each year, according to the American College of Cardiology. The total cost of the procedure runs about $60,000.

Procedures performed in the elderly range from major operations that require lengthy recoveries to relatively minor surgery performed in a doctor’s office, such as the removal of nonfatal skin cancers, that would likely never cause any problems.

Research led by Eleni Linos, MD, has shown that people with limited life expectancies are treated for nonfatal skin cancers as aggressively as younger patients. Among patients with a nonfatal skin cancer and a limited time to live, 70 percent underwent surgery, according to her 2013 study in JAMA Internal Medicine.

When less is more

Surgery poses serious risks for older people, who weather anesthesia poorly and whose skin takes longer to heal. Among seniors who undergo urgent or emergency abdominal surgery, 20% die within 30 days, studies show.

With diminished mental acuity and an old-fashioned respect for the medical profession, some aging patients are vulnerable to unwanted interventions. Stanich agreed to a pacemaker simply because her doctor suggested it, Giaquinto said. Many people of Stanich’s generation “thought doctors were God … They never questioned doctors – ever.”

Margaret Schwarze, MD, a surgeon and associate professor at the University of Wisconsin, said that older patients often don’t feel the financial pain of surgery because insurance pays most of the cost.

When a surgeon offers to “fix” the heart valve in a person with multiple diseases, for example, the patient may assume that surgery will fix all of her medical problems, Dr. Schwarze said. “With older patients with lots of chronic illnesses, we’re not really fixing anything.”

Even as a doctor, Dr. Redberg said, she struggles to prevent other doctors from performing too many procedures on her 92-year-old mother, Mae, who lives in New York City.

Dr. Redberg said doctors recently treated her mother for melanoma – the most serious type of skin cancer. After the cancer was removed from her leg, Dr. Redberg’s mother was urged by a doctor to undergo an additional surgery to cut away more tissue and nearby lymph nodes, which can harbor cancerous cells.

“Every time she went in, the dermatologist wanted to refer her to a surgeon,” Dr. Redberg said. And “Medicare would have been happy to pay for it.”

But her mother often has problems with wounds healing, she said, and recovery would likely have taken 3 months. When Dr. Redberg pressed a surgeon about the benefits, he said the procedure could reduce the chances of cancer coming back within 3-5 years.

Dr. Redberg said her mother laughed and said, “I’m not interested in doing something that will help me in 3-5 years. I doubt I’ll be here.”

Finding solutions

The momentum of hospital care can make people feel as if they’re on a moving train and can’t jump off.

The rush of medical decisions “doesn’t allow time to deliberate or consider the patients’ overall health or what their goals and values might be,” said Jacqueline Kruser, MD, an instructor in pulmonary and critical care medicine and medical social sciences at the Northwestern University.

Many hospitals and health systems are developing “decision aids,” easy-to-understand written materials and videos to help patients make more informed medical decisions, giving them time to develop more realistic expectations.

After Kaiser Permanente Washington introduced the tools relating to joint replacement, the number of patients choosing to have hip replacement surgery fell 26%, while knee replacements declined 38%, according to a study in Health Affairs. (Kaiser Permanente is not affiliated with Kaiser Health News, which is an editorially independent program of the Kaiser Family Foundation.)

In a paper published last year in JAMA Surgery and the Journal of Pain and Symptom Management, Dr. Schwarze, Dr. Kruser and colleagues suggested creating narratives to illustrate surgical risks, rather than relying on statistics.

Instead of telling patients that surgery carries a 20% risk of stroke, for example, doctors should lay out the best, worst and most likely outcomes.

In the best-case scenario, a patient might spend weeks in the hospital after surgery, living the rest of her life in a nursing home. In the worst case, the same patient dies after several weeks in intensive care. In the most likely scenario, the patient survives just 2-3 months after surgery.

Dr. Schwarze said, “If someone says they can’t tolerate the best-case scenario – which involves them being in a nursing home – then maybe we shouldn’t be doing this.”

Maxine Stanich was admitted to the hospital after going to the ER because she felt short of breath. She experienced an abnormal heart rhythm in the procedure room during a cardiac test – not an unusual event during a procedure in which a wire is threaded into the heart. Based on that, doctors decided to implant a pacemaker and defibrillator the next day.

Dr. Redberg was consulted when the patient objected to the device that was now embedded in her chest. She was “very alert. She was very clear about what she did and did not want done. She told me she didn’t want to be shocked,” Redberg said.

After Dr. Redberg deactivated the defibrillator, which can be reprogrammed remotely, Stanich was discharged, with home hospice service. With nothing more than her medicines, she survived another 2 years and 3 months, dying at home just after her 90th birthday in 2010.

Kaiser Health News is a nonprofit news service covering health issues. It is an editorially independent program of the Kaiser Family Foundation that is not affiliated with Kaiser Permanente. KHN’s coverage related to aging and improving care of older adults is supported in part by The John A. Hartford Foundation.

Outrage over the high cost of cancer care has focused on skyrocketing drug prices, including the $475,000 price tag for the country’s first gene therapy, Novartis’ Kymriah (tisagenlecleucel), a leukemia treatment approved in August.

But the total costs of tisagenlecleucel and the 21 similar drugs in development – known as CAR T-cell therapies – will be far higher than many have imagined, reaching $1 million or more per patient, according to leading cancer experts. The next CAR T-cell drug could be approved as soon as November.

Although Kymriah’s price tag has “shattered oncology drug pricing norms,” said Leonard Saltz, MD, chief of gastrointestinal oncology at Memorial Sloan Kettering Cancer Center in New York, “the sticker price is just the starting point.”

These therapies lead to a cascade of costs, propelled by serious side effects that require sophisticated management, Dr. Saltz said. For this class of drugs, Dr, Saltz advised consumers to “think of the $475,000 as parts, not labor.”

Hagop Kantarjian, MD, leukemia specialist and professor at the University of Texas MD Anderson Cancer Center, estimates tisagenlecleucel’s total cost could reach $1.5 million.

CAR T-cell therapy is expensive because of the unique way that it works. Doctors harvest patients’ immune cells, genetically alter them to rev up their ability to fight cancer, then reinfuse them into patients.

Taking the brakes off the immune system, Dr. Kantarjian said, can lead to life-threatening complications that require lengthy hospitalizations and expensive medications, which are prescribed in addition to conventional cancer therapy, rather than in place of it.

Keith D. Eaton, MD, a Seattle oncologist, said he ran up medical bills of $500,000 when he participated in a clinical trial of CAR T cells in 2013, even though all patients in the study received the medication for free. Dr. Eaton, who was diagnosed with acute lymphoblastic leukemia (ALL), spent nearly 2 months in the hospital.

Like Dr. Eaton, nearly half of patients who receive CAR T cells develop cytokine storm. Other serious side effects include strokelike symptoms and coma.

The cytokine storm felt like “the worst flu of your life,” said Dr. Eaton, now aged 51 years. His fever spiked so high that a hospital nurse assumed the thermometer was broken. Dr. Eaton replied, “It’s not broken. My temperature is too high to register on the thermometer.”

Although Dr. Eaton recovered, he wasn’t done with treatment. His doctors recommended a bone marrow transplant, another harrowing procedure, at a cost of hundreds of thousands of dollars.

Dr. Eaton said he feels fortunate to be healthy today, with tests showing no evidence of leukemia. His insurer paid for almost everything.

Kymriah’s sticker price is especially “outrageous” given its relatively low manufacturing costs, said Walid F. Gellad, MD, codirector of the Center for Pharmaceutical Policy and Prescribing at the University of Pittsburgh.

The gene therapy process used to create tisagenlecleucel costs about $15,000, according to a 2012 presentation by Carl H. June, MD, who pioneered CAR T-cell research at the University of Pennsylvania in Philadelphia. Dr. June could not be reached for comment.

To quell unrest about price, Novartis has offered patients and insurers a new twist on the money-back guarantee.

Novartis will charge for the drug only if patients go into remission within 1 month of treatment. In a key clinical trial, 83% of the children and young adults treated with tisagenlecleucel went into remission within 3 months. Novartis calls the plan “outcomes-based pricing.”

Novartis is “working through the specific details” of how the pricing plan will affect the Centers for Medicare & Medicaid Services, which pays for care for many cancer patients, company spokesperson Julie Masow said. “There are many hurdles” to this type of pricing plan but, Ms. Masow said, “Novartis is committed to making this happen.”

She also said that Kymriah’s manufacturing costs are much higher than $15,000, although she didn’t cite a specific dollar amount. She noted that Novartis has invested heavily in the technology, designing “an innovative manufacturing facility and process specifically for cellular therapies.”

As for Kymriah-related hospital and medication charges, “costs will vary from patient to patient and treatment center to treatment center, based on the level of care each patient requires,” Ms. Masow said. “Kymriah is a one-time treatment that has shown remarkable early, deep, and durable responses in these children who are very sick and often out of options.”

Some doctors said tisagenlecleucel, which could be used by about 600 patients a year, offers an incalculable benefit for desperately ill young people. The drug is approved for children and young adults with B-cell ALL who already have been treated with at least two other cancer therapies.

“A kid’s life is priceless,” said Michelle Hermiston, MD, director of pediatric immunotherapy at Benioff Children’s Hospital, at the University of California, San Francisco. “Any given kid has the potential to make financial impacts over a lifetime that far outweigh the cost of their cure. From this perspective, every child in my mind deserves the best curative therapy we can offer.”

Other cancer doctors say the Novartis plan is no bargain.

About 36% of patients who go into remission with tisagenlecleucel relapse within 1 year, said Vinay Prasad, MD, of Oregon Health & Science University, Portland. Many of these patients will need additional treatment, said Dr. Prasad, who wrote an editorial about tisagenlecleucel’s price Oct. 4 in Nature.

“If you’ve paid half a million dollars for drugs and half a million dollars for care, and a year later your cancer is back, is that a good deal?” asked Dr. Saltz, who cowrote a recent editorial on tisagenlecleucel’s price in JAMA.

Steve Miller, MD, chief medical officer for Express Scripts, said it would be more fair to judge Kymriah’s success after 6 months of treatment, rather than 1 month. Dr. Prasad goes even further. He said Novartis should issue refunds for any patient who relapses within 3 years.

A consumer-advocate group called Patients for Affordable Drugs also has said that tisagenlecleucel costs too much, given that the federal government spent more than $200 million over 2 decades to support the basic research into CAR T-cell therapy, long before Novartis bought the rights.

Rep. Lloyd Doggett (D-Texas) wrote a letter to the Medicare program’s director last month asking for details on how the Novartis payment deal will work.

“As Big Pharma continues to put price gouging before patient access, companies will point more and more proudly at their pricing agreements,” Rep. Doggett wrote. “But taxpayers deserve to know more about how these agreements will work – whether they will actually save the government money, defray these massive costs, and ensure that they can access lifesaving medications.”
 

KHN’s coverage related to aging & improving care of older adults is supported by The John A. Hartford Foundation. Kaiser Health News is a national health policy news service that is part of the nonpartisan Henry J. Kaiser Family Foundation.

Outrage over the high cost of cancer care has focused on skyrocketing drug prices, including the $475,000 price tag for the country’s first gene therapy, Novartis’ Kymriah (tisagenlecleucel), a leukemia treatment approved in August.

But the total costs of tisagenlecleucel and the 21 similar drugs in development – known as CAR T-cell therapies – will be far higher than many have imagined, reaching $1 million or more per patient, according to leading cancer experts. The next CAR T-cell drug could be approved as soon as November.

Although Kymriah’s price tag has “shattered oncology drug pricing norms,” said Leonard Saltz, MD, chief of gastrointestinal oncology at Memorial Sloan Kettering Cancer Center in New York, “the sticker price is just the starting point.”

These therapies lead to a cascade of costs, propelled by serious side effects that require sophisticated management, Dr. Saltz said. For this class of drugs, Dr, Saltz advised consumers to “think of the $475,000 as parts, not labor.”

Hagop Kantarjian, MD, leukemia specialist and professor at the University of Texas MD Anderson Cancer Center, estimates tisagenlecleucel’s total cost could reach $1.5 million.

CAR T-cell therapy is expensive because of the unique way that it works. Doctors harvest patients’ immune cells, genetically alter them to rev up their ability to fight cancer, then reinfuse them into patients.

Taking the brakes off the immune system, Dr. Kantarjian said, can lead to life-threatening complications that require lengthy hospitalizations and expensive medications, which are prescribed in addition to conventional cancer therapy, rather than in place of it.

Keith D. Eaton, MD, a Seattle oncologist, said he ran up medical bills of $500,000 when he participated in a clinical trial of CAR T cells in 2013, even though all patients in the study received the medication for free. Dr. Eaton, who was diagnosed with acute lymphoblastic leukemia (ALL), spent nearly 2 months in the hospital.

Like Dr. Eaton, nearly half of patients who receive CAR T cells develop cytokine storm. Other serious side effects include strokelike symptoms and coma.

The cytokine storm felt like “the worst flu of your life,” said Dr. Eaton, now aged 51 years. His fever spiked so high that a hospital nurse assumed the thermometer was broken. Dr. Eaton replied, “It’s not broken. My temperature is too high to register on the thermometer.”

Although Dr. Eaton recovered, he wasn’t done with treatment. His doctors recommended a bone marrow transplant, another harrowing procedure, at a cost of hundreds of thousands of dollars.

Dr. Eaton said he feels fortunate to be healthy today, with tests showing no evidence of leukemia. His insurer paid for almost everything.

Kymriah’s sticker price is especially “outrageous” given its relatively low manufacturing costs, said Walid F. Gellad, MD, codirector of the Center for Pharmaceutical Policy and Prescribing at the University of Pittsburgh.

The gene therapy process used to create tisagenlecleucel costs about $15,000, according to a 2012 presentation by Carl H. June, MD, who pioneered CAR T-cell research at the University of Pennsylvania in Philadelphia. Dr. June could not be reached for comment.

To quell unrest about price, Novartis has offered patients and insurers a new twist on the money-back guarantee.

Novartis will charge for the drug only if patients go into remission within 1 month of treatment. In a key clinical trial, 83% of the children and young adults treated with tisagenlecleucel went into remission within 3 months. Novartis calls the plan “outcomes-based pricing.”

Novartis is “working through the specific details” of how the pricing plan will affect the Centers for Medicare & Medicaid Services, which pays for care for many cancer patients, company spokesperson Julie Masow said. “There are many hurdles” to this type of pricing plan but, Ms. Masow said, “Novartis is committed to making this happen.”

She also said that Kymriah’s manufacturing costs are much higher than $15,000, although she didn’t cite a specific dollar amount. She noted that Novartis has invested heavily in the technology, designing “an innovative manufacturing facility and process specifically for cellular therapies.”

As for Kymriah-related hospital and medication charges, “costs will vary from patient to patient and treatment center to treatment center, based on the level of care each patient requires,” Ms. Masow said. “Kymriah is a one-time treatment that has shown remarkable early, deep, and durable responses in these children who are very sick and often out of options.”

Some doctors said tisagenlecleucel, which could be used by about 600 patients a year, offers an incalculable benefit for desperately ill young people. The drug is approved for children and young adults with B-cell ALL who already have been treated with at least two other cancer therapies.

“A kid’s life is priceless,” said Michelle Hermiston, MD, director of pediatric immunotherapy at Benioff Children’s Hospital, at the University of California, San Francisco. “Any given kid has the potential to make financial impacts over a lifetime that far outweigh the cost of their cure. From this perspective, every child in my mind deserves the best curative therapy we can offer.”

Other cancer doctors say the Novartis plan is no bargain.

About 36% of patients who go into remission with tisagenlecleucel relapse within 1 year, said Vinay Prasad, MD, of Oregon Health & Science University, Portland. Many of these patients will need additional treatment, said Dr. Prasad, who wrote an editorial about tisagenlecleucel’s price Oct. 4 in Nature.

“If you’ve paid half a million dollars for drugs and half a million dollars for care, and a year later your cancer is back, is that a good deal?” asked Dr. Saltz, who cowrote a recent editorial on tisagenlecleucel’s price in JAMA.

Steve Miller, MD, chief medical officer for Express Scripts, said it would be more fair to judge Kymriah’s success after 6 months of treatment, rather than 1 month. Dr. Prasad goes even further. He said Novartis should issue refunds for any patient who relapses within 3 years.

A consumer-advocate group called Patients for Affordable Drugs also has said that tisagenlecleucel costs too much, given that the federal government spent more than $200 million over 2 decades to support the basic research into CAR T-cell therapy, long before Novartis bought the rights.

Rep. Lloyd Doggett (D-Texas) wrote a letter to the Medicare program’s director last month asking for details on how the Novartis payment deal will work.

“As Big Pharma continues to put price gouging before patient access, companies will point more and more proudly at their pricing agreements,” Rep. Doggett wrote. “But taxpayers deserve to know more about how these agreements will work – whether they will actually save the government money, defray these massive costs, and ensure that they can access lifesaving medications.”
 

KHN’s coverage related to aging & improving care of older adults is supported by The John A. Hartford Foundation. Kaiser Health News is a national health policy news service that is part of the nonpartisan Henry J. Kaiser Family Foundation.

Outrage over the high cost of cancer care has focused on skyrocketing drug prices, including the $475,000 price tag for the country’s first gene therapy, Novartis’ Kymriah (tisagenlecleucel), a leukemia treatment approved in August.

But the total costs of tisagenlecleucel and the 21 similar drugs in development – known as CAR T-cell therapies – will be far higher than many have imagined, reaching $1 million or more per patient, according to leading cancer experts. The next CAR T-cell drug could be approved as soon as November.

Although Kymriah’s price tag has “shattered oncology drug pricing norms,” said Leonard Saltz, MD, chief of gastrointestinal oncology at Memorial Sloan Kettering Cancer Center in New York, “the sticker price is just the starting point.”

These therapies lead to a cascade of costs, propelled by serious side effects that require sophisticated management, Dr. Saltz said. For this class of drugs, Dr, Saltz advised consumers to “think of the $475,000 as parts, not labor.”

Hagop Kantarjian, MD, leukemia specialist and professor at the University of Texas MD Anderson Cancer Center, estimates tisagenlecleucel’s total cost could reach $1.5 million.

CAR T-cell therapy is expensive because of the unique way that it works. Doctors harvest patients’ immune cells, genetically alter them to rev up their ability to fight cancer, then reinfuse them into patients.

Taking the brakes off the immune system, Dr. Kantarjian said, can lead to life-threatening complications that require lengthy hospitalizations and expensive medications, which are prescribed in addition to conventional cancer therapy, rather than in place of it.

Keith D. Eaton, MD, a Seattle oncologist, said he ran up medical bills of $500,000 when he participated in a clinical trial of CAR T cells in 2013, even though all patients in the study received the medication for free. Dr. Eaton, who was diagnosed with acute lymphoblastic leukemia (ALL), spent nearly 2 months in the hospital.

Like Dr. Eaton, nearly half of patients who receive CAR T cells develop cytokine storm. Other serious side effects include strokelike symptoms and coma.

The cytokine storm felt like “the worst flu of your life,” said Dr. Eaton, now aged 51 years. His fever spiked so high that a hospital nurse assumed the thermometer was broken. Dr. Eaton replied, “It’s not broken. My temperature is too high to register on the thermometer.”

Although Dr. Eaton recovered, he wasn’t done with treatment. His doctors recommended a bone marrow transplant, another harrowing procedure, at a cost of hundreds of thousands of dollars.

Dr. Eaton said he feels fortunate to be healthy today, with tests showing no evidence of leukemia. His insurer paid for almost everything.

Kymriah’s sticker price is especially “outrageous” given its relatively low manufacturing costs, said Walid F. Gellad, MD, codirector of the Center for Pharmaceutical Policy and Prescribing at the University of Pittsburgh.

The gene therapy process used to create tisagenlecleucel costs about $15,000, according to a 2012 presentation by Carl H. June, MD, who pioneered CAR T-cell research at the University of Pennsylvania in Philadelphia. Dr. June could not be reached for comment.

To quell unrest about price, Novartis has offered patients and insurers a new twist on the money-back guarantee.

Novartis will charge for the drug only if patients go into remission within 1 month of treatment. In a key clinical trial, 83% of the children and young adults treated with tisagenlecleucel went into remission within 3 months. Novartis calls the plan “outcomes-based pricing.”

Novartis is “working through the specific details” of how the pricing plan will affect the Centers for Medicare & Medicaid Services, which pays for care for many cancer patients, company spokesperson Julie Masow said. “There are many hurdles” to this type of pricing plan but, Ms. Masow said, “Novartis is committed to making this happen.”

She also said that Kymriah’s manufacturing costs are much higher than $15,000, although she didn’t cite a specific dollar amount. She noted that Novartis has invested heavily in the technology, designing “an innovative manufacturing facility and process specifically for cellular therapies.”

As for Kymriah-related hospital and medication charges, “costs will vary from patient to patient and treatment center to treatment center, based on the level of care each patient requires,” Ms. Masow said. “Kymriah is a one-time treatment that has shown remarkable early, deep, and durable responses in these children who are very sick and often out of options.”

Some doctors said tisagenlecleucel, which could be used by about 600 patients a year, offers an incalculable benefit for desperately ill young people. The drug is approved for children and young adults with B-cell ALL who already have been treated with at least two other cancer therapies.

“A kid’s life is priceless,” said Michelle Hermiston, MD, director of pediatric immunotherapy at Benioff Children’s Hospital, at the University of California, San Francisco. “Any given kid has the potential to make financial impacts over a lifetime that far outweigh the cost of their cure. From this perspective, every child in my mind deserves the best curative therapy we can offer.”

Other cancer doctors say the Novartis plan is no bargain.

About 36% of patients who go into remission with tisagenlecleucel relapse within 1 year, said Vinay Prasad, MD, of Oregon Health & Science University, Portland. Many of these patients will need additional treatment, said Dr. Prasad, who wrote an editorial about tisagenlecleucel’s price Oct. 4 in Nature.

“If you’ve paid half a million dollars for drugs and half a million dollars for care, and a year later your cancer is back, is that a good deal?” asked Dr. Saltz, who cowrote a recent editorial on tisagenlecleucel’s price in JAMA.

Steve Miller, MD, chief medical officer for Express Scripts, said it would be more fair to judge Kymriah’s success after 6 months of treatment, rather than 1 month. Dr. Prasad goes even further. He said Novartis should issue refunds for any patient who relapses within 3 years.

A consumer-advocate group called Patients for Affordable Drugs also has said that tisagenlecleucel costs too much, given that the federal government spent more than $200 million over 2 decades to support the basic research into CAR T-cell therapy, long before Novartis bought the rights.

Rep. Lloyd Doggett (D-Texas) wrote a letter to the Medicare program’s director last month asking for details on how the Novartis payment deal will work.

“As Big Pharma continues to put price gouging before patient access, companies will point more and more proudly at their pricing agreements,” Rep. Doggett wrote. “But taxpayers deserve to know more about how these agreements will work – whether they will actually save the government money, defray these massive costs, and ensure that they can access lifesaving medications.”
 

KHN’s coverage related to aging & improving care of older adults is supported by The John A. Hartford Foundation. Kaiser Health News is a national health policy news service that is part of the nonpartisan Henry J. Kaiser Family Foundation.

When doctors talk about a new leukemia drug from Novartis, they ooze enthusiasm, using words like “breakthrough,” “revolutionary” and “a watershed moment.”

But when they think about how much the therapy is likely to cost, their tone turns alarmist.

“It’s going to cost a fortune,” said Ivan Borrello, MD, at Johns Hopkins Sidney Kimmel Comprehensive Cancer Center in Baltimore.

“From what we’re hearing, this will be a quantum leap more expensive than other cancer drugs,” said Leonard Saltz, MD, chief of gastrointestinal oncology at Memorial Sloan Kettering Cancer Center in New York.

Switzerland-based Novartis hasn’t announced a price for the medicine, but British health authorities have said a price of $649,000 for a one-time treatment would be justified given the significant benefits.

The cancer therapy was unanimously approved by a Food and Drug Administration advisory committee in July, and its approval seems all but certain.

The treatment, CTL019, belongs to a new class of medications called CAR T-cell therapies, which involve harvesting patients’ immune cells and genetically altering them to kill cancer. It’s been tested in patients whose leukemia has relapsed in spite of the best chemotherapy or a bone-marrow transplant.

The prognosis for these patients is normally bleak. But in a clinical trial, 83% of those treated with CAR T-cell therapy – described as a “living drug” because it derives from a patient’s own cells – have gone into remission.

CAR T cells have been successful only in a limited number of cancers, however, and are being suggested for use as a last resort when all else has failed. As a result, only a few hundred patients a year would be eligible for them, at least initially, said J. Leonard Lichtenfeld, MD, deputy chief medical officer for the American Cancer Society.

The FDA is scheduled to decide on approval by Oct. 3. The agency also is considering a CAR T-cell therapy from Kite Pharma.

A third company, Juno Therapeutics, halted the development of one its CAR T-cell therapies after five patients died from complications of the treatment.

Rather than wait for Novartis to announce a price, an advocacy group called Patients for Affordable Drugs has launched a preemptive strike, asking to meet with company officials to discuss a “fair” price for the therapy. The Novartis drug has the potential to be one of the most expensive drugs ever sold, said David Mitchell, the patients group’s president, who has been treated for multiple myeloma, a blood cancer, since 2010. (The Laura and John Arnold Foundation, which provides some funding for Kaiser Health News, supports Patients for Affordable Drugs.)

“Many people with cancer look forward with great hope to the potential of your new drug,” Mr. Mitchell wrote in a letter to Novartis. “But drugs don’t work if patients can’t afford them.”

Cancer drugs today routinely cost more than $100,000 a year. A combination therapy for melanoma sells for $250,000. Such prices are particularly outrageous, given that taxpayers fund many drugs’ early research, Mr. Mitchell said.

The federal government spent more than $200 million over 2 decades to support the basic research into CAR T-cell therapy, long before Novartis bought the rights.

The patients group urged Novartis to charge no more for the drug in the U.S. than in other developed countries.

Novartis has agreed to meet with the patients group. In a statement, Novartis said the company is “carefully considering the appropriate price for CTL019, taking into consideration the value that this treatment represents for patients, society and the healthcare system, both near-term and long-term.”

Novartis made a significant investment in CAR T-cell therapy, according to the statement.

“We employ hundreds of people around the world who work on CAR Ts, we are conducting ongoing U.S. and global clinical trials and have developed a sophisticated, FDA-validated manufacturing site and process for this personalized therapy.”

Soaring prices for cancer drugs have led many patients to cut back on treatment or skip pills, a recent Kaiser Health News analysis showed.

The effect of CAR T-cell therapies on overall health costs would initially be relatively small, because it would be used by relatively few people, Dr. Lichtenfeld said.

Health systems and insurers may struggle to pay for the treatment, however, if the FDA approves it for wider use, Dr. Lichtenfeld said. Researchers are studying CAR T cells in a number of cancers. So far, the technology seems more effective in blood cancers, such as leukemias and lymphomas.

Hidden costs could further add to patients’ financial burdens, Dr. Borrello said.

Beyond the cost of the procedure, patients would need to pay for traditional chemotherapy, which is given before CAR T-cell therapy to improve its odds of success. They would also have to foot the bill for travel and lodging to one of the 30-35 hospitals in the country equipped to provide the high-tech treatment, said Prakash Satwani, MD, a pediatric hematologist at New York-Presbyterian/Columbia University Medical Center, which plans to offer the therapy.

Because patients can develop life-threatening side effects weeks after the procedure, doctors will ask patients to stay within 2 hours of the hospital for up to a month. In New York, even budget hotels cost more than $200 a night – an expense not typically covered by insurance. Patients who develop a dangerous complication, in which the immune system overreacts and attacks vital organs, might need coverage for emergency room care, as well as lengthy stays in the ICU, Dr. Satwani said.

Doctors don’t yet know what the full range of long-term side effects will be. CAR T-cell therapies can damage healthy immune cells, including the cells that produce the antibodies that fight disease. Some patients will need long-term treatments with a product called intravenous immunoglobulin, which provides the antibodies that patients need to prevent infection, Dr. Lichtenfeld said.

Dr. Saltz, an oncologist who has long spoken out about high drug prices, said he applauded the patients group’s efforts. But he said he doubts their efforts will persuade Novartis to set a reasonably affordable price.

“I’m not optimistic that this will have much effect on the company,” said Dr. Saltz. “There’s no market pressure for the company to respond to.”

High drug prices don’t just hurt patients, Dr. Saltz said. They also drive up insurance premiums for everyone.

“They affect each and every one of us,” he said, “because these costs will be paid by anyone who has any kind of insurance coverage.”
 

KHN’s coverage of prescription drug development, costs and pricing is supported in part by the Laura and John Arnold Foundation. Kaiser Health News is a national health policy news service that is part of the nonpartisan Henry J. Kaiser Family Foundation.

When doctors talk about a new leukemia drug from Novartis, they ooze enthusiasm, using words like “breakthrough,” “revolutionary” and “a watershed moment.”

But when they think about how much the therapy is likely to cost, their tone turns alarmist.

“It’s going to cost a fortune,” said Ivan Borrello, MD, at Johns Hopkins Sidney Kimmel Comprehensive Cancer Center in Baltimore.

“From what we’re hearing, this will be a quantum leap more expensive than other cancer drugs,” said Leonard Saltz, MD, chief of gastrointestinal oncology at Memorial Sloan Kettering Cancer Center in New York.

Switzerland-based Novartis hasn’t announced a price for the medicine, but British health authorities have said a price of $649,000 for a one-time treatment would be justified given the significant benefits.

The cancer therapy was unanimously approved by a Food and Drug Administration advisory committee in July, and its approval seems all but certain.

The treatment, CTL019, belongs to a new class of medications called CAR T-cell therapies, which involve harvesting patients’ immune cells and genetically altering them to kill cancer. It’s been tested in patients whose leukemia has relapsed in spite of the best chemotherapy or a bone-marrow transplant.

The prognosis for these patients is normally bleak. But in a clinical trial, 83% of those treated with CAR T-cell therapy – described as a “living drug” because it derives from a patient’s own cells – have gone into remission.

CAR T cells have been successful only in a limited number of cancers, however, and are being suggested for use as a last resort when all else has failed. As a result, only a few hundred patients a year would be eligible for them, at least initially, said J. Leonard Lichtenfeld, MD, deputy chief medical officer for the American Cancer Society.

The FDA is scheduled to decide on approval by Oct. 3. The agency also is considering a CAR T-cell therapy from Kite Pharma.

A third company, Juno Therapeutics, halted the development of one its CAR T-cell therapies after five patients died from complications of the treatment.

Rather than wait for Novartis to announce a price, an advocacy group called Patients for Affordable Drugs has launched a preemptive strike, asking to meet with company officials to discuss a “fair” price for the therapy. The Novartis drug has the potential to be one of the most expensive drugs ever sold, said David Mitchell, the patients group’s president, who has been treated for multiple myeloma, a blood cancer, since 2010. (The Laura and John Arnold Foundation, which provides some funding for Kaiser Health News, supports Patients for Affordable Drugs.)

“Many people with cancer look forward with great hope to the potential of your new drug,” Mr. Mitchell wrote in a letter to Novartis. “But drugs don’t work if patients can’t afford them.”

Cancer drugs today routinely cost more than $100,000 a year. A combination therapy for melanoma sells for $250,000. Such prices are particularly outrageous, given that taxpayers fund many drugs’ early research, Mr. Mitchell said.

The federal government spent more than $200 million over 2 decades to support the basic research into CAR T-cell therapy, long before Novartis bought the rights.

The patients group urged Novartis to charge no more for the drug in the U.S. than in other developed countries.

Novartis has agreed to meet with the patients group. In a statement, Novartis said the company is “carefully considering the appropriate price for CTL019, taking into consideration the value that this treatment represents for patients, society and the healthcare system, both near-term and long-term.”

Novartis made a significant investment in CAR T-cell therapy, according to the statement.

“We employ hundreds of people around the world who work on CAR Ts, we are conducting ongoing U.S. and global clinical trials and have developed a sophisticated, FDA-validated manufacturing site and process for this personalized therapy.”

Soaring prices for cancer drugs have led many patients to cut back on treatment or skip pills, a recent Kaiser Health News analysis showed.

The effect of CAR T-cell therapies on overall health costs would initially be relatively small, because it would be used by relatively few people, Dr. Lichtenfeld said.

Health systems and insurers may struggle to pay for the treatment, however, if the FDA approves it for wider use, Dr. Lichtenfeld said. Researchers are studying CAR T cells in a number of cancers. So far, the technology seems more effective in blood cancers, such as leukemias and lymphomas.

Hidden costs could further add to patients’ financial burdens, Dr. Borrello said.

Beyond the cost of the procedure, patients would need to pay for traditional chemotherapy, which is given before CAR T-cell therapy to improve its odds of success. They would also have to foot the bill for travel and lodging to one of the 30-35 hospitals in the country equipped to provide the high-tech treatment, said Prakash Satwani, MD, a pediatric hematologist at New York-Presbyterian/Columbia University Medical Center, which plans to offer the therapy.

Because patients can develop life-threatening side effects weeks after the procedure, doctors will ask patients to stay within 2 hours of the hospital for up to a month. In New York, even budget hotels cost more than $200 a night – an expense not typically covered by insurance. Patients who develop a dangerous complication, in which the immune system overreacts and attacks vital organs, might need coverage for emergency room care, as well as lengthy stays in the ICU, Dr. Satwani said.

Doctors don’t yet know what the full range of long-term side effects will be. CAR T-cell therapies can damage healthy immune cells, including the cells that produce the antibodies that fight disease. Some patients will need long-term treatments with a product called intravenous immunoglobulin, which provides the antibodies that patients need to prevent infection, Dr. Lichtenfeld said.

Dr. Saltz, an oncologist who has long spoken out about high drug prices, said he applauded the patients group’s efforts. But he said he doubts their efforts will persuade Novartis to set a reasonably affordable price.

“I’m not optimistic that this will have much effect on the company,” said Dr. Saltz. “There’s no market pressure for the company to respond to.”

High drug prices don’t just hurt patients, Dr. Saltz said. They also drive up insurance premiums for everyone.

“They affect each and every one of us,” he said, “because these costs will be paid by anyone who has any kind of insurance coverage.”
 

KHN’s coverage of prescription drug development, costs and pricing is supported in part by the Laura and John Arnold Foundation. Kaiser Health News is a national health policy news service that is part of the nonpartisan Henry J. Kaiser Family Foundation.

When doctors talk about a new leukemia drug from Novartis, they ooze enthusiasm, using words like “breakthrough,” “revolutionary” and “a watershed moment.”

But when they think about how much the therapy is likely to cost, their tone turns alarmist.

“It’s going to cost a fortune,” said Ivan Borrello, MD, at Johns Hopkins Sidney Kimmel Comprehensive Cancer Center in Baltimore.

“From what we’re hearing, this will be a quantum leap more expensive than other cancer drugs,” said Leonard Saltz, MD, chief of gastrointestinal oncology at Memorial Sloan Kettering Cancer Center in New York.

Switzerland-based Novartis hasn’t announced a price for the medicine, but British health authorities have said a price of $649,000 for a one-time treatment would be justified given the significant benefits.

The cancer therapy was unanimously approved by a Food and Drug Administration advisory committee in July, and its approval seems all but certain.

The treatment, CTL019, belongs to a new class of medications called CAR T-cell therapies, which involve harvesting patients’ immune cells and genetically altering them to kill cancer. It’s been tested in patients whose leukemia has relapsed in spite of the best chemotherapy or a bone-marrow transplant.

The prognosis for these patients is normally bleak. But in a clinical trial, 83% of those treated with CAR T-cell therapy – described as a “living drug” because it derives from a patient’s own cells – have gone into remission.

CAR T cells have been successful only in a limited number of cancers, however, and are being suggested for use as a last resort when all else has failed. As a result, only a few hundred patients a year would be eligible for them, at least initially, said J. Leonard Lichtenfeld, MD, deputy chief medical officer for the American Cancer Society.

The FDA is scheduled to decide on approval by Oct. 3. The agency also is considering a CAR T-cell therapy from Kite Pharma.

A third company, Juno Therapeutics, halted the development of one its CAR T-cell therapies after five patients died from complications of the treatment.

Rather than wait for Novartis to announce a price, an advocacy group called Patients for Affordable Drugs has launched a preemptive strike, asking to meet with company officials to discuss a “fair” price for the therapy. The Novartis drug has the potential to be one of the most expensive drugs ever sold, said David Mitchell, the patients group’s president, who has been treated for multiple myeloma, a blood cancer, since 2010. (The Laura and John Arnold Foundation, which provides some funding for Kaiser Health News, supports Patients for Affordable Drugs.)

“Many people with cancer look forward with great hope to the potential of your new drug,” Mr. Mitchell wrote in a letter to Novartis. “But drugs don’t work if patients can’t afford them.”

Cancer drugs today routinely cost more than $100,000 a year. A combination therapy for melanoma sells for $250,000. Such prices are particularly outrageous, given that taxpayers fund many drugs’ early research, Mr. Mitchell said.

The federal government spent more than $200 million over 2 decades to support the basic research into CAR T-cell therapy, long before Novartis bought the rights.

The patients group urged Novartis to charge no more for the drug in the U.S. than in other developed countries.

Novartis has agreed to meet with the patients group. In a statement, Novartis said the company is “carefully considering the appropriate price for CTL019, taking into consideration the value that this treatment represents for patients, society and the healthcare system, both near-term and long-term.”

Novartis made a significant investment in CAR T-cell therapy, according to the statement.

“We employ hundreds of people around the world who work on CAR Ts, we are conducting ongoing U.S. and global clinical trials and have developed a sophisticated, FDA-validated manufacturing site and process for this personalized therapy.”

Soaring prices for cancer drugs have led many patients to cut back on treatment or skip pills, a recent Kaiser Health News analysis showed.

The effect of CAR T-cell therapies on overall health costs would initially be relatively small, because it would be used by relatively few people, Dr. Lichtenfeld said.

Health systems and insurers may struggle to pay for the treatment, however, if the FDA approves it for wider use, Dr. Lichtenfeld said. Researchers are studying CAR T cells in a number of cancers. So far, the technology seems more effective in blood cancers, such as leukemias and lymphomas.

Hidden costs could further add to patients’ financial burdens, Dr. Borrello said.

Beyond the cost of the procedure, patients would need to pay for traditional chemotherapy, which is given before CAR T-cell therapy to improve its odds of success. They would also have to foot the bill for travel and lodging to one of the 30-35 hospitals in the country equipped to provide the high-tech treatment, said Prakash Satwani, MD, a pediatric hematologist at New York-Presbyterian/Columbia University Medical Center, which plans to offer the therapy.

Because patients can develop life-threatening side effects weeks after the procedure, doctors will ask patients to stay within 2 hours of the hospital for up to a month. In New York, even budget hotels cost more than $200 a night – an expense not typically covered by insurance. Patients who develop a dangerous complication, in which the immune system overreacts and attacks vital organs, might need coverage for emergency room care, as well as lengthy stays in the ICU, Dr. Satwani said.

Doctors don’t yet know what the full range of long-term side effects will be. CAR T-cell therapies can damage healthy immune cells, including the cells that produce the antibodies that fight disease. Some patients will need long-term treatments with a product called intravenous immunoglobulin, which provides the antibodies that patients need to prevent infection, Dr. Lichtenfeld said.

Dr. Saltz, an oncologist who has long spoken out about high drug prices, said he applauded the patients group’s efforts. But he said he doubts their efforts will persuade Novartis to set a reasonably affordable price.

“I’m not optimistic that this will have much effect on the company,” said Dr. Saltz. “There’s no market pressure for the company to respond to.”

High drug prices don’t just hurt patients, Dr. Saltz said. They also drive up insurance premiums for everyone.

“They affect each and every one of us,” he said, “because these costs will be paid by anyone who has any kind of insurance coverage.”
 

KHN’s coverage of prescription drug development, costs and pricing is supported in part by the Laura and John Arnold Foundation. Kaiser Health News is a national health policy news service that is part of the nonpartisan Henry J. Kaiser Family Foundation.

Less and less of the research presented at a prominent cancer conference is supported by the National Institutes of Health, a development that some of the country’s top scientists see as a worrisome trend.

The number of studies fully funded by the NIH at the annual meeting of the American Society of Clinical Oncology (ASCO) – the world’s largest gathering of cancer researchers – has fallen 75% in the past decade, from 575 papers in 2008 to 144 this year, according to the society, which meets June 2-6 in Chicago.

American researchers typically dominate the meeting’s press conferences – designed to feature the most important and newsworthy research. This year, there are 14 studies led by international scientists versus 12 led by U.S.-based research teams. That’s a big shift from just 5 years ago, when 15 studies in the “press program” were led by Americans versus 9 by international researchers.

Several of the studies on this weekend’s press program come from Europe and Canada, along with two from China.

President Donald Trump has proposed cutting the NIH budget for 2018 from $31.8 billion to $26 billion, a decline that many worry would jeopardize the fight against cancer and other diseases. Those cuts include $1 billion less for the National Cancer Institute.

On its website, the NCI notes that its purchasing power already has declined by 25% since 2003, because its budget – while growing – hasn’t kept up with inflation. Congress gave the NCI nearly $5.4 billion in fiscal year 2017, an increase of $174.6 million over last year. The NCI also received $300 million for the Beau Biden Cancer Moonshot through the 21st Century Cures Act in December 2016.

“America may be losing its edge in medical research,” said Otis Brawley, MD, chief medical officer at the American Cancer Society. The brightest young scientists are having trouble finding funding for their research, leading them to look for jobs not at universities but at drug companies “or even Wall Street,” he said. “I fear we are losing a generation of young, talented biomedical scientists.”

Some see America’s leading role in science as a point of national pride.

“Do we want the U.S. to remain at the center of biomedical innovation, or do we want to cede that to China or other countries?” said Dr. Stephan Grupp, director of the Cancer Immunotherapy Frontier Program at Children’s Hospital of Philadelphia. “If you don’t push to stay in front, you don’t stay in front.”

But more than pride is at stake.

Public funding is critical, because it allows researchers to answer questions that don’t interest drug companies, said Richard Schilsky, senior vice president and chief medical officer at ASCO.

While drug companies fund studies that help them get their medications approved, they tend not to pay for studies that focus on cancer prevention, screening, or quality of life, Mr. Schilsky said. The NIH also funds head-to-head comparisons of cancer drugs, which allow patients and doctors to select the most effective treatments.

“If the NIH-funded studies continue to decline, we simply won’t get the answers that patients are looking for,” he said.

While government research often addresses areas of greatest need, “industry research is geared toward marketable products,” Dr. Brawley said.

To help make up the deficit, the American Cancer Society will double its research budget to $240 million by 2021, he added.

But Dr. Grupp notes that charities and the drug industry are often reluctant to cover the indirect costs of research, such as labs. Without steady, predictable support from government grants, Dr. Grupp said he wouldn’t “have a building to do my research in or a way to keep the lights on.”


 

Kaiser Health News is a national health policy news service that is part of the nonpartisan Henry J. Kaiser Family Foundation.

Less and less of the research presented at a prominent cancer conference is supported by the National Institutes of Health, a development that some of the country’s top scientists see as a worrisome trend.

The number of studies fully funded by the NIH at the annual meeting of the American Society of Clinical Oncology (ASCO) – the world’s largest gathering of cancer researchers – has fallen 75% in the past decade, from 575 papers in 2008 to 144 this year, according to the society, which meets June 2-6 in Chicago.

American researchers typically dominate the meeting’s press conferences – designed to feature the most important and newsworthy research. This year, there are 14 studies led by international scientists versus 12 led by U.S.-based research teams. That’s a big shift from just 5 years ago, when 15 studies in the “press program” were led by Americans versus 9 by international researchers.

Several of the studies on this weekend’s press program come from Europe and Canada, along with two from China.

President Donald Trump has proposed cutting the NIH budget for 2018 from $31.8 billion to $26 billion, a decline that many worry would jeopardize the fight against cancer and other diseases. Those cuts include $1 billion less for the National Cancer Institute.

On its website, the NCI notes that its purchasing power already has declined by 25% since 2003, because its budget – while growing – hasn’t kept up with inflation. Congress gave the NCI nearly $5.4 billion in fiscal year 2017, an increase of $174.6 million over last year. The NCI also received $300 million for the Beau Biden Cancer Moonshot through the 21st Century Cures Act in December 2016.

“America may be losing its edge in medical research,” said Otis Brawley, MD, chief medical officer at the American Cancer Society. The brightest young scientists are having trouble finding funding for their research, leading them to look for jobs not at universities but at drug companies “or even Wall Street,” he said. “I fear we are losing a generation of young, talented biomedical scientists.”

Some see America’s leading role in science as a point of national pride.

“Do we want the U.S. to remain at the center of biomedical innovation, or do we want to cede that to China or other countries?” said Dr. Stephan Grupp, director of the Cancer Immunotherapy Frontier Program at Children’s Hospital of Philadelphia. “If you don’t push to stay in front, you don’t stay in front.”

But more than pride is at stake.

Public funding is critical, because it allows researchers to answer questions that don’t interest drug companies, said Richard Schilsky, senior vice president and chief medical officer at ASCO.

While drug companies fund studies that help them get their medications approved, they tend not to pay for studies that focus on cancer prevention, screening, or quality of life, Mr. Schilsky said. The NIH also funds head-to-head comparisons of cancer drugs, which allow patients and doctors to select the most effective treatments.

“If the NIH-funded studies continue to decline, we simply won’t get the answers that patients are looking for,” he said.

While government research often addresses areas of greatest need, “industry research is geared toward marketable products,” Dr. Brawley said.

To help make up the deficit, the American Cancer Society will double its research budget to $240 million by 2021, he added.

But Dr. Grupp notes that charities and the drug industry are often reluctant to cover the indirect costs of research, such as labs. Without steady, predictable support from government grants, Dr. Grupp said he wouldn’t “have a building to do my research in or a way to keep the lights on.”


 

Kaiser Health News is a national health policy news service that is part of the nonpartisan Henry J. Kaiser Family Foundation.

Less and less of the research presented at a prominent cancer conference is supported by the National Institutes of Health, a development that some of the country’s top scientists see as a worrisome trend.

The number of studies fully funded by the NIH at the annual meeting of the American Society of Clinical Oncology (ASCO) – the world’s largest gathering of cancer researchers – has fallen 75% in the past decade, from 575 papers in 2008 to 144 this year, according to the society, which meets June 2-6 in Chicago.

American researchers typically dominate the meeting’s press conferences – designed to feature the most important and newsworthy research. This year, there are 14 studies led by international scientists versus 12 led by U.S.-based research teams. That’s a big shift from just 5 years ago, when 15 studies in the “press program” were led by Americans versus 9 by international researchers.

Several of the studies on this weekend’s press program come from Europe and Canada, along with two from China.

President Donald Trump has proposed cutting the NIH budget for 2018 from $31.8 billion to $26 billion, a decline that many worry would jeopardize the fight against cancer and other diseases. Those cuts include $1 billion less for the National Cancer Institute.

On its website, the NCI notes that its purchasing power already has declined by 25% since 2003, because its budget – while growing – hasn’t kept up with inflation. Congress gave the NCI nearly $5.4 billion in fiscal year 2017, an increase of $174.6 million over last year. The NCI also received $300 million for the Beau Biden Cancer Moonshot through the 21st Century Cures Act in December 2016.

“America may be losing its edge in medical research,” said Otis Brawley, MD, chief medical officer at the American Cancer Society. The brightest young scientists are having trouble finding funding for their research, leading them to look for jobs not at universities but at drug companies “or even Wall Street,” he said. “I fear we are losing a generation of young, talented biomedical scientists.”

Some see America’s leading role in science as a point of national pride.

“Do we want the U.S. to remain at the center of biomedical innovation, or do we want to cede that to China or other countries?” said Dr. Stephan Grupp, director of the Cancer Immunotherapy Frontier Program at Children’s Hospital of Philadelphia. “If you don’t push to stay in front, you don’t stay in front.”

But more than pride is at stake.

Public funding is critical, because it allows researchers to answer questions that don’t interest drug companies, said Richard Schilsky, senior vice president and chief medical officer at ASCO.

While drug companies fund studies that help them get their medications approved, they tend not to pay for studies that focus on cancer prevention, screening, or quality of life, Mr. Schilsky said. The NIH also funds head-to-head comparisons of cancer drugs, which allow patients and doctors to select the most effective treatments.

“If the NIH-funded studies continue to decline, we simply won’t get the answers that patients are looking for,” he said.

While government research often addresses areas of greatest need, “industry research is geared toward marketable products,” Dr. Brawley said.

To help make up the deficit, the American Cancer Society will double its research budget to $240 million by 2021, he added.

But Dr. Grupp notes that charities and the drug industry are often reluctant to cover the indirect costs of research, such as labs. Without steady, predictable support from government grants, Dr. Grupp said he wouldn’t “have a building to do my research in or a way to keep the lights on.”


 

Kaiser Health News is a national health policy news service that is part of the nonpartisan Henry J. Kaiser Family Foundation.