How racist is your algorithm?

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Every time Nathan Chomilo, MD, uses a clinical decision support tool, he tells his patients they have a choice: He can input their race or keep that field blank.

Until recently, many clinicians didn’t question the use of race as a datapoint in tools used to make decisions about diagnosis and care. But that is changing.

“I’ve almost universally had patients appreciate that someone actually told them that their kidney function was being scored differently because of the color of their skin or how they were identified in the medical chart along lines of race,” Dr. Chomilo, an adjunct assistant professor of pediatrics at the University of Minnesota Medical School, Minneapolis, said.

Dr. Chomilo is referring to the estimated glomerular filtration rate (eGFR), which combines results from a blood test with factors such as age, sex, and race to calculate kidney function.

The eGFR weighed an input of “African American” as automatically indicating a higher concentration of serum creatinine than a non African American patient on the basis of the unsubstantiated idea that Black people have more creatinine in their blood at baseline.

The calculator creates a picture of a Black patient who is not as sick as a White patient with the same levels of kidney failure. But race is based on the color of a patient’s skin, not on genetics or other clinical datapoints.

“I often use my own example of being a biracial Black man: My father’s family is from Cameroon, my mother’s family is from Norway. Are you going to assign my kidneys or my lungs to my mom’s side or my dad’s side? That’s not clear at all in the way we use race in medicine,” Dr. Chomilo, an executive committee member on the section on minority health equity and inclusion at the American Academy of Pediatrics (AAP), said.

Long before the COVID-19 pandemic so publicly exposed the depths of inequality in morbidity and mortality in the United States, health advocates had been pointing out these disparities in tools used by medical professionals. But efforts to recognize that race is a poor proxy for genetics is in its infancy.

In May, the AAP published a policy statement that kicked off its examination of clinical guidelines and policies that include race as a biological proxy. A committee for the society is combing through each guideline or calculator, evaluating the scientific basis for the use of race, and examining whether a stronger datapoint could be used instead.

The eGFR is perhaps the best example of a calculator that’s gone through the process: Health care stakeholders questioned the use of race, and investigators went back to study whether race was really a good datapoint. It wasn’t, and Dr. Chamilo’s hospital joined many others in retiring the calculator.

But the eGFR is one of countless clinical tools – from rudimentary algorithms to sophisticated machine-learning instruments – that change the course of care in part on the basis of race in the same way datapoints such as weight, age, and height are used to inform decisions about patient management. But unlike race, height, weight, and age can be objectively measured. A physician either makes a guess, or a patient enters their race on a form. And while that can be useful on a population level, race does not equal genetics or any other measurable datapoint.

In a study published in JAMA Pediatrics, researchers reviewed 414 clinical practice guidelines from sources such as PubMed and MetaLib.gov. Almost 1 in 6 guidelines included race in an inappropriate way, such as by conflating race as a biological risk factor or establishing testing or treatment thresholds using race.
 

 

 

Waiting for alternatives

The University of Maryland Medical System last year embarked on a project similar to the AAP initiative but within its own system. The first use of race to be eliminated was in the eGFR. The health system also recently removed the variable from a tool for diagnosing urinary tract infections (UTIs) in children younger than 2 years.

Part of that tool includes deciding to perform a catheterized urine test. If a doctor chose “White” as the race, the tool would recommend the test. If the doctor chose “Black,” the tool would recommend to not test. Joseph Wright, MD, MPH, chief health equity officer at University of Maryland Medical System, said this step in the tool is based on the unproven assumption that young Black children had a lower likelihood of UTIs than their White peers.

“We simply want folks to not by default lob race in as a decisionmaking point when we have, with a little bit more scientific diligence, the ability to include better clinical variables,” Dr. Wright, who is also an adjunct professor of health policy and management at the University of Maryland School of Public Health, College Park, said.

The developers of the UTI tool recently released a revised version that removes race in favor of two new medical datapoints: whether the patient has had a fever for over 48 hours, and whether the patient has previously had a UTI.

The process of re-examining tools, coming up with new datapoints, and implementing changes is not simple, according to Dr. Wright.

“This is just the baby step to fix the algorithms, because we’re all going to have to examine our own house, where these calculators live, whether it’s in a textbook, whether it’s in an electronic health record, and that’s the heavy lift,” he said. “All sources of clinical guidance have to be scrutinized, and it’s going to literally take years to unroot.”

Electronic medical record vendor Cerner said it generally revises its algorithms after medical societies make changes, then communicates those fixes to providers.

Rebecca C. Winokur, MD, MPH, lead physician executive and health equity service line leader at Cerner, explained that if doctors ordered an eGFR a year ago and then another today, the results might be different because of the new code that eliminates race.

“The numbers are so different, how do you know that the patient may or may not have the same function?” Dr. Winokur said.

Dr. Winokur said the company is trying to determine at which point a message should pop up in the records workflow that would inform clinicians that they may be comparing apples to cherries. The company also is reconsidering the use of race in tools that estimate the probability of a successful vaginal birth after prior cesarean delivery, a calculator that predicts the risk of urethral stones in patients with flank pain, and another that measures lung function to help diagnose pulmonary disease.

In addition to managing the logistics of removing race, health institutions also need buy-in from clinicians. At Mass General Brigham, Boston, Thomas Sequist, MD, MPH, chief medical officer, is leading a project to examine how the system uses race in calculators.

“People struggle mainly with, well, if we shouldn’t use this calculator, what should we use, because we need a calculator. And that’s a legitimate question,” Dr. Sequist said in an interview. “If we’re going to stop using this race-based calculator, I still need to know what dose of medication I give my patient. We’re not going to pull any of these calculators until we have a safe and reliable alternative.”

For each calculator, relevant specialty chiefs come to the table with Dr. Sequist and his team; current projects include examining bone density screenings and cardiac risk scores. A large part of the work is communicating the lack of science behind the inclusion of race as a variable.

“It’s hard because these tools have been in existence for decades, and people are used to using them,” Dr. Sequist said. “So this is a big-change management project.”

Some clinicians also have difficulty discerning why their health system may stratify patient outcomes by race while providers are being told that race is being removed from the calculators they use every day. The key difference is that stratifying outcomes by race illuminates systemic problems that can be targeted by a health system.

For instance, if readmission rates are higher for Black patients overall after surgery, the reason might be that nurses are not delivering the same level of care to them as they are to non-Black patients, possibly because of hidden bias. Or, perhaps Black patients at a hospital have less access to transportation for follow-up appointments after surgery. The potential reasons can be investigated, and solutions can be created.

“If you look at a population level, what you’re looking for is not for the evidence of race as a biological construct,” Dr. Chomilo said. “You’re looking for the impact of racism on populations, and that’s the difference: It’s racism, not race.”

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

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Every time Nathan Chomilo, MD, uses a clinical decision support tool, he tells his patients they have a choice: He can input their race or keep that field blank.

Until recently, many clinicians didn’t question the use of race as a datapoint in tools used to make decisions about diagnosis and care. But that is changing.

“I’ve almost universally had patients appreciate that someone actually told them that their kidney function was being scored differently because of the color of their skin or how they were identified in the medical chart along lines of race,” Dr. Chomilo, an adjunct assistant professor of pediatrics at the University of Minnesota Medical School, Minneapolis, said.

Dr. Chomilo is referring to the estimated glomerular filtration rate (eGFR), which combines results from a blood test with factors such as age, sex, and race to calculate kidney function.

The eGFR weighed an input of “African American” as automatically indicating a higher concentration of serum creatinine than a non African American patient on the basis of the unsubstantiated idea that Black people have more creatinine in their blood at baseline.

The calculator creates a picture of a Black patient who is not as sick as a White patient with the same levels of kidney failure. But race is based on the color of a patient’s skin, not on genetics or other clinical datapoints.

“I often use my own example of being a biracial Black man: My father’s family is from Cameroon, my mother’s family is from Norway. Are you going to assign my kidneys or my lungs to my mom’s side or my dad’s side? That’s not clear at all in the way we use race in medicine,” Dr. Chomilo, an executive committee member on the section on minority health equity and inclusion at the American Academy of Pediatrics (AAP), said.

Long before the COVID-19 pandemic so publicly exposed the depths of inequality in morbidity and mortality in the United States, health advocates had been pointing out these disparities in tools used by medical professionals. But efforts to recognize that race is a poor proxy for genetics is in its infancy.

In May, the AAP published a policy statement that kicked off its examination of clinical guidelines and policies that include race as a biological proxy. A committee for the society is combing through each guideline or calculator, evaluating the scientific basis for the use of race, and examining whether a stronger datapoint could be used instead.

The eGFR is perhaps the best example of a calculator that’s gone through the process: Health care stakeholders questioned the use of race, and investigators went back to study whether race was really a good datapoint. It wasn’t, and Dr. Chamilo’s hospital joined many others in retiring the calculator.

But the eGFR is one of countless clinical tools – from rudimentary algorithms to sophisticated machine-learning instruments – that change the course of care in part on the basis of race in the same way datapoints such as weight, age, and height are used to inform decisions about patient management. But unlike race, height, weight, and age can be objectively measured. A physician either makes a guess, or a patient enters their race on a form. And while that can be useful on a population level, race does not equal genetics or any other measurable datapoint.

In a study published in JAMA Pediatrics, researchers reviewed 414 clinical practice guidelines from sources such as PubMed and MetaLib.gov. Almost 1 in 6 guidelines included race in an inappropriate way, such as by conflating race as a biological risk factor or establishing testing or treatment thresholds using race.
 

 

 

Waiting for alternatives

The University of Maryland Medical System last year embarked on a project similar to the AAP initiative but within its own system. The first use of race to be eliminated was in the eGFR. The health system also recently removed the variable from a tool for diagnosing urinary tract infections (UTIs) in children younger than 2 years.

Part of that tool includes deciding to perform a catheterized urine test. If a doctor chose “White” as the race, the tool would recommend the test. If the doctor chose “Black,” the tool would recommend to not test. Joseph Wright, MD, MPH, chief health equity officer at University of Maryland Medical System, said this step in the tool is based on the unproven assumption that young Black children had a lower likelihood of UTIs than their White peers.

“We simply want folks to not by default lob race in as a decisionmaking point when we have, with a little bit more scientific diligence, the ability to include better clinical variables,” Dr. Wright, who is also an adjunct professor of health policy and management at the University of Maryland School of Public Health, College Park, said.

The developers of the UTI tool recently released a revised version that removes race in favor of two new medical datapoints: whether the patient has had a fever for over 48 hours, and whether the patient has previously had a UTI.

The process of re-examining tools, coming up with new datapoints, and implementing changes is not simple, according to Dr. Wright.

“This is just the baby step to fix the algorithms, because we’re all going to have to examine our own house, where these calculators live, whether it’s in a textbook, whether it’s in an electronic health record, and that’s the heavy lift,” he said. “All sources of clinical guidance have to be scrutinized, and it’s going to literally take years to unroot.”

Electronic medical record vendor Cerner said it generally revises its algorithms after medical societies make changes, then communicates those fixes to providers.

Rebecca C. Winokur, MD, MPH, lead physician executive and health equity service line leader at Cerner, explained that if doctors ordered an eGFR a year ago and then another today, the results might be different because of the new code that eliminates race.

“The numbers are so different, how do you know that the patient may or may not have the same function?” Dr. Winokur said.

Dr. Winokur said the company is trying to determine at which point a message should pop up in the records workflow that would inform clinicians that they may be comparing apples to cherries. The company also is reconsidering the use of race in tools that estimate the probability of a successful vaginal birth after prior cesarean delivery, a calculator that predicts the risk of urethral stones in patients with flank pain, and another that measures lung function to help diagnose pulmonary disease.

In addition to managing the logistics of removing race, health institutions also need buy-in from clinicians. At Mass General Brigham, Boston, Thomas Sequist, MD, MPH, chief medical officer, is leading a project to examine how the system uses race in calculators.

“People struggle mainly with, well, if we shouldn’t use this calculator, what should we use, because we need a calculator. And that’s a legitimate question,” Dr. Sequist said in an interview. “If we’re going to stop using this race-based calculator, I still need to know what dose of medication I give my patient. We’re not going to pull any of these calculators until we have a safe and reliable alternative.”

For each calculator, relevant specialty chiefs come to the table with Dr. Sequist and his team; current projects include examining bone density screenings and cardiac risk scores. A large part of the work is communicating the lack of science behind the inclusion of race as a variable.

“It’s hard because these tools have been in existence for decades, and people are used to using them,” Dr. Sequist said. “So this is a big-change management project.”

Some clinicians also have difficulty discerning why their health system may stratify patient outcomes by race while providers are being told that race is being removed from the calculators they use every day. The key difference is that stratifying outcomes by race illuminates systemic problems that can be targeted by a health system.

For instance, if readmission rates are higher for Black patients overall after surgery, the reason might be that nurses are not delivering the same level of care to them as they are to non-Black patients, possibly because of hidden bias. Or, perhaps Black patients at a hospital have less access to transportation for follow-up appointments after surgery. The potential reasons can be investigated, and solutions can be created.

“If you look at a population level, what you’re looking for is not for the evidence of race as a biological construct,” Dr. Chomilo said. “You’re looking for the impact of racism on populations, and that’s the difference: It’s racism, not race.”

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

Every time Nathan Chomilo, MD, uses a clinical decision support tool, he tells his patients they have a choice: He can input their race or keep that field blank.

Until recently, many clinicians didn’t question the use of race as a datapoint in tools used to make decisions about diagnosis and care. But that is changing.

“I’ve almost universally had patients appreciate that someone actually told them that their kidney function was being scored differently because of the color of their skin or how they were identified in the medical chart along lines of race,” Dr. Chomilo, an adjunct assistant professor of pediatrics at the University of Minnesota Medical School, Minneapolis, said.

Dr. Chomilo is referring to the estimated glomerular filtration rate (eGFR), which combines results from a blood test with factors such as age, sex, and race to calculate kidney function.

The eGFR weighed an input of “African American” as automatically indicating a higher concentration of serum creatinine than a non African American patient on the basis of the unsubstantiated idea that Black people have more creatinine in their blood at baseline.

The calculator creates a picture of a Black patient who is not as sick as a White patient with the same levels of kidney failure. But race is based on the color of a patient’s skin, not on genetics or other clinical datapoints.

“I often use my own example of being a biracial Black man: My father’s family is from Cameroon, my mother’s family is from Norway. Are you going to assign my kidneys or my lungs to my mom’s side or my dad’s side? That’s not clear at all in the way we use race in medicine,” Dr. Chomilo, an executive committee member on the section on minority health equity and inclusion at the American Academy of Pediatrics (AAP), said.

Long before the COVID-19 pandemic so publicly exposed the depths of inequality in morbidity and mortality in the United States, health advocates had been pointing out these disparities in tools used by medical professionals. But efforts to recognize that race is a poor proxy for genetics is in its infancy.

In May, the AAP published a policy statement that kicked off its examination of clinical guidelines and policies that include race as a biological proxy. A committee for the society is combing through each guideline or calculator, evaluating the scientific basis for the use of race, and examining whether a stronger datapoint could be used instead.

The eGFR is perhaps the best example of a calculator that’s gone through the process: Health care stakeholders questioned the use of race, and investigators went back to study whether race was really a good datapoint. It wasn’t, and Dr. Chamilo’s hospital joined many others in retiring the calculator.

But the eGFR is one of countless clinical tools – from rudimentary algorithms to sophisticated machine-learning instruments – that change the course of care in part on the basis of race in the same way datapoints such as weight, age, and height are used to inform decisions about patient management. But unlike race, height, weight, and age can be objectively measured. A physician either makes a guess, or a patient enters their race on a form. And while that can be useful on a population level, race does not equal genetics or any other measurable datapoint.

In a study published in JAMA Pediatrics, researchers reviewed 414 clinical practice guidelines from sources such as PubMed and MetaLib.gov. Almost 1 in 6 guidelines included race in an inappropriate way, such as by conflating race as a biological risk factor or establishing testing or treatment thresholds using race.
 

 

 

Waiting for alternatives

The University of Maryland Medical System last year embarked on a project similar to the AAP initiative but within its own system. The first use of race to be eliminated was in the eGFR. The health system also recently removed the variable from a tool for diagnosing urinary tract infections (UTIs) in children younger than 2 years.

Part of that tool includes deciding to perform a catheterized urine test. If a doctor chose “White” as the race, the tool would recommend the test. If the doctor chose “Black,” the tool would recommend to not test. Joseph Wright, MD, MPH, chief health equity officer at University of Maryland Medical System, said this step in the tool is based on the unproven assumption that young Black children had a lower likelihood of UTIs than their White peers.

“We simply want folks to not by default lob race in as a decisionmaking point when we have, with a little bit more scientific diligence, the ability to include better clinical variables,” Dr. Wright, who is also an adjunct professor of health policy and management at the University of Maryland School of Public Health, College Park, said.

The developers of the UTI tool recently released a revised version that removes race in favor of two new medical datapoints: whether the patient has had a fever for over 48 hours, and whether the patient has previously had a UTI.

The process of re-examining tools, coming up with new datapoints, and implementing changes is not simple, according to Dr. Wright.

“This is just the baby step to fix the algorithms, because we’re all going to have to examine our own house, where these calculators live, whether it’s in a textbook, whether it’s in an electronic health record, and that’s the heavy lift,” he said. “All sources of clinical guidance have to be scrutinized, and it’s going to literally take years to unroot.”

Electronic medical record vendor Cerner said it generally revises its algorithms after medical societies make changes, then communicates those fixes to providers.

Rebecca C. Winokur, MD, MPH, lead physician executive and health equity service line leader at Cerner, explained that if doctors ordered an eGFR a year ago and then another today, the results might be different because of the new code that eliminates race.

“The numbers are so different, how do you know that the patient may or may not have the same function?” Dr. Winokur said.

Dr. Winokur said the company is trying to determine at which point a message should pop up in the records workflow that would inform clinicians that they may be comparing apples to cherries. The company also is reconsidering the use of race in tools that estimate the probability of a successful vaginal birth after prior cesarean delivery, a calculator that predicts the risk of urethral stones in patients with flank pain, and another that measures lung function to help diagnose pulmonary disease.

In addition to managing the logistics of removing race, health institutions also need buy-in from clinicians. At Mass General Brigham, Boston, Thomas Sequist, MD, MPH, chief medical officer, is leading a project to examine how the system uses race in calculators.

“People struggle mainly with, well, if we shouldn’t use this calculator, what should we use, because we need a calculator. And that’s a legitimate question,” Dr. Sequist said in an interview. “If we’re going to stop using this race-based calculator, I still need to know what dose of medication I give my patient. We’re not going to pull any of these calculators until we have a safe and reliable alternative.”

For each calculator, relevant specialty chiefs come to the table with Dr. Sequist and his team; current projects include examining bone density screenings and cardiac risk scores. A large part of the work is communicating the lack of science behind the inclusion of race as a variable.

“It’s hard because these tools have been in existence for decades, and people are used to using them,” Dr. Sequist said. “So this is a big-change management project.”

Some clinicians also have difficulty discerning why their health system may stratify patient outcomes by race while providers are being told that race is being removed from the calculators they use every day. The key difference is that stratifying outcomes by race illuminates systemic problems that can be targeted by a health system.

For instance, if readmission rates are higher for Black patients overall after surgery, the reason might be that nurses are not delivering the same level of care to them as they are to non-Black patients, possibly because of hidden bias. Or, perhaps Black patients at a hospital have less access to transportation for follow-up appointments after surgery. The potential reasons can be investigated, and solutions can be created.

“If you look at a population level, what you’re looking for is not for the evidence of race as a biological construct,” Dr. Chomilo said. “You’re looking for the impact of racism on populations, and that’s the difference: It’s racism, not race.”

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

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Roe v. Wade reversal would rock ob.gyn. residencies

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Roe v. Wade reversal would rock ob.gyn. residencies

A decision by the U.S. Supreme Court to overturn Roe v. Wade could have far-reaching impacts on residents in ob.gyn. medical programs across the country, from learning simple procedures like ultrasounds to how to manage miscarriages.

On Monday, Politico published a leaked initial draft majority opinion written by Justice Samuel Alito, which declares Roe unconstitutional. The actual opinion is due at the end of June. If all the justices keep their positions for that vote, the 1973 court case that legalized abortion rights in the United States would no longer keep states from banning the procedure before viability, which is defined as 24-26 weeks into a pregnancy.

Loss of federal protections provided by Roe would leave a patchwork of states where providers would have to change reproductive health practices. Medical schools in those states would also have to curtail any abortion care training, experts told this news organization.

Constance Bohon, MD, co-chair of the Legislative and Liability Committee of the American College of Obstetricians and Gynecologists (ACOG), said: “As a profession, we are concerned about the possibility that there will be ob.gyn. residents who graduate without the experience and training needed to care for a patient who has complications from an abortion or a missed abortion. We are very concerned that without adequate training and access to care for these patients, the maternal mortality rate will rise.”

The loss of Roe “means that sadly, and realistically, many residents just won’t get the training, or we will need to think about mitigation strategies, like providing simulation training,” said Kavita Vinekar, MD, MPH, assistant clinical professor in the department of obstetrics and gynecology at the David Geffen School of Medicine at UCLA. “I can assure you that without access to abortion training, the quality of ob.gyn. training will absolutely suffer.”

Twenty-two states, including large swaths of the south and Midwest, already have laws that would go into effect immediately to ban abortion in the absence of Roe. Four states, including Florida, Montana, and Indiana, would likely ban abortion, according to an analysis from the Guttmacher Institute.

Almost 45% of ob.gyn. obstetrics and gynecology residency programs are in these states, totaling 2,638 residents as of 2022, according to a study published in April by researchers at the UCLA.

Most reproductive health care training lasts between 1-2 months during residency and includes instruction on ultrasounds, best practices in managing pregnancy complications, learning how to safely evacuate a uterus in the event of a stillbirth or miscarriage, and counseling for family planning options.
 

A glimpse at the future

The potential for a new reality is already playing out in Texas, where a law banning abortions after 6 weeks in pregnancy took effect last Sept. 1. The Ryan Program, a national initiative to teach abortion care to medical school residents, started a pilot to match resident physicians in Texas with hospital programs in other states without abortion restrictions.

The Ryan Program has matched over 40 residents since the law took effect, according to Jody Steinauer, MD, PhD, who oversees the Ryan Program at the Bixby Center for Global Reproductive Health at the University of California, San Francisco. Matching students is an arduous and timely process because students have hectic schedules, and state licensure and other regulatory issues must be worked out, she said.

“It will take a while to get systems in place to really support resident travel, and not every resident is going to be able to travel, so I’m just worried about the future impacts on patient care,” Dr. Steinauer told this news organization. “We have hundreds of residents who are not learning the skills they need, and they’re not going be able to provide evidence-based, patient-centered care.”

But Dr. Bohon expressed skepticism that the Ryan Program and other initiatives to provide training for travel residents would be sufficient. “Unfortunately, it is anticipated that this program will not be sufficient to provide training for all of the ob.gyn. residents who do not have access to abortion training because of the state where their residency is,” she said. “There are residency programs that have the capacity to have ob.gyns. get training at their programs, but there is a limited number of these positions available as well.”

She added that the Council on Resident Education in Obstetrics and Gynecology is actively pursuing options to provide abortion training for residents for whom such training is not available. 
 

 

 

Spillover effect

Health care professionals in states without restrictive abortion laws will also see an increase in patients seeking abortion-related care. This spike already is happening in New Mexico, which borders not only Texas, but Oklahoma and Arizona – states that have severely restricted abortion rights.

“It’s difficult in the states that will be overloaded with additional abortion care to then incorporate learners on top of that,” said Eve Espey, MD, MPH, distinguished professor and chair of the department of obstetrics and gynecology at the University of New Mexico, Albuquerque. “Logistically, to fill that gap in care and train at the same time will be really challenging.”

Dr. Espey said the UNM Center for Reproductive Health has already seen patients from Texas who would likely fall under an exemption in that state’s law that allows abortions if a patient faces a medical emergency if the pregnancy continues.

“Providers are so afraid of these laws, and if they’re risk adverse, they’re always going to err on the side of not taking care of the patient if you think you can get in trouble,” Dr. Espey said, pointing to patients who presented with lethal fetal anomalies or ruptured fetal membranes early in pregnancy that might fall under the medical emergency exemption.

Dr. Steinauer with the Ryan Program said that many Texas residency program directors have told her they’ve heard from applicants who are giving low ranks to residency programs in the state.

“They’re saying, ‘if we want to be able to be trained as an ob.gyn., why would we want to go to a program with such significant restrictions?’” Dr. Steinauer said. “That is a real worry.”

Most ob.gyn. medical residency programs now provide built-in abortion training, which the Accreditation Council for Graduate Medical Education mandated in 1995. A small majority do not. According to a 2020 research letter from the ACOG, about 11% of ob.gyn. residents said their programs hypothetically offer training without a clear process to obtain it, and 8% reported that their programs offered no such training at all.

Residents can also opt out of abortion training, although students increasingly are choosing to only opt out of certain parts, such as training for abortions provided later in pregnancies, according to Dr. Steinauer.
 

A looming ‘pipeline problem’

There were about 3,550 abortion service clinicians in 2020, 72% of whom were ob.gyns., followed by family medicine doctors and advanced practice registered nurses, according to research published in March in JAMA Internal Medicine. This number is likely not an accurate tally, however, because the authors queried a national medical claims database that did not include self-pay patients.

Julia Strasser, DrPH, MPH, a senior research scientist at The George Washington University Milken Institute School of Public Health, Washington, D.C., who led the study, said states could create policies to broaden access to abortion for patients and training for medical residents.

Those include expanding laws to allow advanced practice clinicians to provide abortion care, allowing state Medicaid programs to opt-in for abortion payment, and establishing abortion care continuing medical education requirements for state boards of medicine for both ob.gyn. and primary care.

“Providers that practice in those restricted states will not only stop providing care there but also won’t be able to train the next generation of workforce to be able to provide that care,” Dr. Strasser said. “It’s a pipeline problem.”

States also could encourage their medical residency programs to not hold slots open for students who want to opt out of abortion care training. The University of Washington, Seattle, in 2000 eliminated slots it had been holding open for opt-out students because the state does not have restrictions on abortion.

As abortion becomes increasingly restricted across the country, Dr. Strasser said, for medical schools in “a state that continues to make abortion available, it’s essentially their duty to make that training available.”

 

 

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

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A decision by the U.S. Supreme Court to overturn Roe v. Wade could have far-reaching impacts on residents in ob.gyn. medical programs across the country, from learning simple procedures like ultrasounds to how to manage miscarriages.

On Monday, Politico published a leaked initial draft majority opinion written by Justice Samuel Alito, which declares Roe unconstitutional. The actual opinion is due at the end of June. If all the justices keep their positions for that vote, the 1973 court case that legalized abortion rights in the United States would no longer keep states from banning the procedure before viability, which is defined as 24-26 weeks into a pregnancy.

Loss of federal protections provided by Roe would leave a patchwork of states where providers would have to change reproductive health practices. Medical schools in those states would also have to curtail any abortion care training, experts told this news organization.

Constance Bohon, MD, co-chair of the Legislative and Liability Committee of the American College of Obstetricians and Gynecologists (ACOG), said: “As a profession, we are concerned about the possibility that there will be ob.gyn. residents who graduate without the experience and training needed to care for a patient who has complications from an abortion or a missed abortion. We are very concerned that without adequate training and access to care for these patients, the maternal mortality rate will rise.”

The loss of Roe “means that sadly, and realistically, many residents just won’t get the training, or we will need to think about mitigation strategies, like providing simulation training,” said Kavita Vinekar, MD, MPH, assistant clinical professor in the department of obstetrics and gynecology at the David Geffen School of Medicine at UCLA. “I can assure you that without access to abortion training, the quality of ob.gyn. training will absolutely suffer.”

Twenty-two states, including large swaths of the south and Midwest, already have laws that would go into effect immediately to ban abortion in the absence of Roe. Four states, including Florida, Montana, and Indiana, would likely ban abortion, according to an analysis from the Guttmacher Institute.

Almost 45% of ob.gyn. obstetrics and gynecology residency programs are in these states, totaling 2,638 residents as of 2022, according to a study published in April by researchers at the UCLA.

Most reproductive health care training lasts between 1-2 months during residency and includes instruction on ultrasounds, best practices in managing pregnancy complications, learning how to safely evacuate a uterus in the event of a stillbirth or miscarriage, and counseling for family planning options.
 

A glimpse at the future

The potential for a new reality is already playing out in Texas, where a law banning abortions after 6 weeks in pregnancy took effect last Sept. 1. The Ryan Program, a national initiative to teach abortion care to medical school residents, started a pilot to match resident physicians in Texas with hospital programs in other states without abortion restrictions.

The Ryan Program has matched over 40 residents since the law took effect, according to Jody Steinauer, MD, PhD, who oversees the Ryan Program at the Bixby Center for Global Reproductive Health at the University of California, San Francisco. Matching students is an arduous and timely process because students have hectic schedules, and state licensure and other regulatory issues must be worked out, she said.

“It will take a while to get systems in place to really support resident travel, and not every resident is going to be able to travel, so I’m just worried about the future impacts on patient care,” Dr. Steinauer told this news organization. “We have hundreds of residents who are not learning the skills they need, and they’re not going be able to provide evidence-based, patient-centered care.”

But Dr. Bohon expressed skepticism that the Ryan Program and other initiatives to provide training for travel residents would be sufficient. “Unfortunately, it is anticipated that this program will not be sufficient to provide training for all of the ob.gyn. residents who do not have access to abortion training because of the state where their residency is,” she said. “There are residency programs that have the capacity to have ob.gyns. get training at their programs, but there is a limited number of these positions available as well.”

She added that the Council on Resident Education in Obstetrics and Gynecology is actively pursuing options to provide abortion training for residents for whom such training is not available. 
 

 

 

Spillover effect

Health care professionals in states without restrictive abortion laws will also see an increase in patients seeking abortion-related care. This spike already is happening in New Mexico, which borders not only Texas, but Oklahoma and Arizona – states that have severely restricted abortion rights.

“It’s difficult in the states that will be overloaded with additional abortion care to then incorporate learners on top of that,” said Eve Espey, MD, MPH, distinguished professor and chair of the department of obstetrics and gynecology at the University of New Mexico, Albuquerque. “Logistically, to fill that gap in care and train at the same time will be really challenging.”

Dr. Espey said the UNM Center for Reproductive Health has already seen patients from Texas who would likely fall under an exemption in that state’s law that allows abortions if a patient faces a medical emergency if the pregnancy continues.

“Providers are so afraid of these laws, and if they’re risk adverse, they’re always going to err on the side of not taking care of the patient if you think you can get in trouble,” Dr. Espey said, pointing to patients who presented with lethal fetal anomalies or ruptured fetal membranes early in pregnancy that might fall under the medical emergency exemption.

Dr. Steinauer with the Ryan Program said that many Texas residency program directors have told her they’ve heard from applicants who are giving low ranks to residency programs in the state.

“They’re saying, ‘if we want to be able to be trained as an ob.gyn., why would we want to go to a program with such significant restrictions?’” Dr. Steinauer said. “That is a real worry.”

Most ob.gyn. medical residency programs now provide built-in abortion training, which the Accreditation Council for Graduate Medical Education mandated in 1995. A small majority do not. According to a 2020 research letter from the ACOG, about 11% of ob.gyn. residents said their programs hypothetically offer training without a clear process to obtain it, and 8% reported that their programs offered no such training at all.

Residents can also opt out of abortion training, although students increasingly are choosing to only opt out of certain parts, such as training for abortions provided later in pregnancies, according to Dr. Steinauer.
 

A looming ‘pipeline problem’

There were about 3,550 abortion service clinicians in 2020, 72% of whom were ob.gyns., followed by family medicine doctors and advanced practice registered nurses, according to research published in March in JAMA Internal Medicine. This number is likely not an accurate tally, however, because the authors queried a national medical claims database that did not include self-pay patients.

Julia Strasser, DrPH, MPH, a senior research scientist at The George Washington University Milken Institute School of Public Health, Washington, D.C., who led the study, said states could create policies to broaden access to abortion for patients and training for medical residents.

Those include expanding laws to allow advanced practice clinicians to provide abortion care, allowing state Medicaid programs to opt-in for abortion payment, and establishing abortion care continuing medical education requirements for state boards of medicine for both ob.gyn. and primary care.

“Providers that practice in those restricted states will not only stop providing care there but also won’t be able to train the next generation of workforce to be able to provide that care,” Dr. Strasser said. “It’s a pipeline problem.”

States also could encourage their medical residency programs to not hold slots open for students who want to opt out of abortion care training. The University of Washington, Seattle, in 2000 eliminated slots it had been holding open for opt-out students because the state does not have restrictions on abortion.

As abortion becomes increasingly restricted across the country, Dr. Strasser said, for medical schools in “a state that continues to make abortion available, it’s essentially their duty to make that training available.”

 

 

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

A decision by the U.S. Supreme Court to overturn Roe v. Wade could have far-reaching impacts on residents in ob.gyn. medical programs across the country, from learning simple procedures like ultrasounds to how to manage miscarriages.

On Monday, Politico published a leaked initial draft majority opinion written by Justice Samuel Alito, which declares Roe unconstitutional. The actual opinion is due at the end of June. If all the justices keep their positions for that vote, the 1973 court case that legalized abortion rights in the United States would no longer keep states from banning the procedure before viability, which is defined as 24-26 weeks into a pregnancy.

Loss of federal protections provided by Roe would leave a patchwork of states where providers would have to change reproductive health practices. Medical schools in those states would also have to curtail any abortion care training, experts told this news organization.

Constance Bohon, MD, co-chair of the Legislative and Liability Committee of the American College of Obstetricians and Gynecologists (ACOG), said: “As a profession, we are concerned about the possibility that there will be ob.gyn. residents who graduate without the experience and training needed to care for a patient who has complications from an abortion or a missed abortion. We are very concerned that without adequate training and access to care for these patients, the maternal mortality rate will rise.”

The loss of Roe “means that sadly, and realistically, many residents just won’t get the training, or we will need to think about mitigation strategies, like providing simulation training,” said Kavita Vinekar, MD, MPH, assistant clinical professor in the department of obstetrics and gynecology at the David Geffen School of Medicine at UCLA. “I can assure you that without access to abortion training, the quality of ob.gyn. training will absolutely suffer.”

Twenty-two states, including large swaths of the south and Midwest, already have laws that would go into effect immediately to ban abortion in the absence of Roe. Four states, including Florida, Montana, and Indiana, would likely ban abortion, according to an analysis from the Guttmacher Institute.

Almost 45% of ob.gyn. obstetrics and gynecology residency programs are in these states, totaling 2,638 residents as of 2022, according to a study published in April by researchers at the UCLA.

Most reproductive health care training lasts between 1-2 months during residency and includes instruction on ultrasounds, best practices in managing pregnancy complications, learning how to safely evacuate a uterus in the event of a stillbirth or miscarriage, and counseling for family planning options.
 

A glimpse at the future

The potential for a new reality is already playing out in Texas, where a law banning abortions after 6 weeks in pregnancy took effect last Sept. 1. The Ryan Program, a national initiative to teach abortion care to medical school residents, started a pilot to match resident physicians in Texas with hospital programs in other states without abortion restrictions.

The Ryan Program has matched over 40 residents since the law took effect, according to Jody Steinauer, MD, PhD, who oversees the Ryan Program at the Bixby Center for Global Reproductive Health at the University of California, San Francisco. Matching students is an arduous and timely process because students have hectic schedules, and state licensure and other regulatory issues must be worked out, she said.

“It will take a while to get systems in place to really support resident travel, and not every resident is going to be able to travel, so I’m just worried about the future impacts on patient care,” Dr. Steinauer told this news organization. “We have hundreds of residents who are not learning the skills they need, and they’re not going be able to provide evidence-based, patient-centered care.”

But Dr. Bohon expressed skepticism that the Ryan Program and other initiatives to provide training for travel residents would be sufficient. “Unfortunately, it is anticipated that this program will not be sufficient to provide training for all of the ob.gyn. residents who do not have access to abortion training because of the state where their residency is,” she said. “There are residency programs that have the capacity to have ob.gyns. get training at their programs, but there is a limited number of these positions available as well.”

She added that the Council on Resident Education in Obstetrics and Gynecology is actively pursuing options to provide abortion training for residents for whom such training is not available. 
 

 

 

Spillover effect

Health care professionals in states without restrictive abortion laws will also see an increase in patients seeking abortion-related care. This spike already is happening in New Mexico, which borders not only Texas, but Oklahoma and Arizona – states that have severely restricted abortion rights.

“It’s difficult in the states that will be overloaded with additional abortion care to then incorporate learners on top of that,” said Eve Espey, MD, MPH, distinguished professor and chair of the department of obstetrics and gynecology at the University of New Mexico, Albuquerque. “Logistically, to fill that gap in care and train at the same time will be really challenging.”

Dr. Espey said the UNM Center for Reproductive Health has already seen patients from Texas who would likely fall under an exemption in that state’s law that allows abortions if a patient faces a medical emergency if the pregnancy continues.

“Providers are so afraid of these laws, and if they’re risk adverse, they’re always going to err on the side of not taking care of the patient if you think you can get in trouble,” Dr. Espey said, pointing to patients who presented with lethal fetal anomalies or ruptured fetal membranes early in pregnancy that might fall under the medical emergency exemption.

Dr. Steinauer with the Ryan Program said that many Texas residency program directors have told her they’ve heard from applicants who are giving low ranks to residency programs in the state.

“They’re saying, ‘if we want to be able to be trained as an ob.gyn., why would we want to go to a program with such significant restrictions?’” Dr. Steinauer said. “That is a real worry.”

Most ob.gyn. medical residency programs now provide built-in abortion training, which the Accreditation Council for Graduate Medical Education mandated in 1995. A small majority do not. According to a 2020 research letter from the ACOG, about 11% of ob.gyn. residents said their programs hypothetically offer training without a clear process to obtain it, and 8% reported that their programs offered no such training at all.

Residents can also opt out of abortion training, although students increasingly are choosing to only opt out of certain parts, such as training for abortions provided later in pregnancies, according to Dr. Steinauer.
 

A looming ‘pipeline problem’

There were about 3,550 abortion service clinicians in 2020, 72% of whom were ob.gyns., followed by family medicine doctors and advanced practice registered nurses, according to research published in March in JAMA Internal Medicine. This number is likely not an accurate tally, however, because the authors queried a national medical claims database that did not include self-pay patients.

Julia Strasser, DrPH, MPH, a senior research scientist at The George Washington University Milken Institute School of Public Health, Washington, D.C., who led the study, said states could create policies to broaden access to abortion for patients and training for medical residents.

Those include expanding laws to allow advanced practice clinicians to provide abortion care, allowing state Medicaid programs to opt-in for abortion payment, and establishing abortion care continuing medical education requirements for state boards of medicine for both ob.gyn. and primary care.

“Providers that practice in those restricted states will not only stop providing care there but also won’t be able to train the next generation of workforce to be able to provide that care,” Dr. Strasser said. “It’s a pipeline problem.”

States also could encourage their medical residency programs to not hold slots open for students who want to opt out of abortion care training. The University of Washington, Seattle, in 2000 eliminated slots it had been holding open for opt-out students because the state does not have restrictions on abortion.

As abortion becomes increasingly restricted across the country, Dr. Strasser said, for medical schools in “a state that continues to make abortion available, it’s essentially their duty to make that training available.”

 

 

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

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Bill to speed FDA approvals includes rewards for drugs designed for kids

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Bill to speed FDA approvals includes rewards for drugs designed for kids

Advocates for children with rare diseases are closely watching a congressional effort to streamline the nation’s drug approval process, because the bill includes a provision extending a federal program that rewards companies making remedies for these young patients.

The reward program, the advocates say, offers hope to families that often have very few options. Approximately 15 million children are diagnosed with rare diseases, and 35% of deaths in the first year of life are caused by them.

“Treatments aren’t getting to kids, and kids deserve more than the leftovers,” said Nancy Goodman, the founder and executive director of the advocacy group Kids v Cancer, whose 10-year-old son died from brain cancer. She helped push for the original reward program in the hope that children like her son would have access to a wider range of treatments.

The extension of that program is part of the bipartisan 21st Century Cures bill, which seeks to rewrite the rules for drug development to make innovative treatments available faster. The overall bill has generated support on Capitol Hill, but some critics contend that it has the potential to undermine drug safety and profit drug makers.

Children’s advocates say there is a shortage of good therapies for rare and often deadly pediatric diseases that can include a wide variety of conditions including cancer, skull deformities, or enzyme deficiencies. Pharmaceutical companies have historically been hesitant to test drugs for children because of concerns about potential negative outcomes, children’s inability to consent to treatment, and the perception that the market for these drugs was limited. So doctors often have been left to use adult-tested drugs on sick children without the studies that show pediatric safety or effectiveness. But drugs used on adults don’t always work on children in the same way because of differences in metabolism and maturation of organs.

The advocates say more research needs to be conducted with children. But that testing is a sensitive process. It can be very costly, and it requires extra care because there are more stringent ethical protocols to protect these minors. Bad results – either injuries or deaths – can set back research efforts and have financial consequences for the company.

With that in mind, Congress in 2011 set up a program to help promote more pediatric drug research. It gives creators of medicine for rare pediatric diseases a voucher that they can use to have another one of their drugs approved quicker than usual – 6 months vs. a process that can run a year or often more.

Drug makers can also sell that voucher, which can be a big windfall for a small drug company trying to recoup research and development costs. There have been four vouchers given out since 2014, and one was sold for $67.5 million and a second for $125 million.

The voucher program, which advocates say holds big potential, expires next year. The cures bill seeks to extend it another 3 years.

“A lot of companies are reluctant to get into pediatric drug development because it’s very difficult if something goes really wrong,” said Alexander Gaffney of the Regulatory Affairs Professionals Society, an association for people involved in overseeing health care or the quality of health care products.

Drugs that are approved for cancer in adults are commonly not approved in kids. “Children are not simply small adults, they metabolize drugs very differently,” Mr. Gaffney said.

Critics say that however well-intentioned the voucher program is, it could have some unintended consequences. For example, a company could get a drug approved by the Food and Drug Administration but never bring it to market, if the maker decides it would not generate enough money. Yet the company would still pocket the priority review voucher.

Because of the speed sought by the program, vouchers could be given out without some of the safeguards that come in more traditional testing. For example, the research might not uncover that the drug could be fatal to a child after a few months or years.

Diana Zuckerman, president of the National Center for Health Research, a nonprofit group that seeks to represent children and families on health research policy issues, says the rush in moving drugs through the system can obscure problems. Drug makers “shouldn’t be able to sell it [or use it] unless it works,” she said.

She noted that in some studies, as few as 10 kids are included because the disease is so rare. With such a small population size, the company is not likely looking at big profits.

 

 

“When you’re doing a study of rare disease, it’s a small sample size and it’s easy to manipulate the data to make it look better than it is,” said. “You don’t want an incentive to represent the company wrongly in the short-term,” to get the voucher for another larger drug.

Julia Jenkins, executive director of the EveryLife Foundation for Rare Diseases, an advocacy group pushing for drug companies to spend more on drug development, wants the pediatric drug voucher program extended. She notes that the program is still too new for officials to evaluate whether it is effective.

One problematic part of the current House version, she said, is that it only extends the program for 3 years, and drug companies generally need 10 years to scratch up investors and research a new drug. The potential reward of expedited drug review might not be enough to allow a company to make a financial plan for a drug based on the program.

The bigger cures bill covers more than 60 health issues, including a $10 billion boost in funding for the National Institutes of Health and $550 million in extra money for the FDA over the next 5 years. Other provisions include creating a database of genomic information from a million U.S. patient volunteers and allowing the FDA to approve drugs without the standard clinical trial, instead using smaller observational studies or clinical experiences.

The bill passed the House Energy and Commerce Committee unanimously in May and is expected to come up for a vote in the full House. Senators are in the early stages of working on a similar bill.

Kaiser Health News (KHN) is a nonprofit national health policy news service.

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Advocates for children with rare diseases are closely watching a congressional effort to streamline the nation’s drug approval process, because the bill includes a provision extending a federal program that rewards companies making remedies for these young patients.

The reward program, the advocates say, offers hope to families that often have very few options. Approximately 15 million children are diagnosed with rare diseases, and 35% of deaths in the first year of life are caused by them.

“Treatments aren’t getting to kids, and kids deserve more than the leftovers,” said Nancy Goodman, the founder and executive director of the advocacy group Kids v Cancer, whose 10-year-old son died from brain cancer. She helped push for the original reward program in the hope that children like her son would have access to a wider range of treatments.

The extension of that program is part of the bipartisan 21st Century Cures bill, which seeks to rewrite the rules for drug development to make innovative treatments available faster. The overall bill has generated support on Capitol Hill, but some critics contend that it has the potential to undermine drug safety and profit drug makers.

Children’s advocates say there is a shortage of good therapies for rare and often deadly pediatric diseases that can include a wide variety of conditions including cancer, skull deformities, or enzyme deficiencies. Pharmaceutical companies have historically been hesitant to test drugs for children because of concerns about potential negative outcomes, children’s inability to consent to treatment, and the perception that the market for these drugs was limited. So doctors often have been left to use adult-tested drugs on sick children without the studies that show pediatric safety or effectiveness. But drugs used on adults don’t always work on children in the same way because of differences in metabolism and maturation of organs.

The advocates say more research needs to be conducted with children. But that testing is a sensitive process. It can be very costly, and it requires extra care because there are more stringent ethical protocols to protect these minors. Bad results – either injuries or deaths – can set back research efforts and have financial consequences for the company.

With that in mind, Congress in 2011 set up a program to help promote more pediatric drug research. It gives creators of medicine for rare pediatric diseases a voucher that they can use to have another one of their drugs approved quicker than usual – 6 months vs. a process that can run a year or often more.

Drug makers can also sell that voucher, which can be a big windfall for a small drug company trying to recoup research and development costs. There have been four vouchers given out since 2014, and one was sold for $67.5 million and a second for $125 million.

The voucher program, which advocates say holds big potential, expires next year. The cures bill seeks to extend it another 3 years.

“A lot of companies are reluctant to get into pediatric drug development because it’s very difficult if something goes really wrong,” said Alexander Gaffney of the Regulatory Affairs Professionals Society, an association for people involved in overseeing health care or the quality of health care products.

Drugs that are approved for cancer in adults are commonly not approved in kids. “Children are not simply small adults, they metabolize drugs very differently,” Mr. Gaffney said.

Critics say that however well-intentioned the voucher program is, it could have some unintended consequences. For example, a company could get a drug approved by the Food and Drug Administration but never bring it to market, if the maker decides it would not generate enough money. Yet the company would still pocket the priority review voucher.

Because of the speed sought by the program, vouchers could be given out without some of the safeguards that come in more traditional testing. For example, the research might not uncover that the drug could be fatal to a child after a few months or years.

Diana Zuckerman, president of the National Center for Health Research, a nonprofit group that seeks to represent children and families on health research policy issues, says the rush in moving drugs through the system can obscure problems. Drug makers “shouldn’t be able to sell it [or use it] unless it works,” she said.

She noted that in some studies, as few as 10 kids are included because the disease is so rare. With such a small population size, the company is not likely looking at big profits.

 

 

“When you’re doing a study of rare disease, it’s a small sample size and it’s easy to manipulate the data to make it look better than it is,” said. “You don’t want an incentive to represent the company wrongly in the short-term,” to get the voucher for another larger drug.

Julia Jenkins, executive director of the EveryLife Foundation for Rare Diseases, an advocacy group pushing for drug companies to spend more on drug development, wants the pediatric drug voucher program extended. She notes that the program is still too new for officials to evaluate whether it is effective.

One problematic part of the current House version, she said, is that it only extends the program for 3 years, and drug companies generally need 10 years to scratch up investors and research a new drug. The potential reward of expedited drug review might not be enough to allow a company to make a financial plan for a drug based on the program.

The bigger cures bill covers more than 60 health issues, including a $10 billion boost in funding for the National Institutes of Health and $550 million in extra money for the FDA over the next 5 years. Other provisions include creating a database of genomic information from a million U.S. patient volunteers and allowing the FDA to approve drugs without the standard clinical trial, instead using smaller observational studies or clinical experiences.

The bill passed the House Energy and Commerce Committee unanimously in May and is expected to come up for a vote in the full House. Senators are in the early stages of working on a similar bill.

Kaiser Health News (KHN) is a nonprofit national health policy news service.

Advocates for children with rare diseases are closely watching a congressional effort to streamline the nation’s drug approval process, because the bill includes a provision extending a federal program that rewards companies making remedies for these young patients.

The reward program, the advocates say, offers hope to families that often have very few options. Approximately 15 million children are diagnosed with rare diseases, and 35% of deaths in the first year of life are caused by them.

“Treatments aren’t getting to kids, and kids deserve more than the leftovers,” said Nancy Goodman, the founder and executive director of the advocacy group Kids v Cancer, whose 10-year-old son died from brain cancer. She helped push for the original reward program in the hope that children like her son would have access to a wider range of treatments.

The extension of that program is part of the bipartisan 21st Century Cures bill, which seeks to rewrite the rules for drug development to make innovative treatments available faster. The overall bill has generated support on Capitol Hill, but some critics contend that it has the potential to undermine drug safety and profit drug makers.

Children’s advocates say there is a shortage of good therapies for rare and often deadly pediatric diseases that can include a wide variety of conditions including cancer, skull deformities, or enzyme deficiencies. Pharmaceutical companies have historically been hesitant to test drugs for children because of concerns about potential negative outcomes, children’s inability to consent to treatment, and the perception that the market for these drugs was limited. So doctors often have been left to use adult-tested drugs on sick children without the studies that show pediatric safety or effectiveness. But drugs used on adults don’t always work on children in the same way because of differences in metabolism and maturation of organs.

The advocates say more research needs to be conducted with children. But that testing is a sensitive process. It can be very costly, and it requires extra care because there are more stringent ethical protocols to protect these minors. Bad results – either injuries or deaths – can set back research efforts and have financial consequences for the company.

With that in mind, Congress in 2011 set up a program to help promote more pediatric drug research. It gives creators of medicine for rare pediatric diseases a voucher that they can use to have another one of their drugs approved quicker than usual – 6 months vs. a process that can run a year or often more.

Drug makers can also sell that voucher, which can be a big windfall for a small drug company trying to recoup research and development costs. There have been four vouchers given out since 2014, and one was sold for $67.5 million and a second for $125 million.

The voucher program, which advocates say holds big potential, expires next year. The cures bill seeks to extend it another 3 years.

“A lot of companies are reluctant to get into pediatric drug development because it’s very difficult if something goes really wrong,” said Alexander Gaffney of the Regulatory Affairs Professionals Society, an association for people involved in overseeing health care or the quality of health care products.

Drugs that are approved for cancer in adults are commonly not approved in kids. “Children are not simply small adults, they metabolize drugs very differently,” Mr. Gaffney said.

Critics say that however well-intentioned the voucher program is, it could have some unintended consequences. For example, a company could get a drug approved by the Food and Drug Administration but never bring it to market, if the maker decides it would not generate enough money. Yet the company would still pocket the priority review voucher.

Because of the speed sought by the program, vouchers could be given out without some of the safeguards that come in more traditional testing. For example, the research might not uncover that the drug could be fatal to a child after a few months or years.

Diana Zuckerman, president of the National Center for Health Research, a nonprofit group that seeks to represent children and families on health research policy issues, says the rush in moving drugs through the system can obscure problems. Drug makers “shouldn’t be able to sell it [or use it] unless it works,” she said.

She noted that in some studies, as few as 10 kids are included because the disease is so rare. With such a small population size, the company is not likely looking at big profits.

 

 

“When you’re doing a study of rare disease, it’s a small sample size and it’s easy to manipulate the data to make it look better than it is,” said. “You don’t want an incentive to represent the company wrongly in the short-term,” to get the voucher for another larger drug.

Julia Jenkins, executive director of the EveryLife Foundation for Rare Diseases, an advocacy group pushing for drug companies to spend more on drug development, wants the pediatric drug voucher program extended. She notes that the program is still too new for officials to evaluate whether it is effective.

One problematic part of the current House version, she said, is that it only extends the program for 3 years, and drug companies generally need 10 years to scratch up investors and research a new drug. The potential reward of expedited drug review might not be enough to allow a company to make a financial plan for a drug based on the program.

The bigger cures bill covers more than 60 health issues, including a $10 billion boost in funding for the National Institutes of Health and $550 million in extra money for the FDA over the next 5 years. Other provisions include creating a database of genomic information from a million U.S. patient volunteers and allowing the FDA to approve drugs without the standard clinical trial, instead using smaller observational studies or clinical experiences.

The bill passed the House Energy and Commerce Committee unanimously in May and is expected to come up for a vote in the full House. Senators are in the early stages of working on a similar bill.

Kaiser Health News (KHN) is a nonprofit national health policy news service.

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