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Crossing State Lines: PA Licensure Compact Coming Soon
For decades, physicians and nurses who ventured across state lines to practice, particularly in locum tenens roles, have reaped the benefits of medical licensure compacts. Yet, the same courtesy has eluded physician assistants (PAs), until now.
In April, Virginia Governor Glenn Youngkin signed the bill enacting the PA Compact making Virginia the seventh state to join. The legislation opens a cross-state agreement with seven states and finally allows locum tenens PAs to practice across these state’s borders.
How the PA Compact Works
The interstate arrangement recognizes valid, unencumbered PA licenses issued by other states in the compact. PAs working within the seven states won’t need a separate license from any of those states to practice.
The states include Delaware, Nebraska, Utah, Washington, West Virginia, Wisconsin, and Virginia. While the compact has been approved, the American Academy of Physician Associates said it could take an additional 18-24 months for the states to execute it, giving PAs the access they need to work in the compact states.
How the PA Compact Helps
The PA Compact holds the promise of alleviating some of the travel barriers that PAs often encounter, especially when they work locum tenens or in telehealth and must traverse state lines to deliver essential healthcare. This agreement not only enhances healthcare access but also empowers facilities to recruit new PAs, thereby bridging gaps in their healthcare staffing and addressing public health emergencies more effectively.
PAs will also gain increased flexibility and additional opportunities to earn and benefit from the right to practice in more states without requiring a time-consuming and expensive licensure from each state.
One motivating factor behind developing an interstate compact for physician assistants is that the same types of compacts for physicians and nurses are highly successful. The Nurse Licensure Compact and the Interstate Medical Licensure Compact for physicians encompass 37 and 41 states, respectively. While the seven-state PA Compact is in its earliest stages, it will likely be equally beneficial for PAs.
A survey by Barton Associates found that 95% of PAs said they would be more likely to consider working in a different state if the PA Compact made it more accessible.
Other states have begun legislation to enact a PA Compact, including Colorado, New Hampshire, Maine, Michigan New York, Ohio, Oklahoma, Rhode Island, Tennessee, and Vermont.
If your state still needs to enact a compact or file for compact legislation, let your elected officials know that the PAs in your state want to join a compact.
A version of this article appeared on Medscape.com .
For decades, physicians and nurses who ventured across state lines to practice, particularly in locum tenens roles, have reaped the benefits of medical licensure compacts. Yet, the same courtesy has eluded physician assistants (PAs), until now.
In April, Virginia Governor Glenn Youngkin signed the bill enacting the PA Compact making Virginia the seventh state to join. The legislation opens a cross-state agreement with seven states and finally allows locum tenens PAs to practice across these state’s borders.
How the PA Compact Works
The interstate arrangement recognizes valid, unencumbered PA licenses issued by other states in the compact. PAs working within the seven states won’t need a separate license from any of those states to practice.
The states include Delaware, Nebraska, Utah, Washington, West Virginia, Wisconsin, and Virginia. While the compact has been approved, the American Academy of Physician Associates said it could take an additional 18-24 months for the states to execute it, giving PAs the access they need to work in the compact states.
How the PA Compact Helps
The PA Compact holds the promise of alleviating some of the travel barriers that PAs often encounter, especially when they work locum tenens or in telehealth and must traverse state lines to deliver essential healthcare. This agreement not only enhances healthcare access but also empowers facilities to recruit new PAs, thereby bridging gaps in their healthcare staffing and addressing public health emergencies more effectively.
PAs will also gain increased flexibility and additional opportunities to earn and benefit from the right to practice in more states without requiring a time-consuming and expensive licensure from each state.
One motivating factor behind developing an interstate compact for physician assistants is that the same types of compacts for physicians and nurses are highly successful. The Nurse Licensure Compact and the Interstate Medical Licensure Compact for physicians encompass 37 and 41 states, respectively. While the seven-state PA Compact is in its earliest stages, it will likely be equally beneficial for PAs.
A survey by Barton Associates found that 95% of PAs said they would be more likely to consider working in a different state if the PA Compact made it more accessible.
Other states have begun legislation to enact a PA Compact, including Colorado, New Hampshire, Maine, Michigan New York, Ohio, Oklahoma, Rhode Island, Tennessee, and Vermont.
If your state still needs to enact a compact or file for compact legislation, let your elected officials know that the PAs in your state want to join a compact.
A version of this article appeared on Medscape.com .
For decades, physicians and nurses who ventured across state lines to practice, particularly in locum tenens roles, have reaped the benefits of medical licensure compacts. Yet, the same courtesy has eluded physician assistants (PAs), until now.
In April, Virginia Governor Glenn Youngkin signed the bill enacting the PA Compact making Virginia the seventh state to join. The legislation opens a cross-state agreement with seven states and finally allows locum tenens PAs to practice across these state’s borders.
How the PA Compact Works
The interstate arrangement recognizes valid, unencumbered PA licenses issued by other states in the compact. PAs working within the seven states won’t need a separate license from any of those states to practice.
The states include Delaware, Nebraska, Utah, Washington, West Virginia, Wisconsin, and Virginia. While the compact has been approved, the American Academy of Physician Associates said it could take an additional 18-24 months for the states to execute it, giving PAs the access they need to work in the compact states.
How the PA Compact Helps
The PA Compact holds the promise of alleviating some of the travel barriers that PAs often encounter, especially when they work locum tenens or in telehealth and must traverse state lines to deliver essential healthcare. This agreement not only enhances healthcare access but also empowers facilities to recruit new PAs, thereby bridging gaps in their healthcare staffing and addressing public health emergencies more effectively.
PAs will also gain increased flexibility and additional opportunities to earn and benefit from the right to practice in more states without requiring a time-consuming and expensive licensure from each state.
One motivating factor behind developing an interstate compact for physician assistants is that the same types of compacts for physicians and nurses are highly successful. The Nurse Licensure Compact and the Interstate Medical Licensure Compact for physicians encompass 37 and 41 states, respectively. While the seven-state PA Compact is in its earliest stages, it will likely be equally beneficial for PAs.
A survey by Barton Associates found that 95% of PAs said they would be more likely to consider working in a different state if the PA Compact made it more accessible.
Other states have begun legislation to enact a PA Compact, including Colorado, New Hampshire, Maine, Michigan New York, Ohio, Oklahoma, Rhode Island, Tennessee, and Vermont.
If your state still needs to enact a compact or file for compact legislation, let your elected officials know that the PAs in your state want to join a compact.
A version of this article appeared on Medscape.com .
Follow-Up Outcomes Data Often Missing for FDA Drug Approvals Based on Surrogate Markers
Over the past few decades, the US Food and Drug Administration (FDA) has increasingly relied on surrogate measures such as blood tests instead of clinical outcomes for medication approvals. But critics say the agency lacks consistent standards to ensure the surrogate aligns with clinical outcomes that matter to patients — things like improvements in symptoms and gains in function.
Sometimes those decisions backfire. Consider: In July 2021, the FDA approved aducanumab for the treatment of Alzheimer’s disease, bucking the advice of an advisory panel for the agency that questioned the effectiveness of the medication. Regulators relied on data from the drugmaker, Biogen, showing the monoclonal antibody could reduce levels of amyloid beta plaques in blood — a surrogate marker officials hoped would translate to clinical benefit.
The FDA’s decision triggered significant controversy, and Biogen in January announced it is pulling it from the market this year, citing disappointing sales.
Although the case of aducanumab might seem extreme, given the stakes — Alzheimer’s remains a disease without an effective treatment — it’s far from unusual.
“When we prescribe a drug, there is an underlying assumption that the FDA has done its due diligence to confirm the drug is safe and of benefit,” said Reshma Ramachandran, MD, MPP, MHS, a researcher at Yale School of Medicine, New Haven, Connecticut, and a coauthor of a recent review of surrogate outcomes. “In fact, we found either no evidence or low-quality evidence.” Such markers are associated with clinical outcomes. “We just don’t know if they work meaningfully to treat the patient’s condition. The results were pretty shocking for us,” she said.
The FDA in 2018 released an Adult Surrogate Endpoint Table listing markers that can be used as substitutes for clinical outcomes to more quickly test, review, and approve new therapies. The analysis found the majority of these endpoints lacked subsequent confirmations, defined as published meta-analyses of clinical studies to validate the association between the marker and a clinical outcome important to patients.
In a paper published in JAMA, Dr. Ramachandran and her colleagues looked at 37 surrogate endpoints for nearly 3 dozen nononcologic diseases in the table.
Approval with surrogate markers implies responsibility for postapproval or validation studies — not just lab measures or imaging findings but mortality, morbidity, or improved quality of life, said Joshua D. Wallach, PhD, MS, assistant professor in the department of epidemiology at the Emory Rollins School of Public Health in Atlanta and lead author of the JAMA review.
Dr. Wallach said surrogate markers are easier to measure and do not require large and long trials. But the FDA has not provided clear rules for what makes a surrogate marker valid in clinical trials.
“They’ve said that at a minimum, it requires meta-analytical evidence from studies that have looked at the correlation or the association between the surrogate and the clinical outcome,” Dr. Wallach said. “Our understanding was that if that’s a minimum expectation, we should be able to find those studies in the literature. And the reality is that we were unable to find evidence from those types of studies supporting the association between the surrogate and the clinical outcome.”
Physicians generally do not receive training about the FDA approval process and the difference between biomarkers, surrogate markers, and clinical endpoints, Dr. Ramachandran said. “Our study shows that things are much more uncertain than we thought when it comes to the prescribing of new drugs,” she said.
Surrogate Markers on the Rise
Dr. Wallach’s group looked for published meta-analyses compiling randomized controlled trials reporting surrogate endpoints for more than 3 dozen chronic nononcologic conditions, including type 2 diabetes, Alzheimer’s, kidney disease, HIV, gout, and lupus. They found no meta-analyses at all for 59% of the surrogate markers, while for those that were studied, few reported high-strength evidence of an association with clinical outcomes.
The findings echo previous research. In a 2020 study in JAMA Network Open, researchers tallied primary endpoints for all FDA approvals of new drugs and therapies during three 3-year periods: 1995-1997, 2005-2007, and 2015-2017. The proportion of products whose approvals were based on the use of clinical endpoints decreased from 43.8% in 1995-1997 to 28.4% in 2005-2007 to 23.3% in 2015-2017. The share based on surrogate endpoints rose from 43.3% to roughly 60% over the same interval.
A 2017 study in the Journal of Health Economics found the use of “imperfect” surrogate endpoints helped support the approval of an average of 16 new drugs per year between 2010 and 2014 compared with six per year from 1998 to 2008.
Similar concerns about weak associations between surrogate markers and drugs used to treat cancer have been documented before, including in a 2020 study published in eClinicalMedicine. The researchers found the surrogate endpoints in the FDA table either were not tested or were tested but proven to be weak surrogates.
“And yet the FDA considered these as good enough not only for accelerated approval but also for regular approval,” said Bishal Gyawali, MD, PhD, associate professor in the department of oncology at Queen’s University, Kingston, Ontario, Canada, who led the group.
The use of surrogate endpoints is also increasing in Europe, said Huseyin Naci, MHS, PhD, associate professor of health policy at the London School of Economics and Political Science in England. He cited a cohort study of 298 randomized clinical trials (RCTs) in JAMA Oncology suggesting “contemporary oncology RCTs now largely measure putative surrogate endpoints.” Dr. Wallach called the FDA’s surrogate table “a great first step toward transparency. But a key column is missing from that table, telling us what is the basis for which the FDA allows drug companies to use the recognized surrogate markers. What is the evidence they are considering?”
If the agency allows companies the flexibility to validate surrogate endpoints, postmarketing studies designed to confirm the clinical utility of those endpoints should follow.
“We obviously want physicians to be guided by evidence when they’re selecting treatments, and they need to be able to interpret the clinical benefits of the drug that they’re prescribing,” he said. “This is really about having the research consumer, patients, and physicians, as well as industry, understand why certain markers are considered and not considered.”
Dr. Wallach reported receiving grants from the FDA (through the Yale University — Mayo Clinic Center of Excellence in Regulatory Science and Innovation), National Institute on Alcohol Abuse and Alcoholism (1K01AA028258), and Johnson & Johnson (through the Yale University Open Data Access Project); and consulting fees from Hagens Berman Sobol Shapiro LLP and Dugan Law Firm APLC outside the submitted work. Dr. Ramachandran reported receiving grants from the Stavros Niarchos Foundation and FDA; receiving consulting fees from ReAct Action on Antibiotic Resistance strategy policy program outside the submitted work; and serving in an unpaid capacity as chair of the FDA task force for the nonprofit organization Doctors for America and in an unpaid capacity as board president for Universities Allied for Essential Medicines North America.
A version of this article appeared on Medscape.com.
Over the past few decades, the US Food and Drug Administration (FDA) has increasingly relied on surrogate measures such as blood tests instead of clinical outcomes for medication approvals. But critics say the agency lacks consistent standards to ensure the surrogate aligns with clinical outcomes that matter to patients — things like improvements in symptoms and gains in function.
Sometimes those decisions backfire. Consider: In July 2021, the FDA approved aducanumab for the treatment of Alzheimer’s disease, bucking the advice of an advisory panel for the agency that questioned the effectiveness of the medication. Regulators relied on data from the drugmaker, Biogen, showing the monoclonal antibody could reduce levels of amyloid beta plaques in blood — a surrogate marker officials hoped would translate to clinical benefit.
The FDA’s decision triggered significant controversy, and Biogen in January announced it is pulling it from the market this year, citing disappointing sales.
Although the case of aducanumab might seem extreme, given the stakes — Alzheimer’s remains a disease without an effective treatment — it’s far from unusual.
“When we prescribe a drug, there is an underlying assumption that the FDA has done its due diligence to confirm the drug is safe and of benefit,” said Reshma Ramachandran, MD, MPP, MHS, a researcher at Yale School of Medicine, New Haven, Connecticut, and a coauthor of a recent review of surrogate outcomes. “In fact, we found either no evidence or low-quality evidence.” Such markers are associated with clinical outcomes. “We just don’t know if they work meaningfully to treat the patient’s condition. The results were pretty shocking for us,” she said.
The FDA in 2018 released an Adult Surrogate Endpoint Table listing markers that can be used as substitutes for clinical outcomes to more quickly test, review, and approve new therapies. The analysis found the majority of these endpoints lacked subsequent confirmations, defined as published meta-analyses of clinical studies to validate the association between the marker and a clinical outcome important to patients.
In a paper published in JAMA, Dr. Ramachandran and her colleagues looked at 37 surrogate endpoints for nearly 3 dozen nononcologic diseases in the table.
Approval with surrogate markers implies responsibility for postapproval or validation studies — not just lab measures or imaging findings but mortality, morbidity, or improved quality of life, said Joshua D. Wallach, PhD, MS, assistant professor in the department of epidemiology at the Emory Rollins School of Public Health in Atlanta and lead author of the JAMA review.
Dr. Wallach said surrogate markers are easier to measure and do not require large and long trials. But the FDA has not provided clear rules for what makes a surrogate marker valid in clinical trials.
“They’ve said that at a minimum, it requires meta-analytical evidence from studies that have looked at the correlation or the association between the surrogate and the clinical outcome,” Dr. Wallach said. “Our understanding was that if that’s a minimum expectation, we should be able to find those studies in the literature. And the reality is that we were unable to find evidence from those types of studies supporting the association between the surrogate and the clinical outcome.”
Physicians generally do not receive training about the FDA approval process and the difference between biomarkers, surrogate markers, and clinical endpoints, Dr. Ramachandran said. “Our study shows that things are much more uncertain than we thought when it comes to the prescribing of new drugs,” she said.
Surrogate Markers on the Rise
Dr. Wallach’s group looked for published meta-analyses compiling randomized controlled trials reporting surrogate endpoints for more than 3 dozen chronic nononcologic conditions, including type 2 diabetes, Alzheimer’s, kidney disease, HIV, gout, and lupus. They found no meta-analyses at all for 59% of the surrogate markers, while for those that were studied, few reported high-strength evidence of an association with clinical outcomes.
The findings echo previous research. In a 2020 study in JAMA Network Open, researchers tallied primary endpoints for all FDA approvals of new drugs and therapies during three 3-year periods: 1995-1997, 2005-2007, and 2015-2017. The proportion of products whose approvals were based on the use of clinical endpoints decreased from 43.8% in 1995-1997 to 28.4% in 2005-2007 to 23.3% in 2015-2017. The share based on surrogate endpoints rose from 43.3% to roughly 60% over the same interval.
A 2017 study in the Journal of Health Economics found the use of “imperfect” surrogate endpoints helped support the approval of an average of 16 new drugs per year between 2010 and 2014 compared with six per year from 1998 to 2008.
Similar concerns about weak associations between surrogate markers and drugs used to treat cancer have been documented before, including in a 2020 study published in eClinicalMedicine. The researchers found the surrogate endpoints in the FDA table either were not tested or were tested but proven to be weak surrogates.
“And yet the FDA considered these as good enough not only for accelerated approval but also for regular approval,” said Bishal Gyawali, MD, PhD, associate professor in the department of oncology at Queen’s University, Kingston, Ontario, Canada, who led the group.
The use of surrogate endpoints is also increasing in Europe, said Huseyin Naci, MHS, PhD, associate professor of health policy at the London School of Economics and Political Science in England. He cited a cohort study of 298 randomized clinical trials (RCTs) in JAMA Oncology suggesting “contemporary oncology RCTs now largely measure putative surrogate endpoints.” Dr. Wallach called the FDA’s surrogate table “a great first step toward transparency. But a key column is missing from that table, telling us what is the basis for which the FDA allows drug companies to use the recognized surrogate markers. What is the evidence they are considering?”
If the agency allows companies the flexibility to validate surrogate endpoints, postmarketing studies designed to confirm the clinical utility of those endpoints should follow.
“We obviously want physicians to be guided by evidence when they’re selecting treatments, and they need to be able to interpret the clinical benefits of the drug that they’re prescribing,” he said. “This is really about having the research consumer, patients, and physicians, as well as industry, understand why certain markers are considered and not considered.”
Dr. Wallach reported receiving grants from the FDA (through the Yale University — Mayo Clinic Center of Excellence in Regulatory Science and Innovation), National Institute on Alcohol Abuse and Alcoholism (1K01AA028258), and Johnson & Johnson (through the Yale University Open Data Access Project); and consulting fees from Hagens Berman Sobol Shapiro LLP and Dugan Law Firm APLC outside the submitted work. Dr. Ramachandran reported receiving grants from the Stavros Niarchos Foundation and FDA; receiving consulting fees from ReAct Action on Antibiotic Resistance strategy policy program outside the submitted work; and serving in an unpaid capacity as chair of the FDA task force for the nonprofit organization Doctors for America and in an unpaid capacity as board president for Universities Allied for Essential Medicines North America.
A version of this article appeared on Medscape.com.
Over the past few decades, the US Food and Drug Administration (FDA) has increasingly relied on surrogate measures such as blood tests instead of clinical outcomes for medication approvals. But critics say the agency lacks consistent standards to ensure the surrogate aligns with clinical outcomes that matter to patients — things like improvements in symptoms and gains in function.
Sometimes those decisions backfire. Consider: In July 2021, the FDA approved aducanumab for the treatment of Alzheimer’s disease, bucking the advice of an advisory panel for the agency that questioned the effectiveness of the medication. Regulators relied on data from the drugmaker, Biogen, showing the monoclonal antibody could reduce levels of amyloid beta plaques in blood — a surrogate marker officials hoped would translate to clinical benefit.
The FDA’s decision triggered significant controversy, and Biogen in January announced it is pulling it from the market this year, citing disappointing sales.
Although the case of aducanumab might seem extreme, given the stakes — Alzheimer’s remains a disease without an effective treatment — it’s far from unusual.
“When we prescribe a drug, there is an underlying assumption that the FDA has done its due diligence to confirm the drug is safe and of benefit,” said Reshma Ramachandran, MD, MPP, MHS, a researcher at Yale School of Medicine, New Haven, Connecticut, and a coauthor of a recent review of surrogate outcomes. “In fact, we found either no evidence or low-quality evidence.” Such markers are associated with clinical outcomes. “We just don’t know if they work meaningfully to treat the patient’s condition. The results were pretty shocking for us,” she said.
The FDA in 2018 released an Adult Surrogate Endpoint Table listing markers that can be used as substitutes for clinical outcomes to more quickly test, review, and approve new therapies. The analysis found the majority of these endpoints lacked subsequent confirmations, defined as published meta-analyses of clinical studies to validate the association between the marker and a clinical outcome important to patients.
In a paper published in JAMA, Dr. Ramachandran and her colleagues looked at 37 surrogate endpoints for nearly 3 dozen nononcologic diseases in the table.
Approval with surrogate markers implies responsibility for postapproval or validation studies — not just lab measures or imaging findings but mortality, morbidity, or improved quality of life, said Joshua D. Wallach, PhD, MS, assistant professor in the department of epidemiology at the Emory Rollins School of Public Health in Atlanta and lead author of the JAMA review.
Dr. Wallach said surrogate markers are easier to measure and do not require large and long trials. But the FDA has not provided clear rules for what makes a surrogate marker valid in clinical trials.
“They’ve said that at a minimum, it requires meta-analytical evidence from studies that have looked at the correlation or the association between the surrogate and the clinical outcome,” Dr. Wallach said. “Our understanding was that if that’s a minimum expectation, we should be able to find those studies in the literature. And the reality is that we were unable to find evidence from those types of studies supporting the association between the surrogate and the clinical outcome.”
Physicians generally do not receive training about the FDA approval process and the difference between biomarkers, surrogate markers, and clinical endpoints, Dr. Ramachandran said. “Our study shows that things are much more uncertain than we thought when it comes to the prescribing of new drugs,” she said.
Surrogate Markers on the Rise
Dr. Wallach’s group looked for published meta-analyses compiling randomized controlled trials reporting surrogate endpoints for more than 3 dozen chronic nononcologic conditions, including type 2 diabetes, Alzheimer’s, kidney disease, HIV, gout, and lupus. They found no meta-analyses at all for 59% of the surrogate markers, while for those that were studied, few reported high-strength evidence of an association with clinical outcomes.
The findings echo previous research. In a 2020 study in JAMA Network Open, researchers tallied primary endpoints for all FDA approvals of new drugs and therapies during three 3-year periods: 1995-1997, 2005-2007, and 2015-2017. The proportion of products whose approvals were based on the use of clinical endpoints decreased from 43.8% in 1995-1997 to 28.4% in 2005-2007 to 23.3% in 2015-2017. The share based on surrogate endpoints rose from 43.3% to roughly 60% over the same interval.
A 2017 study in the Journal of Health Economics found the use of “imperfect” surrogate endpoints helped support the approval of an average of 16 new drugs per year between 2010 and 2014 compared with six per year from 1998 to 2008.
Similar concerns about weak associations between surrogate markers and drugs used to treat cancer have been documented before, including in a 2020 study published in eClinicalMedicine. The researchers found the surrogate endpoints in the FDA table either were not tested or were tested but proven to be weak surrogates.
“And yet the FDA considered these as good enough not only for accelerated approval but also for regular approval,” said Bishal Gyawali, MD, PhD, associate professor in the department of oncology at Queen’s University, Kingston, Ontario, Canada, who led the group.
The use of surrogate endpoints is also increasing in Europe, said Huseyin Naci, MHS, PhD, associate professor of health policy at the London School of Economics and Political Science in England. He cited a cohort study of 298 randomized clinical trials (RCTs) in JAMA Oncology suggesting “contemporary oncology RCTs now largely measure putative surrogate endpoints.” Dr. Wallach called the FDA’s surrogate table “a great first step toward transparency. But a key column is missing from that table, telling us what is the basis for which the FDA allows drug companies to use the recognized surrogate markers. What is the evidence they are considering?”
If the agency allows companies the flexibility to validate surrogate endpoints, postmarketing studies designed to confirm the clinical utility of those endpoints should follow.
“We obviously want physicians to be guided by evidence when they’re selecting treatments, and they need to be able to interpret the clinical benefits of the drug that they’re prescribing,” he said. “This is really about having the research consumer, patients, and physicians, as well as industry, understand why certain markers are considered and not considered.”
Dr. Wallach reported receiving grants from the FDA (through the Yale University — Mayo Clinic Center of Excellence in Regulatory Science and Innovation), National Institute on Alcohol Abuse and Alcoholism (1K01AA028258), and Johnson & Johnson (through the Yale University Open Data Access Project); and consulting fees from Hagens Berman Sobol Shapiro LLP and Dugan Law Firm APLC outside the submitted work. Dr. Ramachandran reported receiving grants from the Stavros Niarchos Foundation and FDA; receiving consulting fees from ReAct Action on Antibiotic Resistance strategy policy program outside the submitted work; and serving in an unpaid capacity as chair of the FDA task force for the nonprofit organization Doctors for America and in an unpaid capacity as board president for Universities Allied for Essential Medicines North America.
A version of this article appeared on Medscape.com.
FROM JAMA
AAP Shifts Stance, Updates Guidance on Breastfeeding With HIV
aside from avoiding breastfeeding altogether, according to a new clinical report from the American Academy of Pediatrics (AAP).
“The risk of HIV transmission via breastfeeding from a parent with HIV who is receiving antiretroviral treatment (ART) and is virally suppressed is estimated to be less than 1%,” Lisa Abuogi, MD, an associate professor of pediatric infectious disease at the University of Colorado Anschutz Medical Campus, and her colleagues wrote in Pediatrics. For people living with HIV in the United States, however, the AAP recommends that “avoidance of breastfeeding is the only infant feeding option with 0% risk of HIV transmission.”
The authors go on to suggest that pediatricians “be prepared to offer a family-centered, nonjudgmental, harm reduction approach” to support people with HIV who do want to breastfeed and have sustained viral suppression. Parents with HIV who are not on ART or who do not have adequate viral suppression should be advised against breastfeeding, the report states. Members of the AAP Committee on Pediatric and Adolescent HIV and of the AAP Section on Breastfeeding coauthored the clinical report.
“The new guidelines emphasize the importance of patient-centered counseling as the foundation for shared decision-making, allowing patients and pediatric providers to make feeding decisions together and for the first time really giving support to people with HIV in the U.S. who want to breastfeed,” Danna Biala, MD, MS, an attending pediatrician at Children’s Hospital at Montefiore and an assistant professor at Albert Einstein College of Medicine, told MDedge News.
Dr. Biala was not involved in the development of the report, but she said the AAP’s guidance reflects the recent shift in the stance of the Centers for Disease Control and Prevention (CDC) regarding breastfeeding among people who are HIV+. Recommendations from the CDC and the U.S. Department of Health and Human Services (HHS) were updated in 2023.
“I’m glad that the AAP is putting out guidelines on infant feeding for people with HIV,” Dr. Biala said. “For so long in the U.S., pediatricians have been advising all mothers with HIV to avoid breastfeeding, believing that the risk of transmission outweighed the benefits of breastfeeding.”
The updated guidance from HHS in 2023 “was revolutionary in supporting people with HIV in low-risk situations who want to breastfeed,” Dr. Biala said, but “clear protocols for monitoring and follow-up were not in place,” which these AAP guidelines help address.
Prior Discordance Between Global, U.S. Guidance
An estimated 5,000 people with HIV give birth each year in the United States, and up to one third of pregnant people with HIV may be unaware of their HIV status, the AAP report notes. Pediatric healthcare professionals in the United States therefore need to be aware of recommendations related to HIV testing of pregnant people and of counseling the feeding of infants exposed to HIV. The report recommends opt-out HIV testing at the first prenatal visit and then possibly retesting in the third trimester in areas with high HIV incidence or for people at high risk for HIV or with signs or symptoms of acute HIV infection.
The report also highlights the health benefits of breastfeeding to both the infant and the breastfeeding parent, but notes the CDC’s historical recommendation against breastfeeding for people who are HIV+. The WHO, meanwhile, began recommending in 2016 that infants be breastfed through 12 to 24 months old if the parent was on ART and/or the infant was receiving antiretroviral (ARV) prophylaxis, since research showed those treatments were effective in reducing transmission risk.
Still, an estimated 30% of perinatal HIV transmission occurs via breastfeeding worldwide, primarily from people with HIV who are not on ART or are not adequately virally suppressed. Without parental ART or infant ARV prophylaxis, HIV transmission risk to infants via breastfeeding is highest, about 5%-6%, in the first 4-6 weeks of life. Risk then declines to about 0.9% a month thereafter. The AAP report goes on to describe factors that increase or decrease the likelihood of transmission during breastfeeding, but it notes that neither ART in the breastfeeding person nor ARV prophylaxis in the infant completely eliminates the risk of HIV transmission during breastfeeding. There have been rare cases where transmission occurred despite viral suppression in the person with HIV.
Among the reasons people with HIV have expressed a desire to breastfeed are wanting to bond with their infant, wanting to provide optimal nutrition and health benefits to their baby, and meeting cultural expectations, including the desire not to disclose their HIV infection status to family or friends by virtue of not breastfeeding.
“Among immigrant and refugee populations, the discordance between infant feeding guidelines in the United States and their country of birth may result in confusion, especially among parents who breastfed previous infants,” the AAP report also notes. Further, not breastfeeding could compound health disparities already more likely to be present among those living with HIV.
Discussions about infant feeding with parents with HIV should therefore “begin as early as possible and involve a multidisciplinary team that might include the pediatric primary care provider (once identified), a pediatric HIV expert, the breastfeeding parent’s HIV care and obstetric providers, and lactation consultants,” the report states. ”The parent’s motivations for breastfeeding should be explored and counseling provided on the risks and benefits of each feeding option, including breastfeeding, formula feeding, or certified, banked donor human milk.” The statement emphasizes the need for providing counseling in a “non-judgmental, respectful way, recognizing potential drivers for their decisions such as avoidance of stigma, prior experience with breastfeeding, and cultural contributors.”
Clear Recommendations Can Help Providers
The AAP’s statement that “replacement feeding (with formula or certified, banked donor human milk) is the only option that is 100% certain to prevent postnatal transmission of HIV” feels like it takes a “more conservative or discouraging approach” to breastfeeding than the CDC or WHO guidelines, Alissa Parker-Smith, APRN, DNP, CPNP-PC, IBCLC, a nurse practitioner and lactation consultant at PrimaryPlus, a Federally Qualified Health Clinic in Ashland, Kentucky, told MDedge. But she said they do clearly align with the CDC guidelines, and their differences from the WHO guidelines make sense in light of the different populations served by the WHO versus the U.S. agencies.
“Unclean water for formula preparation and a reduced or lack of access to formula in general can lead to many other risks of death for the infant other than the very small risk of HIV infection from breastfeeding from an HIV+ parent,” Ms. Parker-Smith said. “In the U.S. we generally have consistent access to clean water and safe formula as well as social structures to help families have access to formula, so the very small risk of HIV being passed to the infant is far greater than an infant in the U.S. dying as a result of unclean water or formula contamination.”
Ms. Parker-Smith also said the AAP recommendations seem thorough in helping pediatric practitioners counsel and support parents with HIV. “If I had a parent who is HIV+ walk in the door today wanting to breastfeed their infant, the AAP guidelines give me specific steps to make me feel confident in helping that parent breastfeed as safely as possible as well as providing education to assist that parent through the decision process,” she said.
Dr. Biala agreed, noting that the clinical report “very clearly delineates recommendations for different groups of people: those in labor or postpartum with undocumented HIV infection status, pregnant and postpartum people with HIV, those without HIV but at high risk of acquiring it, and those with suspected acute HIV infection while breastfeeding.” Dr. Biala said the report “provides concrete, detailed, and easy-to-follow guidance on comprehensive counseling, strategies to minimize risk of transmission, and infant screening timelines.”
How easily the guidelines can be implemented will depend on the existing resources at different institutions in the United States, Dr. Biala added.
“In hospitals and clinics that have, or could easily have, systems in place to ensure follow-up and regular assessment during breastfeeding, the guidelines could be implemented fairly quickly,” she said. “It might be more challenging in areas with inadequate or limited access to multidisciplinary team members, including HIV care providers and lactation consultants.”
The report did not use external funding, and the authors reported no disclosures. Dr. Abuogi and Ms. Parker-Smith have no disclosures.
aside from avoiding breastfeeding altogether, according to a new clinical report from the American Academy of Pediatrics (AAP).
“The risk of HIV transmission via breastfeeding from a parent with HIV who is receiving antiretroviral treatment (ART) and is virally suppressed is estimated to be less than 1%,” Lisa Abuogi, MD, an associate professor of pediatric infectious disease at the University of Colorado Anschutz Medical Campus, and her colleagues wrote in Pediatrics. For people living with HIV in the United States, however, the AAP recommends that “avoidance of breastfeeding is the only infant feeding option with 0% risk of HIV transmission.”
The authors go on to suggest that pediatricians “be prepared to offer a family-centered, nonjudgmental, harm reduction approach” to support people with HIV who do want to breastfeed and have sustained viral suppression. Parents with HIV who are not on ART or who do not have adequate viral suppression should be advised against breastfeeding, the report states. Members of the AAP Committee on Pediatric and Adolescent HIV and of the AAP Section on Breastfeeding coauthored the clinical report.
“The new guidelines emphasize the importance of patient-centered counseling as the foundation for shared decision-making, allowing patients and pediatric providers to make feeding decisions together and for the first time really giving support to people with HIV in the U.S. who want to breastfeed,” Danna Biala, MD, MS, an attending pediatrician at Children’s Hospital at Montefiore and an assistant professor at Albert Einstein College of Medicine, told MDedge News.
Dr. Biala was not involved in the development of the report, but she said the AAP’s guidance reflects the recent shift in the stance of the Centers for Disease Control and Prevention (CDC) regarding breastfeeding among people who are HIV+. Recommendations from the CDC and the U.S. Department of Health and Human Services (HHS) were updated in 2023.
“I’m glad that the AAP is putting out guidelines on infant feeding for people with HIV,” Dr. Biala said. “For so long in the U.S., pediatricians have been advising all mothers with HIV to avoid breastfeeding, believing that the risk of transmission outweighed the benefits of breastfeeding.”
The updated guidance from HHS in 2023 “was revolutionary in supporting people with HIV in low-risk situations who want to breastfeed,” Dr. Biala said, but “clear protocols for monitoring and follow-up were not in place,” which these AAP guidelines help address.
Prior Discordance Between Global, U.S. Guidance
An estimated 5,000 people with HIV give birth each year in the United States, and up to one third of pregnant people with HIV may be unaware of their HIV status, the AAP report notes. Pediatric healthcare professionals in the United States therefore need to be aware of recommendations related to HIV testing of pregnant people and of counseling the feeding of infants exposed to HIV. The report recommends opt-out HIV testing at the first prenatal visit and then possibly retesting in the third trimester in areas with high HIV incidence or for people at high risk for HIV or with signs or symptoms of acute HIV infection.
The report also highlights the health benefits of breastfeeding to both the infant and the breastfeeding parent, but notes the CDC’s historical recommendation against breastfeeding for people who are HIV+. The WHO, meanwhile, began recommending in 2016 that infants be breastfed through 12 to 24 months old if the parent was on ART and/or the infant was receiving antiretroviral (ARV) prophylaxis, since research showed those treatments were effective in reducing transmission risk.
Still, an estimated 30% of perinatal HIV transmission occurs via breastfeeding worldwide, primarily from people with HIV who are not on ART or are not adequately virally suppressed. Without parental ART or infant ARV prophylaxis, HIV transmission risk to infants via breastfeeding is highest, about 5%-6%, in the first 4-6 weeks of life. Risk then declines to about 0.9% a month thereafter. The AAP report goes on to describe factors that increase or decrease the likelihood of transmission during breastfeeding, but it notes that neither ART in the breastfeeding person nor ARV prophylaxis in the infant completely eliminates the risk of HIV transmission during breastfeeding. There have been rare cases where transmission occurred despite viral suppression in the person with HIV.
Among the reasons people with HIV have expressed a desire to breastfeed are wanting to bond with their infant, wanting to provide optimal nutrition and health benefits to their baby, and meeting cultural expectations, including the desire not to disclose their HIV infection status to family or friends by virtue of not breastfeeding.
“Among immigrant and refugee populations, the discordance between infant feeding guidelines in the United States and their country of birth may result in confusion, especially among parents who breastfed previous infants,” the AAP report also notes. Further, not breastfeeding could compound health disparities already more likely to be present among those living with HIV.
Discussions about infant feeding with parents with HIV should therefore “begin as early as possible and involve a multidisciplinary team that might include the pediatric primary care provider (once identified), a pediatric HIV expert, the breastfeeding parent’s HIV care and obstetric providers, and lactation consultants,” the report states. ”The parent’s motivations for breastfeeding should be explored and counseling provided on the risks and benefits of each feeding option, including breastfeeding, formula feeding, or certified, banked donor human milk.” The statement emphasizes the need for providing counseling in a “non-judgmental, respectful way, recognizing potential drivers for their decisions such as avoidance of stigma, prior experience with breastfeeding, and cultural contributors.”
Clear Recommendations Can Help Providers
The AAP’s statement that “replacement feeding (with formula or certified, banked donor human milk) is the only option that is 100% certain to prevent postnatal transmission of HIV” feels like it takes a “more conservative or discouraging approach” to breastfeeding than the CDC or WHO guidelines, Alissa Parker-Smith, APRN, DNP, CPNP-PC, IBCLC, a nurse practitioner and lactation consultant at PrimaryPlus, a Federally Qualified Health Clinic in Ashland, Kentucky, told MDedge. But she said they do clearly align with the CDC guidelines, and their differences from the WHO guidelines make sense in light of the different populations served by the WHO versus the U.S. agencies.
“Unclean water for formula preparation and a reduced or lack of access to formula in general can lead to many other risks of death for the infant other than the very small risk of HIV infection from breastfeeding from an HIV+ parent,” Ms. Parker-Smith said. “In the U.S. we generally have consistent access to clean water and safe formula as well as social structures to help families have access to formula, so the very small risk of HIV being passed to the infant is far greater than an infant in the U.S. dying as a result of unclean water or formula contamination.”
Ms. Parker-Smith also said the AAP recommendations seem thorough in helping pediatric practitioners counsel and support parents with HIV. “If I had a parent who is HIV+ walk in the door today wanting to breastfeed their infant, the AAP guidelines give me specific steps to make me feel confident in helping that parent breastfeed as safely as possible as well as providing education to assist that parent through the decision process,” she said.
Dr. Biala agreed, noting that the clinical report “very clearly delineates recommendations for different groups of people: those in labor or postpartum with undocumented HIV infection status, pregnant and postpartum people with HIV, those without HIV but at high risk of acquiring it, and those with suspected acute HIV infection while breastfeeding.” Dr. Biala said the report “provides concrete, detailed, and easy-to-follow guidance on comprehensive counseling, strategies to minimize risk of transmission, and infant screening timelines.”
How easily the guidelines can be implemented will depend on the existing resources at different institutions in the United States, Dr. Biala added.
“In hospitals and clinics that have, or could easily have, systems in place to ensure follow-up and regular assessment during breastfeeding, the guidelines could be implemented fairly quickly,” she said. “It might be more challenging in areas with inadequate or limited access to multidisciplinary team members, including HIV care providers and lactation consultants.”
The report did not use external funding, and the authors reported no disclosures. Dr. Abuogi and Ms. Parker-Smith have no disclosures.
aside from avoiding breastfeeding altogether, according to a new clinical report from the American Academy of Pediatrics (AAP).
“The risk of HIV transmission via breastfeeding from a parent with HIV who is receiving antiretroviral treatment (ART) and is virally suppressed is estimated to be less than 1%,” Lisa Abuogi, MD, an associate professor of pediatric infectious disease at the University of Colorado Anschutz Medical Campus, and her colleagues wrote in Pediatrics. For people living with HIV in the United States, however, the AAP recommends that “avoidance of breastfeeding is the only infant feeding option with 0% risk of HIV transmission.”
The authors go on to suggest that pediatricians “be prepared to offer a family-centered, nonjudgmental, harm reduction approach” to support people with HIV who do want to breastfeed and have sustained viral suppression. Parents with HIV who are not on ART or who do not have adequate viral suppression should be advised against breastfeeding, the report states. Members of the AAP Committee on Pediatric and Adolescent HIV and of the AAP Section on Breastfeeding coauthored the clinical report.
“The new guidelines emphasize the importance of patient-centered counseling as the foundation for shared decision-making, allowing patients and pediatric providers to make feeding decisions together and for the first time really giving support to people with HIV in the U.S. who want to breastfeed,” Danna Biala, MD, MS, an attending pediatrician at Children’s Hospital at Montefiore and an assistant professor at Albert Einstein College of Medicine, told MDedge News.
Dr. Biala was not involved in the development of the report, but she said the AAP’s guidance reflects the recent shift in the stance of the Centers for Disease Control and Prevention (CDC) regarding breastfeeding among people who are HIV+. Recommendations from the CDC and the U.S. Department of Health and Human Services (HHS) were updated in 2023.
“I’m glad that the AAP is putting out guidelines on infant feeding for people with HIV,” Dr. Biala said. “For so long in the U.S., pediatricians have been advising all mothers with HIV to avoid breastfeeding, believing that the risk of transmission outweighed the benefits of breastfeeding.”
The updated guidance from HHS in 2023 “was revolutionary in supporting people with HIV in low-risk situations who want to breastfeed,” Dr. Biala said, but “clear protocols for monitoring and follow-up were not in place,” which these AAP guidelines help address.
Prior Discordance Between Global, U.S. Guidance
An estimated 5,000 people with HIV give birth each year in the United States, and up to one third of pregnant people with HIV may be unaware of their HIV status, the AAP report notes. Pediatric healthcare professionals in the United States therefore need to be aware of recommendations related to HIV testing of pregnant people and of counseling the feeding of infants exposed to HIV. The report recommends opt-out HIV testing at the first prenatal visit and then possibly retesting in the third trimester in areas with high HIV incidence or for people at high risk for HIV or with signs or symptoms of acute HIV infection.
The report also highlights the health benefits of breastfeeding to both the infant and the breastfeeding parent, but notes the CDC’s historical recommendation against breastfeeding for people who are HIV+. The WHO, meanwhile, began recommending in 2016 that infants be breastfed through 12 to 24 months old if the parent was on ART and/or the infant was receiving antiretroviral (ARV) prophylaxis, since research showed those treatments were effective in reducing transmission risk.
Still, an estimated 30% of perinatal HIV transmission occurs via breastfeeding worldwide, primarily from people with HIV who are not on ART or are not adequately virally suppressed. Without parental ART or infant ARV prophylaxis, HIV transmission risk to infants via breastfeeding is highest, about 5%-6%, in the first 4-6 weeks of life. Risk then declines to about 0.9% a month thereafter. The AAP report goes on to describe factors that increase or decrease the likelihood of transmission during breastfeeding, but it notes that neither ART in the breastfeeding person nor ARV prophylaxis in the infant completely eliminates the risk of HIV transmission during breastfeeding. There have been rare cases where transmission occurred despite viral suppression in the person with HIV.
Among the reasons people with HIV have expressed a desire to breastfeed are wanting to bond with their infant, wanting to provide optimal nutrition and health benefits to their baby, and meeting cultural expectations, including the desire not to disclose their HIV infection status to family or friends by virtue of not breastfeeding.
“Among immigrant and refugee populations, the discordance between infant feeding guidelines in the United States and their country of birth may result in confusion, especially among parents who breastfed previous infants,” the AAP report also notes. Further, not breastfeeding could compound health disparities already more likely to be present among those living with HIV.
Discussions about infant feeding with parents with HIV should therefore “begin as early as possible and involve a multidisciplinary team that might include the pediatric primary care provider (once identified), a pediatric HIV expert, the breastfeeding parent’s HIV care and obstetric providers, and lactation consultants,” the report states. ”The parent’s motivations for breastfeeding should be explored and counseling provided on the risks and benefits of each feeding option, including breastfeeding, formula feeding, or certified, banked donor human milk.” The statement emphasizes the need for providing counseling in a “non-judgmental, respectful way, recognizing potential drivers for their decisions such as avoidance of stigma, prior experience with breastfeeding, and cultural contributors.”
Clear Recommendations Can Help Providers
The AAP’s statement that “replacement feeding (with formula or certified, banked donor human milk) is the only option that is 100% certain to prevent postnatal transmission of HIV” feels like it takes a “more conservative or discouraging approach” to breastfeeding than the CDC or WHO guidelines, Alissa Parker-Smith, APRN, DNP, CPNP-PC, IBCLC, a nurse practitioner and lactation consultant at PrimaryPlus, a Federally Qualified Health Clinic in Ashland, Kentucky, told MDedge. But she said they do clearly align with the CDC guidelines, and their differences from the WHO guidelines make sense in light of the different populations served by the WHO versus the U.S. agencies.
“Unclean water for formula preparation and a reduced or lack of access to formula in general can lead to many other risks of death for the infant other than the very small risk of HIV infection from breastfeeding from an HIV+ parent,” Ms. Parker-Smith said. “In the U.S. we generally have consistent access to clean water and safe formula as well as social structures to help families have access to formula, so the very small risk of HIV being passed to the infant is far greater than an infant in the U.S. dying as a result of unclean water or formula contamination.”
Ms. Parker-Smith also said the AAP recommendations seem thorough in helping pediatric practitioners counsel and support parents with HIV. “If I had a parent who is HIV+ walk in the door today wanting to breastfeed their infant, the AAP guidelines give me specific steps to make me feel confident in helping that parent breastfeed as safely as possible as well as providing education to assist that parent through the decision process,” she said.
Dr. Biala agreed, noting that the clinical report “very clearly delineates recommendations for different groups of people: those in labor or postpartum with undocumented HIV infection status, pregnant and postpartum people with HIV, those without HIV but at high risk of acquiring it, and those with suspected acute HIV infection while breastfeeding.” Dr. Biala said the report “provides concrete, detailed, and easy-to-follow guidance on comprehensive counseling, strategies to minimize risk of transmission, and infant screening timelines.”
How easily the guidelines can be implemented will depend on the existing resources at different institutions in the United States, Dr. Biala added.
“In hospitals and clinics that have, or could easily have, systems in place to ensure follow-up and regular assessment during breastfeeding, the guidelines could be implemented fairly quickly,” she said. “It might be more challenging in areas with inadequate or limited access to multidisciplinary team members, including HIV care providers and lactation consultants.”
The report did not use external funding, and the authors reported no disclosures. Dr. Abuogi and Ms. Parker-Smith have no disclosures.
FROM PEDIATRICS
An 8-year-old girl presented with papules on her bilateral eyelid margins
, with an equal distribution across genders and ethnicities.1 It is caused by mutations in the ECM1 gene2 on chromosome 1q21. This leads to the abnormal deposition of hyaline material in various tissues across different organ systems, with the classic manifestations known as the “string of pearls” sign and a hoarse cry or voice.
The rarity of lipoid proteinosis often leads to challenges in diagnosis. Particularly when deviating from the common association with consanguinity, the potential for de novo mutations or a broader genetic variability in disease expression is highlighted. Our patient presents with symptoms that are pathognomonic to LP with moniliform blepharosis and hoarseness of the voice, in addition to scarring of the extremities.
Other common clinical manifestations in patients with LP include cobblestoning of the mucosa; hyperkeratosis of the elbows, knees, and hands; and calcification of the amygdala with neuroimaging.3
Genetic testing that identifies a loss-of-function mutation in ECM1 offers diagnostic confirmation. Patients often need multidisciplinary care involving dermatology; ear, nose, throat; neurology; and genetics. Treatment of LP is mostly symptomatic with unsatisfactory resolution of cutaneous changes, with retinoids such as acitretin used as the first-line option and surgery as a consideration for laryngeal hyaline deposits.2 Although LP can affect different organ systems, patients tend to have a normal lifespan.
LP is a rare disorder that dermatologists often learn about during textbook sessions or didactics in residency but do not see in practice for decades, or if ever. This case highlights the need to review the classic presentations of rare conditions.
This case and the photos were submitted by Ms. Chang, BS, Western University of Health Sciences, College of Osteopathic Medicine, Pomona, California; Dr. Connie Chang, Verdugo Dermatology, Glendale, California; and Dr. Yuchieh Kathryn Chang, MD Anderson Cancer Center, Houston, Texas. The column was edited by Donna Bilu Martin, MD.
Dr. Bilu Martin is a board-certified dermatologist in private practice at Premier Dermatology, MD, in Aventura, Florida. More diagnostic cases are available at mdedge.com/dermatology. To submit a case for possible publication, send an email to [email protected].
References
1. Mcgrath JA. Handb Clin Neurol. 2015:132:317-22. doi: 10.1016/B978-0-444-62702-5.00023-8.
2. Hamada Tet al. Hum Mol Genet. 2002 Apr 1;11(7):833-40. doi: 10.1093/hmg/11.7.833.
3. Frenkel B et al. Clin Oral Investig. 2017 Sep;21(7):2245-51 doi: 10.1007/s00784-016-2017-7.
, with an equal distribution across genders and ethnicities.1 It is caused by mutations in the ECM1 gene2 on chromosome 1q21. This leads to the abnormal deposition of hyaline material in various tissues across different organ systems, with the classic manifestations known as the “string of pearls” sign and a hoarse cry or voice.
The rarity of lipoid proteinosis often leads to challenges in diagnosis. Particularly when deviating from the common association with consanguinity, the potential for de novo mutations or a broader genetic variability in disease expression is highlighted. Our patient presents with symptoms that are pathognomonic to LP with moniliform blepharosis and hoarseness of the voice, in addition to scarring of the extremities.
Other common clinical manifestations in patients with LP include cobblestoning of the mucosa; hyperkeratosis of the elbows, knees, and hands; and calcification of the amygdala with neuroimaging.3
Genetic testing that identifies a loss-of-function mutation in ECM1 offers diagnostic confirmation. Patients often need multidisciplinary care involving dermatology; ear, nose, throat; neurology; and genetics. Treatment of LP is mostly symptomatic with unsatisfactory resolution of cutaneous changes, with retinoids such as acitretin used as the first-line option and surgery as a consideration for laryngeal hyaline deposits.2 Although LP can affect different organ systems, patients tend to have a normal lifespan.
LP is a rare disorder that dermatologists often learn about during textbook sessions or didactics in residency but do not see in practice for decades, or if ever. This case highlights the need to review the classic presentations of rare conditions.
This case and the photos were submitted by Ms. Chang, BS, Western University of Health Sciences, College of Osteopathic Medicine, Pomona, California; Dr. Connie Chang, Verdugo Dermatology, Glendale, California; and Dr. Yuchieh Kathryn Chang, MD Anderson Cancer Center, Houston, Texas. The column was edited by Donna Bilu Martin, MD.
Dr. Bilu Martin is a board-certified dermatologist in private practice at Premier Dermatology, MD, in Aventura, Florida. More diagnostic cases are available at mdedge.com/dermatology. To submit a case for possible publication, send an email to [email protected].
References
1. Mcgrath JA. Handb Clin Neurol. 2015:132:317-22. doi: 10.1016/B978-0-444-62702-5.00023-8.
2. Hamada Tet al. Hum Mol Genet. 2002 Apr 1;11(7):833-40. doi: 10.1093/hmg/11.7.833.
3. Frenkel B et al. Clin Oral Investig. 2017 Sep;21(7):2245-51 doi: 10.1007/s00784-016-2017-7.
, with an equal distribution across genders and ethnicities.1 It is caused by mutations in the ECM1 gene2 on chromosome 1q21. This leads to the abnormal deposition of hyaline material in various tissues across different organ systems, with the classic manifestations known as the “string of pearls” sign and a hoarse cry or voice.
The rarity of lipoid proteinosis often leads to challenges in diagnosis. Particularly when deviating from the common association with consanguinity, the potential for de novo mutations or a broader genetic variability in disease expression is highlighted. Our patient presents with symptoms that are pathognomonic to LP with moniliform blepharosis and hoarseness of the voice, in addition to scarring of the extremities.
Other common clinical manifestations in patients with LP include cobblestoning of the mucosa; hyperkeratosis of the elbows, knees, and hands; and calcification of the amygdala with neuroimaging.3
Genetic testing that identifies a loss-of-function mutation in ECM1 offers diagnostic confirmation. Patients often need multidisciplinary care involving dermatology; ear, nose, throat; neurology; and genetics. Treatment of LP is mostly symptomatic with unsatisfactory resolution of cutaneous changes, with retinoids such as acitretin used as the first-line option and surgery as a consideration for laryngeal hyaline deposits.2 Although LP can affect different organ systems, patients tend to have a normal lifespan.
LP is a rare disorder that dermatologists often learn about during textbook sessions or didactics in residency but do not see in practice for decades, or if ever. This case highlights the need to review the classic presentations of rare conditions.
This case and the photos were submitted by Ms. Chang, BS, Western University of Health Sciences, College of Osteopathic Medicine, Pomona, California; Dr. Connie Chang, Verdugo Dermatology, Glendale, California; and Dr. Yuchieh Kathryn Chang, MD Anderson Cancer Center, Houston, Texas. The column was edited by Donna Bilu Martin, MD.
Dr. Bilu Martin is a board-certified dermatologist in private practice at Premier Dermatology, MD, in Aventura, Florida. More diagnostic cases are available at mdedge.com/dermatology. To submit a case for possible publication, send an email to [email protected].
References
1. Mcgrath JA. Handb Clin Neurol. 2015:132:317-22. doi: 10.1016/B978-0-444-62702-5.00023-8.
2. Hamada Tet al. Hum Mol Genet. 2002 Apr 1;11(7):833-40. doi: 10.1093/hmg/11.7.833.
3. Frenkel B et al. Clin Oral Investig. 2017 Sep;21(7):2245-51 doi: 10.1007/s00784-016-2017-7.
An 8-year-old girl with no significant past medical history presented with papules on her bilateral eyelid margins that had developed over the past few months. The papules were slightly itchy but otherwise asymptomatic. Notably, the patient has always had a hoarse voice.
Psychiatric Comorbidity Prevalent in Eating Disorders
NEW YORK — Psychiatric comorbidities are highly prevalent in patients with eating disorders (EDs), a large study showed.
In a propensity-matched cohort of young adults with and without EDs, a wide variety of psychiatric disorders including depression and anxiety, as well as cannabis and alcohol use disorders, were more common in those with EDs, investigators found.
Comorbid psychiatric disorders should be “top of mind when working with someone with an eating disorder. If you are able to treat the comorbid psychiatric conditions, they might have a better recovery from the eating disorder,” study investigator Angela Liu, MD, with Northwell Health at Zucker Hillside Hospital, Glen Oaks, New York, told this news organization.
The findings were presented at the annual meeting of the American Psychiatric Association.
Data Gap
As previously reported by this news organization, more than one in five children worldwide are at risk for an ED and US medical admissions for adolescents with restrictive EDs more than doubled during the pandemic.
Yet there remains a “gap in the literature” about the prevalence of comorbid psychiatric conditions in people with EDs, specifically in young people, Dr. Liu explained.
“To our knowledge, this is the first study using a real-world, multistate administrative dataset to estimate the prevalence of psychiatric comorbidities in young people diagnosed with an eating disorder,” Dr. Liu said.
Using the TriNetX database, the researchers identified through ICD-10 codes 14,524 individuals with EDs (mean age, 15.9 years; 79% women) and 110,051 without EDs who were receiving antidepressants (mean age, 17.8 years; 65% women).
“There was a much higher prevalence of almost all other psychiatric conditions in those with an eating disorder compared to the general psychiatry population,” coinvestigator Binx Y. Lin, MD, MSc, with Virginia Tech Carilion School of Medicine in Roanoke, Virginia, told this news organization.
In the baseline comparison (before matching), psychiatric disorders seen more often in adults with than in those without EDs included (but were not limited to) mood disorders (51% vs 23%), generalized anxiety disorder (GAD; 30% vs 8%), posttraumatic stress disorder (PTSD; 10% vs 2%), obsessive-compulsive disorder (OCD; 8% vs 1%), panic disorder (6% vs 2%), substance use disorder (8% vs 5%), and adjustment disorders (5% vs 2%).
The results held after propensity score matching, with numerous psychiatric conditions significantly (P < .001) more prevalent in the ED cohort.
Understanding the burden of comorbid psychiatric disorders in young people with EDs is important to design comprehensive, evidence-based interventions, the researchers said.
Providing perspective on this topic, Petros Levounis, MD, professor and chair, Department of Psychiatry, Rutgers New Jersey Medical School, Newark, New Jersey, noted that “comorbidity between substance use disorders and other psychiatric disorders has both been grossly underestimated and grossly overestimated.
“I go around the country and see rehab programs, and there are people that very strongly believe that if you stop using the drugs, you won’t have problems with depression or anxiety or whatever,” Dr. Levounis, immediate past president of the APA, shared with this news organization.
“Others say they have never seen somebody who’s addicted to something that doesn’t also have some other psychiatric disorders and if you just scratch the surface, you always find some other psychological or psychiatric problem lying behind. Neither of them are true,” he cautioned.
Dr. Levounis said it’s important to recognize that “some people with addiction will also have another psychiatric disorder. But clearly there are people who just have a mental illness without addiction, and there are clearly people who will just have addiction without other mental illness.”
This research had no commercial funding and was supported in part by the American Psychiatric Association Research Fellowship. Dr. Liu, Dr. Lin, and Dr. Levounis had no relevant disclosures.
A version of this article appeared on Medscape.com .
NEW YORK — Psychiatric comorbidities are highly prevalent in patients with eating disorders (EDs), a large study showed.
In a propensity-matched cohort of young adults with and without EDs, a wide variety of psychiatric disorders including depression and anxiety, as well as cannabis and alcohol use disorders, were more common in those with EDs, investigators found.
Comorbid psychiatric disorders should be “top of mind when working with someone with an eating disorder. If you are able to treat the comorbid psychiatric conditions, they might have a better recovery from the eating disorder,” study investigator Angela Liu, MD, with Northwell Health at Zucker Hillside Hospital, Glen Oaks, New York, told this news organization.
The findings were presented at the annual meeting of the American Psychiatric Association.
Data Gap
As previously reported by this news organization, more than one in five children worldwide are at risk for an ED and US medical admissions for adolescents with restrictive EDs more than doubled during the pandemic.
Yet there remains a “gap in the literature” about the prevalence of comorbid psychiatric conditions in people with EDs, specifically in young people, Dr. Liu explained.
“To our knowledge, this is the first study using a real-world, multistate administrative dataset to estimate the prevalence of psychiatric comorbidities in young people diagnosed with an eating disorder,” Dr. Liu said.
Using the TriNetX database, the researchers identified through ICD-10 codes 14,524 individuals with EDs (mean age, 15.9 years; 79% women) and 110,051 without EDs who were receiving antidepressants (mean age, 17.8 years; 65% women).
“There was a much higher prevalence of almost all other psychiatric conditions in those with an eating disorder compared to the general psychiatry population,” coinvestigator Binx Y. Lin, MD, MSc, with Virginia Tech Carilion School of Medicine in Roanoke, Virginia, told this news organization.
In the baseline comparison (before matching), psychiatric disorders seen more often in adults with than in those without EDs included (but were not limited to) mood disorders (51% vs 23%), generalized anxiety disorder (GAD; 30% vs 8%), posttraumatic stress disorder (PTSD; 10% vs 2%), obsessive-compulsive disorder (OCD; 8% vs 1%), panic disorder (6% vs 2%), substance use disorder (8% vs 5%), and adjustment disorders (5% vs 2%).
The results held after propensity score matching, with numerous psychiatric conditions significantly (P < .001) more prevalent in the ED cohort.
Understanding the burden of comorbid psychiatric disorders in young people with EDs is important to design comprehensive, evidence-based interventions, the researchers said.
Providing perspective on this topic, Petros Levounis, MD, professor and chair, Department of Psychiatry, Rutgers New Jersey Medical School, Newark, New Jersey, noted that “comorbidity between substance use disorders and other psychiatric disorders has both been grossly underestimated and grossly overestimated.
“I go around the country and see rehab programs, and there are people that very strongly believe that if you stop using the drugs, you won’t have problems with depression or anxiety or whatever,” Dr. Levounis, immediate past president of the APA, shared with this news organization.
“Others say they have never seen somebody who’s addicted to something that doesn’t also have some other psychiatric disorders and if you just scratch the surface, you always find some other psychological or psychiatric problem lying behind. Neither of them are true,” he cautioned.
Dr. Levounis said it’s important to recognize that “some people with addiction will also have another psychiatric disorder. But clearly there are people who just have a mental illness without addiction, and there are clearly people who will just have addiction without other mental illness.”
This research had no commercial funding and was supported in part by the American Psychiatric Association Research Fellowship. Dr. Liu, Dr. Lin, and Dr. Levounis had no relevant disclosures.
A version of this article appeared on Medscape.com .
NEW YORK — Psychiatric comorbidities are highly prevalent in patients with eating disorders (EDs), a large study showed.
In a propensity-matched cohort of young adults with and without EDs, a wide variety of psychiatric disorders including depression and anxiety, as well as cannabis and alcohol use disorders, were more common in those with EDs, investigators found.
Comorbid psychiatric disorders should be “top of mind when working with someone with an eating disorder. If you are able to treat the comorbid psychiatric conditions, they might have a better recovery from the eating disorder,” study investigator Angela Liu, MD, with Northwell Health at Zucker Hillside Hospital, Glen Oaks, New York, told this news organization.
The findings were presented at the annual meeting of the American Psychiatric Association.
Data Gap
As previously reported by this news organization, more than one in five children worldwide are at risk for an ED and US medical admissions for adolescents with restrictive EDs more than doubled during the pandemic.
Yet there remains a “gap in the literature” about the prevalence of comorbid psychiatric conditions in people with EDs, specifically in young people, Dr. Liu explained.
“To our knowledge, this is the first study using a real-world, multistate administrative dataset to estimate the prevalence of psychiatric comorbidities in young people diagnosed with an eating disorder,” Dr. Liu said.
Using the TriNetX database, the researchers identified through ICD-10 codes 14,524 individuals with EDs (mean age, 15.9 years; 79% women) and 110,051 without EDs who were receiving antidepressants (mean age, 17.8 years; 65% women).
“There was a much higher prevalence of almost all other psychiatric conditions in those with an eating disorder compared to the general psychiatry population,” coinvestigator Binx Y. Lin, MD, MSc, with Virginia Tech Carilion School of Medicine in Roanoke, Virginia, told this news organization.
In the baseline comparison (before matching), psychiatric disorders seen more often in adults with than in those without EDs included (but were not limited to) mood disorders (51% vs 23%), generalized anxiety disorder (GAD; 30% vs 8%), posttraumatic stress disorder (PTSD; 10% vs 2%), obsessive-compulsive disorder (OCD; 8% vs 1%), panic disorder (6% vs 2%), substance use disorder (8% vs 5%), and adjustment disorders (5% vs 2%).
The results held after propensity score matching, with numerous psychiatric conditions significantly (P < .001) more prevalent in the ED cohort.
Understanding the burden of comorbid psychiatric disorders in young people with EDs is important to design comprehensive, evidence-based interventions, the researchers said.
Providing perspective on this topic, Petros Levounis, MD, professor and chair, Department of Psychiatry, Rutgers New Jersey Medical School, Newark, New Jersey, noted that “comorbidity between substance use disorders and other psychiatric disorders has both been grossly underestimated and grossly overestimated.
“I go around the country and see rehab programs, and there are people that very strongly believe that if you stop using the drugs, you won’t have problems with depression or anxiety or whatever,” Dr. Levounis, immediate past president of the APA, shared with this news organization.
“Others say they have never seen somebody who’s addicted to something that doesn’t also have some other psychiatric disorders and if you just scratch the surface, you always find some other psychological or psychiatric problem lying behind. Neither of them are true,” he cautioned.
Dr. Levounis said it’s important to recognize that “some people with addiction will also have another psychiatric disorder. But clearly there are people who just have a mental illness without addiction, and there are clearly people who will just have addiction without other mental illness.”
This research had no commercial funding and was supported in part by the American Psychiatric Association Research Fellowship. Dr. Liu, Dr. Lin, and Dr. Levounis had no relevant disclosures.
A version of this article appeared on Medscape.com .
Resource Menu Gives Choice to Caregivers Struggling to Meet Basic Needs
Screenings may not be the way to get needed resources to children and their caregivers, according to new research presented at the annual meeting of the Pediatric Academic Societies (PAS).
Caregivers and parents who were asked if they wanted assistance in several areas of need, including transportation and childcare, were nearly twice as likely to say they wanted such help than those who received a screening on current hardships. Generally, each questionnaire is administered in front of their children in primary care or pediatric hospital settings.
“Families have a lot of concern about being seen a different way by their healthcare team, being seen as unfit, and having child protective services involved in their childcare for issues related to poverty,” said Danielle Cullen, MD, a pediatric emergency medicine specialist at Children’s Hospital of Philadelphia (CHOP) and assistant professor of pediatrics at the University of Pennsylvania in Philadelphia.
Dr. Cullen and her colleagues analyzed data from nearly 4000 caregivers of children up to age 21 at emergency departments or primary care clinics at CHOP between 2021 and 2023.
Caregivers were randomly assigned to one of three arms — screening with a version of WE CARE (Well Child Care, Evaluation, Community Resources, Advocacy, Referral, Education), use of an online menu of options for help in areas like housing, or neither approach.
Caregivers in all three arms received a map of resources and a follow-up text from a resource navigator to assist them as needed.
Nearly 40% of caregivers who presented with the digital menu said they wanted resources compared with 29% of those who were screened (P < .001). Non-native English speakers given the menu were 2.5 times more likely to say yes to resources compared with those who were screened.
“We need to be thoughtful about these mandates to screen for social determinants of health: It’s not that straightforward,” said Esther K. Chung, MD, a pediatrician and professor of pediatrics at the University of Washington Medicine in Seattle, who was not involved in the study. “What we’re getting from this study is that patients want choice, and the menu provides them choice.”
Dr. Cullen said the menu option allows caregivers to make choices based on their priorities and not on whether they meet the screening thresholds for need.
While some health clinics utilize tablet forms for screenings to offer more privacy with questions, asking direct questions about income, food insecurity, and housing stability can be stigmatizing, Dr. Cullen said.
“Screening positive for social risk doesn’t mean that you actually want resources, and on the flip side, the literature shows that about half of the people who screen negative want resources,” she said.
Dr. Cullen and her team also conducted follow-up interviews with caregivers and found many feared that their clinician would assume a medical condition was connected to living conditions. They also had concerns about insurance companies gaining access to the data and using it to deny coverage or raise costs.
Spanish-speaking caregivers cited fears about their immigration status, experiences of discrimination, and language barriers when trying to access resources.
Participants said a few key strategies could make screening less intimidating, such as abstaining from screening during a serious medical visit, asking for consent to record answers in medical records, and communicating in an empathetic manner.
“Some families are a bit surprised when we ask about things like housing and food insecurity, but I think as long as we contextualize it, we can minimize the stigma associated with it,” Dr. Chung said. “That takes quite a bit of nuance and skill.”
The study was funded by the William T. Grant Foundation and the Emergency Medicine Foundation. The authors reported no disclosures.
A version of this article appeared on Medscape.com.
Screenings may not be the way to get needed resources to children and their caregivers, according to new research presented at the annual meeting of the Pediatric Academic Societies (PAS).
Caregivers and parents who were asked if they wanted assistance in several areas of need, including transportation and childcare, were nearly twice as likely to say they wanted such help than those who received a screening on current hardships. Generally, each questionnaire is administered in front of their children in primary care or pediatric hospital settings.
“Families have a lot of concern about being seen a different way by their healthcare team, being seen as unfit, and having child protective services involved in their childcare for issues related to poverty,” said Danielle Cullen, MD, a pediatric emergency medicine specialist at Children’s Hospital of Philadelphia (CHOP) and assistant professor of pediatrics at the University of Pennsylvania in Philadelphia.
Dr. Cullen and her colleagues analyzed data from nearly 4000 caregivers of children up to age 21 at emergency departments or primary care clinics at CHOP between 2021 and 2023.
Caregivers were randomly assigned to one of three arms — screening with a version of WE CARE (Well Child Care, Evaluation, Community Resources, Advocacy, Referral, Education), use of an online menu of options for help in areas like housing, or neither approach.
Caregivers in all three arms received a map of resources and a follow-up text from a resource navigator to assist them as needed.
Nearly 40% of caregivers who presented with the digital menu said they wanted resources compared with 29% of those who were screened (P < .001). Non-native English speakers given the menu were 2.5 times more likely to say yes to resources compared with those who were screened.
“We need to be thoughtful about these mandates to screen for social determinants of health: It’s not that straightforward,” said Esther K. Chung, MD, a pediatrician and professor of pediatrics at the University of Washington Medicine in Seattle, who was not involved in the study. “What we’re getting from this study is that patients want choice, and the menu provides them choice.”
Dr. Cullen said the menu option allows caregivers to make choices based on their priorities and not on whether they meet the screening thresholds for need.
While some health clinics utilize tablet forms for screenings to offer more privacy with questions, asking direct questions about income, food insecurity, and housing stability can be stigmatizing, Dr. Cullen said.
“Screening positive for social risk doesn’t mean that you actually want resources, and on the flip side, the literature shows that about half of the people who screen negative want resources,” she said.
Dr. Cullen and her team also conducted follow-up interviews with caregivers and found many feared that their clinician would assume a medical condition was connected to living conditions. They also had concerns about insurance companies gaining access to the data and using it to deny coverage or raise costs.
Spanish-speaking caregivers cited fears about their immigration status, experiences of discrimination, and language barriers when trying to access resources.
Participants said a few key strategies could make screening less intimidating, such as abstaining from screening during a serious medical visit, asking for consent to record answers in medical records, and communicating in an empathetic manner.
“Some families are a bit surprised when we ask about things like housing and food insecurity, but I think as long as we contextualize it, we can minimize the stigma associated with it,” Dr. Chung said. “That takes quite a bit of nuance and skill.”
The study was funded by the William T. Grant Foundation and the Emergency Medicine Foundation. The authors reported no disclosures.
A version of this article appeared on Medscape.com.
Screenings may not be the way to get needed resources to children and their caregivers, according to new research presented at the annual meeting of the Pediatric Academic Societies (PAS).
Caregivers and parents who were asked if they wanted assistance in several areas of need, including transportation and childcare, were nearly twice as likely to say they wanted such help than those who received a screening on current hardships. Generally, each questionnaire is administered in front of their children in primary care or pediatric hospital settings.
“Families have a lot of concern about being seen a different way by their healthcare team, being seen as unfit, and having child protective services involved in their childcare for issues related to poverty,” said Danielle Cullen, MD, a pediatric emergency medicine specialist at Children’s Hospital of Philadelphia (CHOP) and assistant professor of pediatrics at the University of Pennsylvania in Philadelphia.
Dr. Cullen and her colleagues analyzed data from nearly 4000 caregivers of children up to age 21 at emergency departments or primary care clinics at CHOP between 2021 and 2023.
Caregivers were randomly assigned to one of three arms — screening with a version of WE CARE (Well Child Care, Evaluation, Community Resources, Advocacy, Referral, Education), use of an online menu of options for help in areas like housing, or neither approach.
Caregivers in all three arms received a map of resources and a follow-up text from a resource navigator to assist them as needed.
Nearly 40% of caregivers who presented with the digital menu said they wanted resources compared with 29% of those who were screened (P < .001). Non-native English speakers given the menu were 2.5 times more likely to say yes to resources compared with those who were screened.
“We need to be thoughtful about these mandates to screen for social determinants of health: It’s not that straightforward,” said Esther K. Chung, MD, a pediatrician and professor of pediatrics at the University of Washington Medicine in Seattle, who was not involved in the study. “What we’re getting from this study is that patients want choice, and the menu provides them choice.”
Dr. Cullen said the menu option allows caregivers to make choices based on their priorities and not on whether they meet the screening thresholds for need.
While some health clinics utilize tablet forms for screenings to offer more privacy with questions, asking direct questions about income, food insecurity, and housing stability can be stigmatizing, Dr. Cullen said.
“Screening positive for social risk doesn’t mean that you actually want resources, and on the flip side, the literature shows that about half of the people who screen negative want resources,” she said.
Dr. Cullen and her team also conducted follow-up interviews with caregivers and found many feared that their clinician would assume a medical condition was connected to living conditions. They also had concerns about insurance companies gaining access to the data and using it to deny coverage or raise costs.
Spanish-speaking caregivers cited fears about their immigration status, experiences of discrimination, and language barriers when trying to access resources.
Participants said a few key strategies could make screening less intimidating, such as abstaining from screening during a serious medical visit, asking for consent to record answers in medical records, and communicating in an empathetic manner.
“Some families are a bit surprised when we ask about things like housing and food insecurity, but I think as long as we contextualize it, we can minimize the stigma associated with it,” Dr. Chung said. “That takes quite a bit of nuance and skill.”
The study was funded by the William T. Grant Foundation and the Emergency Medicine Foundation. The authors reported no disclosures.
A version of this article appeared on Medscape.com.
FROM PAS 2024
Little Less Talk and a Lot More Action
No matter where one looks for the statistics, no matter what words one chooses to describe it, this country has a child and adolescent mental health crisis. Almost 20% of young people in the 3-17 age bracket have a mental, emotional, developmental, or behavioral disorder. COVID-19 has certainly exacerbated the problem, but the downward trend in the mental health of this nation has been going on for decades.
The voices calling for more services to address the problem are getting more numerous and louder. But, what exactly should those services look like and who should be delivering them?
When considered together, two recent research papers suggest that we should be venturing well beyond the usual mental health strategies if we are going to be successful in addressing the current crisis.
The first paper is an analysis by two psychologists who contend that our efforts to raise the awareness of mental issues may be contributing to the increase in reported mental health problems. The authors agree that more attention paid to mental health conditions can result in “more accurate reporting of previous under-recognized symptoms” and would seem to be a positive. However, the investigators also observe that when exposed to this flood of information, some individuals who are only experiencing minor distress may report their symptoms as mental problems. The authors of the paper have coined the term for this phenomenon as “prevalence inflation.” Their preliminary investigation suggests it may be much more common than once believed and they present numerous situations in which prevalence inflation seems to have occurred.
A New York Times article about this hypothesis reports on a British study in which nearly 30,000 teenagers were instructed by their teachers to “direct their attentions to the present moment” and utilize other mindfulness strategies. The educators had hoped that after 8 years of this indoctrination, the students’ mental health would have improved. The bottom line was that this mindfulness-based program was of no help and may have actually made things worse for a subgroup of students who were at greatest risk for mental health challenges.
Dr. Jack Andrews, one of the authors, feels that mindfulness training may encourage what he calls “co-rumination,” which he describes as “the kind of long, unresolved group discussion that churns up problems without finding solutions.” One has to wonder if “prevalence inflation” and “co-rumination,” if they do exist, may be playing a role in the hotly debated phenomenon some have termed “late-onset gender dysphoria.”
Never having been a fan of mindfulness training as an effective strategy, I am relieved to learn that serious investigators are finding evidence that supports my gut reaction.
If raising awareness, “education,” and group discussion aren’t working, and in some cases are actually contributing to the crisis, or at least making the data difficult to interpret, what should we be doing to turn this foundering ship around?
A second paper, coming from Taiwan, may provide an answer. Huey-Ling Chiang and fellow investigators have reported on a study of nearly two million children and adolescents in which they found improved performance in a variety of physical fitness challenges “was linked with a lower risk of mental health disorder.” The dose-dependent effect resulted in less anxiety and depressive disorders as well as less attention-deficit/hyperactivity disorder when cardio-respiratory, muscle endurance, and power indices improved.
There have been other observers who have suggested a link between physical fitness and improved mental health, but this Taiwanese study is by far one of the largest. And, the discovery of a dose-dependent effect makes it particularly convincing.
As I reviewed these two papers, I became increasingly frustrated because this is another example in which one of the answers is staring us in the face and we continue to do nothing more than talk about it.
We already know that physically active people are healthier both physically and mentally, but we do little more than talk. It may be helpful for some people to become a bit more self-aware. However, it is becoming increasingly clear that you can’t talk yourself into being mentally healthy without a concurrent effort to actually do the things that can improve your overall health, such as being physically active and adopting healthy sleep habits. A political advisor once said, “It’s the economy, stupid.” As a community interested in the health of our children and the adults they will become, we need to remind ourselves again, “It’s the old Mind-Body Thing, Stupid.” Our children need a little less talk and a lot more action.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at [email protected].
No matter where one looks for the statistics, no matter what words one chooses to describe it, this country has a child and adolescent mental health crisis. Almost 20% of young people in the 3-17 age bracket have a mental, emotional, developmental, or behavioral disorder. COVID-19 has certainly exacerbated the problem, but the downward trend in the mental health of this nation has been going on for decades.
The voices calling for more services to address the problem are getting more numerous and louder. But, what exactly should those services look like and who should be delivering them?
When considered together, two recent research papers suggest that we should be venturing well beyond the usual mental health strategies if we are going to be successful in addressing the current crisis.
The first paper is an analysis by two psychologists who contend that our efforts to raise the awareness of mental issues may be contributing to the increase in reported mental health problems. The authors agree that more attention paid to mental health conditions can result in “more accurate reporting of previous under-recognized symptoms” and would seem to be a positive. However, the investigators also observe that when exposed to this flood of information, some individuals who are only experiencing minor distress may report their symptoms as mental problems. The authors of the paper have coined the term for this phenomenon as “prevalence inflation.” Their preliminary investigation suggests it may be much more common than once believed and they present numerous situations in which prevalence inflation seems to have occurred.
A New York Times article about this hypothesis reports on a British study in which nearly 30,000 teenagers were instructed by their teachers to “direct their attentions to the present moment” and utilize other mindfulness strategies. The educators had hoped that after 8 years of this indoctrination, the students’ mental health would have improved. The bottom line was that this mindfulness-based program was of no help and may have actually made things worse for a subgroup of students who were at greatest risk for mental health challenges.
Dr. Jack Andrews, one of the authors, feels that mindfulness training may encourage what he calls “co-rumination,” which he describes as “the kind of long, unresolved group discussion that churns up problems without finding solutions.” One has to wonder if “prevalence inflation” and “co-rumination,” if they do exist, may be playing a role in the hotly debated phenomenon some have termed “late-onset gender dysphoria.”
Never having been a fan of mindfulness training as an effective strategy, I am relieved to learn that serious investigators are finding evidence that supports my gut reaction.
If raising awareness, “education,” and group discussion aren’t working, and in some cases are actually contributing to the crisis, or at least making the data difficult to interpret, what should we be doing to turn this foundering ship around?
A second paper, coming from Taiwan, may provide an answer. Huey-Ling Chiang and fellow investigators have reported on a study of nearly two million children and adolescents in which they found improved performance in a variety of physical fitness challenges “was linked with a lower risk of mental health disorder.” The dose-dependent effect resulted in less anxiety and depressive disorders as well as less attention-deficit/hyperactivity disorder when cardio-respiratory, muscle endurance, and power indices improved.
There have been other observers who have suggested a link between physical fitness and improved mental health, but this Taiwanese study is by far one of the largest. And, the discovery of a dose-dependent effect makes it particularly convincing.
As I reviewed these two papers, I became increasingly frustrated because this is another example in which one of the answers is staring us in the face and we continue to do nothing more than talk about it.
We already know that physically active people are healthier both physically and mentally, but we do little more than talk. It may be helpful for some people to become a bit more self-aware. However, it is becoming increasingly clear that you can’t talk yourself into being mentally healthy without a concurrent effort to actually do the things that can improve your overall health, such as being physically active and adopting healthy sleep habits. A political advisor once said, “It’s the economy, stupid.” As a community interested in the health of our children and the adults they will become, we need to remind ourselves again, “It’s the old Mind-Body Thing, Stupid.” Our children need a little less talk and a lot more action.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at [email protected].
No matter where one looks for the statistics, no matter what words one chooses to describe it, this country has a child and adolescent mental health crisis. Almost 20% of young people in the 3-17 age bracket have a mental, emotional, developmental, or behavioral disorder. COVID-19 has certainly exacerbated the problem, but the downward trend in the mental health of this nation has been going on for decades.
The voices calling for more services to address the problem are getting more numerous and louder. But, what exactly should those services look like and who should be delivering them?
When considered together, two recent research papers suggest that we should be venturing well beyond the usual mental health strategies if we are going to be successful in addressing the current crisis.
The first paper is an analysis by two psychologists who contend that our efforts to raise the awareness of mental issues may be contributing to the increase in reported mental health problems. The authors agree that more attention paid to mental health conditions can result in “more accurate reporting of previous under-recognized symptoms” and would seem to be a positive. However, the investigators also observe that when exposed to this flood of information, some individuals who are only experiencing minor distress may report their symptoms as mental problems. The authors of the paper have coined the term for this phenomenon as “prevalence inflation.” Their preliminary investigation suggests it may be much more common than once believed and they present numerous situations in which prevalence inflation seems to have occurred.
A New York Times article about this hypothesis reports on a British study in which nearly 30,000 teenagers were instructed by their teachers to “direct their attentions to the present moment” and utilize other mindfulness strategies. The educators had hoped that after 8 years of this indoctrination, the students’ mental health would have improved. The bottom line was that this mindfulness-based program was of no help and may have actually made things worse for a subgroup of students who were at greatest risk for mental health challenges.
Dr. Jack Andrews, one of the authors, feels that mindfulness training may encourage what he calls “co-rumination,” which he describes as “the kind of long, unresolved group discussion that churns up problems without finding solutions.” One has to wonder if “prevalence inflation” and “co-rumination,” if they do exist, may be playing a role in the hotly debated phenomenon some have termed “late-onset gender dysphoria.”
Never having been a fan of mindfulness training as an effective strategy, I am relieved to learn that serious investigators are finding evidence that supports my gut reaction.
If raising awareness, “education,” and group discussion aren’t working, and in some cases are actually contributing to the crisis, or at least making the data difficult to interpret, what should we be doing to turn this foundering ship around?
A second paper, coming from Taiwan, may provide an answer. Huey-Ling Chiang and fellow investigators have reported on a study of nearly two million children and adolescents in which they found improved performance in a variety of physical fitness challenges “was linked with a lower risk of mental health disorder.” The dose-dependent effect resulted in less anxiety and depressive disorders as well as less attention-deficit/hyperactivity disorder when cardio-respiratory, muscle endurance, and power indices improved.
There have been other observers who have suggested a link between physical fitness and improved mental health, but this Taiwanese study is by far one of the largest. And, the discovery of a dose-dependent effect makes it particularly convincing.
As I reviewed these two papers, I became increasingly frustrated because this is another example in which one of the answers is staring us in the face and we continue to do nothing more than talk about it.
We already know that physically active people are healthier both physically and mentally, but we do little more than talk. It may be helpful for some people to become a bit more self-aware. However, it is becoming increasingly clear that you can’t talk yourself into being mentally healthy without a concurrent effort to actually do the things that can improve your overall health, such as being physically active and adopting healthy sleep habits. A political advisor once said, “It’s the economy, stupid.” As a community interested in the health of our children and the adults they will become, we need to remind ourselves again, “It’s the old Mind-Body Thing, Stupid.” Our children need a little less talk and a lot more action.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at [email protected].
Specialists Are ‘Underwater’ With Some Insurance-Preferred Biosimilars
Editor’s note: This article is adapted from an explanatory statement that Dr. Feldman wrote for the Coalition of State Rheumatology Organizations (CSRO).
According to the Guinness Book of World records, the longest time someone has held their breath underwater voluntarily is 24 minutes and 37.36 seconds. While certainly an amazing feat, UnitedHealthcare, many of the Blues, and other national “payers” are expecting rheumatologists and other specialists to live “underwater” in order to take care of their patients. In other words, these insurance companies are mandating that specialists use certain provider-administered biosimilars whose acquisition cost is higher than what the insurance company is willing to reimburse them. Essentially, the insurance companies expect the rheumatologists to pay them to take care of their patients. Because of the substantial and destabilizing financial losses incurred, many practices and free-standing infusion centers have been forced to cease offering these biosimilars. Most rheumatologists will provide patients with appropriate alternatives when available and permitted by the insurer; otherwise, they must refer patients to hospital-based infusion centers. That results in delayed care and increased costs for patients and the system, because hospital-based infusion typically costs more than twice what office-based infusion costs.
Quantifying the Problem
To help quantify the magnitude of this issue, the Coalition of State Rheumatology Organizations (CSRO) recently conducted a survey of its membership. A shocking 97% of respondents reported that their practice had been affected by reimbursement rates for some biosimilars being lower than acquisition costs, with 91% of respondents stating that this issue is more pronounced for certain biosimilars than others. Across the board, respondents most frequently identified Inflectra (infliximab-dyyb) and Avsola (infliximab-axxq) as being especially affected: Over 88% and over 85% of respondents identified these two products, respectively, as being underwater. These results support the ongoing anecdotal reports CSRO continues to receive from rheumatology practices.
However, the survey results indicated that this issue is by no means confined to those two biosimilars. Truxima (rituximab-abbs) — a biosimilar for Rituxan — was frequently mentioned as well. Notably, respondents almost uniformly identified biosimilars in the infliximab and rituximab families, which illustrates that this issue is no longer confined to one or two early-to-market biosimilars but has almost become a hallmark of this particular biosimilars market. Remarkably, one respondent commented that the brand products are now cheaper to acquire than the biosimilars. Furthermore, the survey included respondents from across the country, indicating that this issue is not confined to a particular region.
How Did This Happen?
Biosimilars held promise for increasing availability and decreasing biologic costs for patients but, thus far, no patients have seen their cost go down. It appears that the only biosimilars that have made it to “preferred” status on the formulary are the ones that have made more money for the middlemen in the drug supply chain, particularly those that construct formularies. Now, we have provider-administered biosimilars whose acquisition cost exceeds the reimbursement for these drugs. This disparity was ultimately created by biosimilar manufacturers “over-rebating” their drugs to health insurance companies to gain “fail-first” status on the formulary.
For example, the manufacturer of Inflectra offered substantial rebates to health insurers for preferred formulary placement. These rebates are factored into the sales price of the medication, which then results in a rapidly declining average sales price (ASP) for the biosimilar. Unfortunately, the acquisition cost for the drug does not experience commensurate reductions, resulting in physicians being reimbursed far less for the drug than it costs to acquire. The financial losses for physicians put them underwater as a result of the acquisition costs for the preferred drugs far surpassing the reimbursement from the health insurance company that constructed the formulary.
While various factors affect ASPs and acquisition costs, this particular consequence of formulary placement based on price concessions is a major driver of the underwater situation in which physicians have found themselves with many biosimilars. Not only does that lead to a lower uptake of biosimilars, but it also results in patients being referred to the hospital outpatient infusion sites to receive this care, as freestanding infusion centers cannot treat these patients either. Hospitals incur higher costs because of facility fees and elevated rates, and this makes private rheumatology in-office infusion centers a much lower-cost option. Similarly, home infusion services, while convenient, are marginally more expensive than private practices and, in cases of biologic infusions, it is important to note that physicians’ offices have a greater safety profile than home infusion of biologics. The overall result of these “fail-first underwater drugs” is delayed and more costly care for the patient and the “system,” particularly self-insured employers.
What Is Being Done to Correct This?
Since ASPs are updated quarterly, it is possible that acquisition costs and reimbursements might stabilize over time, making the drugs affordable again to practices. However, that does not appear to be happening in the near future, so that possibility does not offer immediate relief to struggling practices. It doesn’t promise a favorable outlook for future biosimilar entries of provider-administered medications if formularies continue to prefer the highest-rebated medication.
This dynamic between ASP and acquisition cost does not happen on the pharmacy side because the price concessions on specific drug rebates and fees are proprietary. There appears to be no equivalent to a publicly known ASP on the pharmacy side, which has led to myriad pricing definitions and manipulation on the pharmacy benefit side of medications. In any event, the savings from rebates and other manufacturer price concessions on pharmacy drugs do not influence ASPs of medical benefit drugs.
The Inflation Reduction Act provided a temporary increase in the add-on payment for biosimilars from ASP+6% to ASP+8%, but as long as the biosimilar’s ASP is lower than the reference brand’s ASP, that temporary increase does not appear to make up for the large differential between ASP and acquisition cost. It should be noted that any federal attempt to artificially lower the ASP of a provider-administered drug without a pathway assuring that the acquisition cost for the provider is less than the reimbursement is going to result in loss of access for patients to those medications and/or higher hospital site of care costs.
A Few Partial Fixes, But Most Complaints Go Ignored
Considering the higher costs of hospital-based infusion, insurers should be motivated to keep patients within private practices. Perhaps through insurers’ recognition of that fact, some practices have successfully negotiated exceptions for specific patients by discussing this situation with insurers. From the feedback that CSRO has received from rheumatology practices, it appears that most insurers have been ignoring the complaints from physicians. The few who have responded have resulted in only partial fixes, with some of the biosimilars still left underwater.
Ultimate Solution?
This issue is a direct result of the “rebate game,” whereby price concessions from drug manufacturers drive formulary placement. For provider-administered medications, this results in an artificially lowered ASP, not as a consequence of free-market incentives that benefit the patient, but as a result of misaligned incentives created by Safe Harbor–protected “kickbacks,” distorting the free market and paradoxically reducing access to these medications, delaying care, and increasing prices for patients and the healthcare system.
While federal and state governments are not likely to address this particular situation in the biosimilars market, CSRO is highlighting this issue as a prime example of why the current formulary construction system urgently requires federal reform. At this time, the biosimilars most affected are Inflectra and Avsola, but if nothing changes, more and more biosimilars will fall victim to the short-sighted pricing strategy of aggressive rebating to gain formulary position, with physician purchasers and patients left to navigate the aftermath. The existing system, which necessitates drug companies purchasing formulary access from pharmacy benefit managers, has led to delayed and even denied patient access to certain provider-administered drugs. Moreover, it now appears to be hindering the adoption of biosimilars.
To address this, a multifaceted approach is required. It not only involves reevaluating the rebate system and its impact on formulary construction and ASP, but also ensuring that acquisition costs for providers are aligned with reimbursement rates. Insurers must recognize the economic and clinical value of maintaining infusions within private practices and immediately update their policies to ensure that physician in-office infusion is financially feasible for these “fail-first” biosimilars.
Ultimately, the goal should be to create a sustainable model that promotes the use of affordable biosimilars, enhances patient access to affordable care, and supports the financial viability of medical practices. Concerted efforts to reform the current formulary construction system are required to achieve a healthcare environment that is both cost effective and patient centric.
Dr. Feldman is a rheumatologist in private practice with The Rheumatology Group in New Orleans. She is the CSRO’s vice president of advocacy and government affairs and its immediate past president, as well as past chair of the Alliance for Safe Biologic Medicines and a past member of the American College of Rheumatology insurance subcommittee. You can reach her at [email protected].
Editor’s note: This article is adapted from an explanatory statement that Dr. Feldman wrote for the Coalition of State Rheumatology Organizations (CSRO).
According to the Guinness Book of World records, the longest time someone has held their breath underwater voluntarily is 24 minutes and 37.36 seconds. While certainly an amazing feat, UnitedHealthcare, many of the Blues, and other national “payers” are expecting rheumatologists and other specialists to live “underwater” in order to take care of their patients. In other words, these insurance companies are mandating that specialists use certain provider-administered biosimilars whose acquisition cost is higher than what the insurance company is willing to reimburse them. Essentially, the insurance companies expect the rheumatologists to pay them to take care of their patients. Because of the substantial and destabilizing financial losses incurred, many practices and free-standing infusion centers have been forced to cease offering these biosimilars. Most rheumatologists will provide patients with appropriate alternatives when available and permitted by the insurer; otherwise, they must refer patients to hospital-based infusion centers. That results in delayed care and increased costs for patients and the system, because hospital-based infusion typically costs more than twice what office-based infusion costs.
Quantifying the Problem
To help quantify the magnitude of this issue, the Coalition of State Rheumatology Organizations (CSRO) recently conducted a survey of its membership. A shocking 97% of respondents reported that their practice had been affected by reimbursement rates for some biosimilars being lower than acquisition costs, with 91% of respondents stating that this issue is more pronounced for certain biosimilars than others. Across the board, respondents most frequently identified Inflectra (infliximab-dyyb) and Avsola (infliximab-axxq) as being especially affected: Over 88% and over 85% of respondents identified these two products, respectively, as being underwater. These results support the ongoing anecdotal reports CSRO continues to receive from rheumatology practices.
However, the survey results indicated that this issue is by no means confined to those two biosimilars. Truxima (rituximab-abbs) — a biosimilar for Rituxan — was frequently mentioned as well. Notably, respondents almost uniformly identified biosimilars in the infliximab and rituximab families, which illustrates that this issue is no longer confined to one or two early-to-market biosimilars but has almost become a hallmark of this particular biosimilars market. Remarkably, one respondent commented that the brand products are now cheaper to acquire than the biosimilars. Furthermore, the survey included respondents from across the country, indicating that this issue is not confined to a particular region.
How Did This Happen?
Biosimilars held promise for increasing availability and decreasing biologic costs for patients but, thus far, no patients have seen their cost go down. It appears that the only biosimilars that have made it to “preferred” status on the formulary are the ones that have made more money for the middlemen in the drug supply chain, particularly those that construct formularies. Now, we have provider-administered biosimilars whose acquisition cost exceeds the reimbursement for these drugs. This disparity was ultimately created by biosimilar manufacturers “over-rebating” their drugs to health insurance companies to gain “fail-first” status on the formulary.
For example, the manufacturer of Inflectra offered substantial rebates to health insurers for preferred formulary placement. These rebates are factored into the sales price of the medication, which then results in a rapidly declining average sales price (ASP) for the biosimilar. Unfortunately, the acquisition cost for the drug does not experience commensurate reductions, resulting in physicians being reimbursed far less for the drug than it costs to acquire. The financial losses for physicians put them underwater as a result of the acquisition costs for the preferred drugs far surpassing the reimbursement from the health insurance company that constructed the formulary.
While various factors affect ASPs and acquisition costs, this particular consequence of formulary placement based on price concessions is a major driver of the underwater situation in which physicians have found themselves with many biosimilars. Not only does that lead to a lower uptake of biosimilars, but it also results in patients being referred to the hospital outpatient infusion sites to receive this care, as freestanding infusion centers cannot treat these patients either. Hospitals incur higher costs because of facility fees and elevated rates, and this makes private rheumatology in-office infusion centers a much lower-cost option. Similarly, home infusion services, while convenient, are marginally more expensive than private practices and, in cases of biologic infusions, it is important to note that physicians’ offices have a greater safety profile than home infusion of biologics. The overall result of these “fail-first underwater drugs” is delayed and more costly care for the patient and the “system,” particularly self-insured employers.
What Is Being Done to Correct This?
Since ASPs are updated quarterly, it is possible that acquisition costs and reimbursements might stabilize over time, making the drugs affordable again to practices. However, that does not appear to be happening in the near future, so that possibility does not offer immediate relief to struggling practices. It doesn’t promise a favorable outlook for future biosimilar entries of provider-administered medications if formularies continue to prefer the highest-rebated medication.
This dynamic between ASP and acquisition cost does not happen on the pharmacy side because the price concessions on specific drug rebates and fees are proprietary. There appears to be no equivalent to a publicly known ASP on the pharmacy side, which has led to myriad pricing definitions and manipulation on the pharmacy benefit side of medications. In any event, the savings from rebates and other manufacturer price concessions on pharmacy drugs do not influence ASPs of medical benefit drugs.
The Inflation Reduction Act provided a temporary increase in the add-on payment for biosimilars from ASP+6% to ASP+8%, but as long as the biosimilar’s ASP is lower than the reference brand’s ASP, that temporary increase does not appear to make up for the large differential between ASP and acquisition cost. It should be noted that any federal attempt to artificially lower the ASP of a provider-administered drug without a pathway assuring that the acquisition cost for the provider is less than the reimbursement is going to result in loss of access for patients to those medications and/or higher hospital site of care costs.
A Few Partial Fixes, But Most Complaints Go Ignored
Considering the higher costs of hospital-based infusion, insurers should be motivated to keep patients within private practices. Perhaps through insurers’ recognition of that fact, some practices have successfully negotiated exceptions for specific patients by discussing this situation with insurers. From the feedback that CSRO has received from rheumatology practices, it appears that most insurers have been ignoring the complaints from physicians. The few who have responded have resulted in only partial fixes, with some of the biosimilars still left underwater.
Ultimate Solution?
This issue is a direct result of the “rebate game,” whereby price concessions from drug manufacturers drive formulary placement. For provider-administered medications, this results in an artificially lowered ASP, not as a consequence of free-market incentives that benefit the patient, but as a result of misaligned incentives created by Safe Harbor–protected “kickbacks,” distorting the free market and paradoxically reducing access to these medications, delaying care, and increasing prices for patients and the healthcare system.
While federal and state governments are not likely to address this particular situation in the biosimilars market, CSRO is highlighting this issue as a prime example of why the current formulary construction system urgently requires federal reform. At this time, the biosimilars most affected are Inflectra and Avsola, but if nothing changes, more and more biosimilars will fall victim to the short-sighted pricing strategy of aggressive rebating to gain formulary position, with physician purchasers and patients left to navigate the aftermath. The existing system, which necessitates drug companies purchasing formulary access from pharmacy benefit managers, has led to delayed and even denied patient access to certain provider-administered drugs. Moreover, it now appears to be hindering the adoption of biosimilars.
To address this, a multifaceted approach is required. It not only involves reevaluating the rebate system and its impact on formulary construction and ASP, but also ensuring that acquisition costs for providers are aligned with reimbursement rates. Insurers must recognize the economic and clinical value of maintaining infusions within private practices and immediately update their policies to ensure that physician in-office infusion is financially feasible for these “fail-first” biosimilars.
Ultimately, the goal should be to create a sustainable model that promotes the use of affordable biosimilars, enhances patient access to affordable care, and supports the financial viability of medical practices. Concerted efforts to reform the current formulary construction system are required to achieve a healthcare environment that is both cost effective and patient centric.
Dr. Feldman is a rheumatologist in private practice with The Rheumatology Group in New Orleans. She is the CSRO’s vice president of advocacy and government affairs and its immediate past president, as well as past chair of the Alliance for Safe Biologic Medicines and a past member of the American College of Rheumatology insurance subcommittee. You can reach her at [email protected].
Editor’s note: This article is adapted from an explanatory statement that Dr. Feldman wrote for the Coalition of State Rheumatology Organizations (CSRO).
According to the Guinness Book of World records, the longest time someone has held their breath underwater voluntarily is 24 minutes and 37.36 seconds. While certainly an amazing feat, UnitedHealthcare, many of the Blues, and other national “payers” are expecting rheumatologists and other specialists to live “underwater” in order to take care of their patients. In other words, these insurance companies are mandating that specialists use certain provider-administered biosimilars whose acquisition cost is higher than what the insurance company is willing to reimburse them. Essentially, the insurance companies expect the rheumatologists to pay them to take care of their patients. Because of the substantial and destabilizing financial losses incurred, many practices and free-standing infusion centers have been forced to cease offering these biosimilars. Most rheumatologists will provide patients with appropriate alternatives when available and permitted by the insurer; otherwise, they must refer patients to hospital-based infusion centers. That results in delayed care and increased costs for patients and the system, because hospital-based infusion typically costs more than twice what office-based infusion costs.
Quantifying the Problem
To help quantify the magnitude of this issue, the Coalition of State Rheumatology Organizations (CSRO) recently conducted a survey of its membership. A shocking 97% of respondents reported that their practice had been affected by reimbursement rates for some biosimilars being lower than acquisition costs, with 91% of respondents stating that this issue is more pronounced for certain biosimilars than others. Across the board, respondents most frequently identified Inflectra (infliximab-dyyb) and Avsola (infliximab-axxq) as being especially affected: Over 88% and over 85% of respondents identified these two products, respectively, as being underwater. These results support the ongoing anecdotal reports CSRO continues to receive from rheumatology practices.
However, the survey results indicated that this issue is by no means confined to those two biosimilars. Truxima (rituximab-abbs) — a biosimilar for Rituxan — was frequently mentioned as well. Notably, respondents almost uniformly identified biosimilars in the infliximab and rituximab families, which illustrates that this issue is no longer confined to one or two early-to-market biosimilars but has almost become a hallmark of this particular biosimilars market. Remarkably, one respondent commented that the brand products are now cheaper to acquire than the biosimilars. Furthermore, the survey included respondents from across the country, indicating that this issue is not confined to a particular region.
How Did This Happen?
Biosimilars held promise for increasing availability and decreasing biologic costs for patients but, thus far, no patients have seen their cost go down. It appears that the only biosimilars that have made it to “preferred” status on the formulary are the ones that have made more money for the middlemen in the drug supply chain, particularly those that construct formularies. Now, we have provider-administered biosimilars whose acquisition cost exceeds the reimbursement for these drugs. This disparity was ultimately created by biosimilar manufacturers “over-rebating” their drugs to health insurance companies to gain “fail-first” status on the formulary.
For example, the manufacturer of Inflectra offered substantial rebates to health insurers for preferred formulary placement. These rebates are factored into the sales price of the medication, which then results in a rapidly declining average sales price (ASP) for the biosimilar. Unfortunately, the acquisition cost for the drug does not experience commensurate reductions, resulting in physicians being reimbursed far less for the drug than it costs to acquire. The financial losses for physicians put them underwater as a result of the acquisition costs for the preferred drugs far surpassing the reimbursement from the health insurance company that constructed the formulary.
While various factors affect ASPs and acquisition costs, this particular consequence of formulary placement based on price concessions is a major driver of the underwater situation in which physicians have found themselves with many biosimilars. Not only does that lead to a lower uptake of biosimilars, but it also results in patients being referred to the hospital outpatient infusion sites to receive this care, as freestanding infusion centers cannot treat these patients either. Hospitals incur higher costs because of facility fees and elevated rates, and this makes private rheumatology in-office infusion centers a much lower-cost option. Similarly, home infusion services, while convenient, are marginally more expensive than private practices and, in cases of biologic infusions, it is important to note that physicians’ offices have a greater safety profile than home infusion of biologics. The overall result of these “fail-first underwater drugs” is delayed and more costly care for the patient and the “system,” particularly self-insured employers.
What Is Being Done to Correct This?
Since ASPs are updated quarterly, it is possible that acquisition costs and reimbursements might stabilize over time, making the drugs affordable again to practices. However, that does not appear to be happening in the near future, so that possibility does not offer immediate relief to struggling practices. It doesn’t promise a favorable outlook for future biosimilar entries of provider-administered medications if formularies continue to prefer the highest-rebated medication.
This dynamic between ASP and acquisition cost does not happen on the pharmacy side because the price concessions on specific drug rebates and fees are proprietary. There appears to be no equivalent to a publicly known ASP on the pharmacy side, which has led to myriad pricing definitions and manipulation on the pharmacy benefit side of medications. In any event, the savings from rebates and other manufacturer price concessions on pharmacy drugs do not influence ASPs of medical benefit drugs.
The Inflation Reduction Act provided a temporary increase in the add-on payment for biosimilars from ASP+6% to ASP+8%, but as long as the biosimilar’s ASP is lower than the reference brand’s ASP, that temporary increase does not appear to make up for the large differential between ASP and acquisition cost. It should be noted that any federal attempt to artificially lower the ASP of a provider-administered drug without a pathway assuring that the acquisition cost for the provider is less than the reimbursement is going to result in loss of access for patients to those medications and/or higher hospital site of care costs.
A Few Partial Fixes, But Most Complaints Go Ignored
Considering the higher costs of hospital-based infusion, insurers should be motivated to keep patients within private practices. Perhaps through insurers’ recognition of that fact, some practices have successfully negotiated exceptions for specific patients by discussing this situation with insurers. From the feedback that CSRO has received from rheumatology practices, it appears that most insurers have been ignoring the complaints from physicians. The few who have responded have resulted in only partial fixes, with some of the biosimilars still left underwater.
Ultimate Solution?
This issue is a direct result of the “rebate game,” whereby price concessions from drug manufacturers drive formulary placement. For provider-administered medications, this results in an artificially lowered ASP, not as a consequence of free-market incentives that benefit the patient, but as a result of misaligned incentives created by Safe Harbor–protected “kickbacks,” distorting the free market and paradoxically reducing access to these medications, delaying care, and increasing prices for patients and the healthcare system.
While federal and state governments are not likely to address this particular situation in the biosimilars market, CSRO is highlighting this issue as a prime example of why the current formulary construction system urgently requires federal reform. At this time, the biosimilars most affected are Inflectra and Avsola, but if nothing changes, more and more biosimilars will fall victim to the short-sighted pricing strategy of aggressive rebating to gain formulary position, with physician purchasers and patients left to navigate the aftermath. The existing system, which necessitates drug companies purchasing formulary access from pharmacy benefit managers, has led to delayed and even denied patient access to certain provider-administered drugs. Moreover, it now appears to be hindering the adoption of biosimilars.
To address this, a multifaceted approach is required. It not only involves reevaluating the rebate system and its impact on formulary construction and ASP, but also ensuring that acquisition costs for providers are aligned with reimbursement rates. Insurers must recognize the economic and clinical value of maintaining infusions within private practices and immediately update their policies to ensure that physician in-office infusion is financially feasible for these “fail-first” biosimilars.
Ultimately, the goal should be to create a sustainable model that promotes the use of affordable biosimilars, enhances patient access to affordable care, and supports the financial viability of medical practices. Concerted efforts to reform the current formulary construction system are required to achieve a healthcare environment that is both cost effective and patient centric.
Dr. Feldman is a rheumatologist in private practice with The Rheumatology Group in New Orleans. She is the CSRO’s vice president of advocacy and government affairs and its immediate past president, as well as past chair of the Alliance for Safe Biologic Medicines and a past member of the American College of Rheumatology insurance subcommittee. You can reach her at [email protected].
Pediatric Dermatologists Beat ChatGPT on Board Questions
In an experiment that pitted the wits of results from a small single-center study showed.
“We were relieved to find that the pediatric dermatologists in our study performed better than ChatGPT on both multiple choice and case-based questions; however, the latest iteration of ChatGPT (4.0) was very close,” one of the study’s first authors Charles Huang, a fourth-year medical student at Thomas Jefferson University, Philadelphia, said in an interview. “Something else that was interesting in our data was that the pediatric dermatologists performed much better than ChatGPT on questions related to procedural dermatology/surgical techniques, perhaps indicating that knowledge/reasoning gained through practical experience isn’t easily replicated in AI tools such as ChatGPT.”
For the study, which was published on May 9 in Pediatric Dermatology, Mr. Huang, and co-first author Esther Zhang, BS, a medical student at the University of Pennsylvania, Philadelphia, and coauthors from the Department of Dermatology, Children’s Hospital of Philadelphia, asked five pediatric dermatologists to answer 24 text-based questions including 16 single-answer, multiple-choice questions and two multiple answer questions drawn from the American Board of Dermatology 2021 Certification Sample Test and six free-response case-based questions drawn from the “Photoquiz” section of Pediatric Dermatology between July 2022 and July 2023. The researchers then processed the same set of questions through ChatGPT versions 3.5 and 4.0 and used statistical analysis to compare responses between the pediatric dermatologists and ChatGPT. A 5-point scale adapted from current AI tools was used to score replies to case-based questions.
On average, study participants had 5.6 years of clinical experience. Pediatric dermatologists performed significantly better than ChatGPT version 3.5 on multiple-choice and multiple answer questions (91.4% vs 76.2%, respectively; P = .021) but not significantly better than ChatGPT version 4.0 (90.5%; P = .44). As for replies to case-based questions, the average performance based on the 5-point scale was 3.81 for pediatric dermatologists and 3.53 for ChatGPT overall. The mean scores were significantly greater for pediatric dermatologists than for ChatGPT version 3.5 (P = .039) but not ChatGPT version 4.0 (P = .43).
The researchers acknowledged certain limitations of the analysis, including the evolving nature of AI tools, which may affect the reproducibility of results with subsequent model updates. And, while participating pediatric dermatologists said they were unfamiliar with the questions and cases used in the study, “there is potential for prior exposure through other dermatology board examination review processes,” they wrote.
“AI tools such as ChatGPT and similar large language models can be a valuable tool in your clinical practice, but be aware of potential pitfalls such as patient privacy, medical inaccuracies, [and] intrinsic biases in the tools,” Mr. Huang told this news organization. “As these technologies continue to advance, it is essential for all of us as medical clinicians to gain familiarity and stay abreast of new developments, just as we adapted to electronic health records and the use of the Internet.”
Maria Buethe, MD, PhD, a pediatric dermatology fellow at Rady Children’s Hospital–San Diego, who was asked to comment on the study, said she found it “interesting” that ChatGPT’s version 4.0 started to produce comparable results to clinician responses in some of the tested scenarios.
“The authors propose a set of best practices for pediatric dermatology clinicians using ChatGPT and other AI tools,” said Dr. Buethe, who was senior author of a recent literature review on AI and its application to pediatric dermatology. It was published in SKIN The Journal of Cutaneous Medicine. “One interesting recommended use for AI tools is to utilize it to generate differential diagnosis, which can broaden the list of pathologies previously considered.”
Asked to comment on the study, Erum Ilyas, MD, who practices dermatology in King of Prussia, Pennsylvania, and is a member of the Society for Pediatric Dermatology, said she was not surprised that ChatGPT “can perform fairly well on multiple-choice questions as we find available in testing circumstances,” as presented in the study. “Just as board questions only support testing a base of medical knowledge and facts for clinicians to master, they do not necessarily provide real-life circumstances that apply to caring for patients, which is inherently nuanced.”
In addition, the study “highlights that ChatGPT can be an aid to support thinking through differentials based on data entered by a clinician who understands how to phrase queries, especially if provided with enough data while respecting patient privacy, in the context of fact checking responses,” Dr. Ilyas said. “This underscores the fact that AI tools can be helpful to clinicians in assimilating various data points entered. However, ultimately, the tool is only able to support an output based on the information it has access to.” She added, “ChatGPT cannot be relied on to provide a single diagnosis with the clinician still responsible for making a final diagnosis. The tool is not definitive and cannot assimilate data that is not entered correctly.”
The study was not funded, and the study authors reported having no disclosures. Dr. Buethe and Dr. Ilyas, who were not involved with the study, had no disclosures.
A version of this article appeared on Medscape.com .
In an experiment that pitted the wits of results from a small single-center study showed.
“We were relieved to find that the pediatric dermatologists in our study performed better than ChatGPT on both multiple choice and case-based questions; however, the latest iteration of ChatGPT (4.0) was very close,” one of the study’s first authors Charles Huang, a fourth-year medical student at Thomas Jefferson University, Philadelphia, said in an interview. “Something else that was interesting in our data was that the pediatric dermatologists performed much better than ChatGPT on questions related to procedural dermatology/surgical techniques, perhaps indicating that knowledge/reasoning gained through practical experience isn’t easily replicated in AI tools such as ChatGPT.”
For the study, which was published on May 9 in Pediatric Dermatology, Mr. Huang, and co-first author Esther Zhang, BS, a medical student at the University of Pennsylvania, Philadelphia, and coauthors from the Department of Dermatology, Children’s Hospital of Philadelphia, asked five pediatric dermatologists to answer 24 text-based questions including 16 single-answer, multiple-choice questions and two multiple answer questions drawn from the American Board of Dermatology 2021 Certification Sample Test and six free-response case-based questions drawn from the “Photoquiz” section of Pediatric Dermatology between July 2022 and July 2023. The researchers then processed the same set of questions through ChatGPT versions 3.5 and 4.0 and used statistical analysis to compare responses between the pediatric dermatologists and ChatGPT. A 5-point scale adapted from current AI tools was used to score replies to case-based questions.
On average, study participants had 5.6 years of clinical experience. Pediatric dermatologists performed significantly better than ChatGPT version 3.5 on multiple-choice and multiple answer questions (91.4% vs 76.2%, respectively; P = .021) but not significantly better than ChatGPT version 4.0 (90.5%; P = .44). As for replies to case-based questions, the average performance based on the 5-point scale was 3.81 for pediatric dermatologists and 3.53 for ChatGPT overall. The mean scores were significantly greater for pediatric dermatologists than for ChatGPT version 3.5 (P = .039) but not ChatGPT version 4.0 (P = .43).
The researchers acknowledged certain limitations of the analysis, including the evolving nature of AI tools, which may affect the reproducibility of results with subsequent model updates. And, while participating pediatric dermatologists said they were unfamiliar with the questions and cases used in the study, “there is potential for prior exposure through other dermatology board examination review processes,” they wrote.
“AI tools such as ChatGPT and similar large language models can be a valuable tool in your clinical practice, but be aware of potential pitfalls such as patient privacy, medical inaccuracies, [and] intrinsic biases in the tools,” Mr. Huang told this news organization. “As these technologies continue to advance, it is essential for all of us as medical clinicians to gain familiarity and stay abreast of new developments, just as we adapted to electronic health records and the use of the Internet.”
Maria Buethe, MD, PhD, a pediatric dermatology fellow at Rady Children’s Hospital–San Diego, who was asked to comment on the study, said she found it “interesting” that ChatGPT’s version 4.0 started to produce comparable results to clinician responses in some of the tested scenarios.
“The authors propose a set of best practices for pediatric dermatology clinicians using ChatGPT and other AI tools,” said Dr. Buethe, who was senior author of a recent literature review on AI and its application to pediatric dermatology. It was published in SKIN The Journal of Cutaneous Medicine. “One interesting recommended use for AI tools is to utilize it to generate differential diagnosis, which can broaden the list of pathologies previously considered.”
Asked to comment on the study, Erum Ilyas, MD, who practices dermatology in King of Prussia, Pennsylvania, and is a member of the Society for Pediatric Dermatology, said she was not surprised that ChatGPT “can perform fairly well on multiple-choice questions as we find available in testing circumstances,” as presented in the study. “Just as board questions only support testing a base of medical knowledge and facts for clinicians to master, they do not necessarily provide real-life circumstances that apply to caring for patients, which is inherently nuanced.”
In addition, the study “highlights that ChatGPT can be an aid to support thinking through differentials based on data entered by a clinician who understands how to phrase queries, especially if provided with enough data while respecting patient privacy, in the context of fact checking responses,” Dr. Ilyas said. “This underscores the fact that AI tools can be helpful to clinicians in assimilating various data points entered. However, ultimately, the tool is only able to support an output based on the information it has access to.” She added, “ChatGPT cannot be relied on to provide a single diagnosis with the clinician still responsible for making a final diagnosis. The tool is not definitive and cannot assimilate data that is not entered correctly.”
The study was not funded, and the study authors reported having no disclosures. Dr. Buethe and Dr. Ilyas, who were not involved with the study, had no disclosures.
A version of this article appeared on Medscape.com .
In an experiment that pitted the wits of results from a small single-center study showed.
“We were relieved to find that the pediatric dermatologists in our study performed better than ChatGPT on both multiple choice and case-based questions; however, the latest iteration of ChatGPT (4.0) was very close,” one of the study’s first authors Charles Huang, a fourth-year medical student at Thomas Jefferson University, Philadelphia, said in an interview. “Something else that was interesting in our data was that the pediatric dermatologists performed much better than ChatGPT on questions related to procedural dermatology/surgical techniques, perhaps indicating that knowledge/reasoning gained through practical experience isn’t easily replicated in AI tools such as ChatGPT.”
For the study, which was published on May 9 in Pediatric Dermatology, Mr. Huang, and co-first author Esther Zhang, BS, a medical student at the University of Pennsylvania, Philadelphia, and coauthors from the Department of Dermatology, Children’s Hospital of Philadelphia, asked five pediatric dermatologists to answer 24 text-based questions including 16 single-answer, multiple-choice questions and two multiple answer questions drawn from the American Board of Dermatology 2021 Certification Sample Test and six free-response case-based questions drawn from the “Photoquiz” section of Pediatric Dermatology between July 2022 and July 2023. The researchers then processed the same set of questions through ChatGPT versions 3.5 and 4.0 and used statistical analysis to compare responses between the pediatric dermatologists and ChatGPT. A 5-point scale adapted from current AI tools was used to score replies to case-based questions.
On average, study participants had 5.6 years of clinical experience. Pediatric dermatologists performed significantly better than ChatGPT version 3.5 on multiple-choice and multiple answer questions (91.4% vs 76.2%, respectively; P = .021) but not significantly better than ChatGPT version 4.0 (90.5%; P = .44). As for replies to case-based questions, the average performance based on the 5-point scale was 3.81 for pediatric dermatologists and 3.53 for ChatGPT overall. The mean scores were significantly greater for pediatric dermatologists than for ChatGPT version 3.5 (P = .039) but not ChatGPT version 4.0 (P = .43).
The researchers acknowledged certain limitations of the analysis, including the evolving nature of AI tools, which may affect the reproducibility of results with subsequent model updates. And, while participating pediatric dermatologists said they were unfamiliar with the questions and cases used in the study, “there is potential for prior exposure through other dermatology board examination review processes,” they wrote.
“AI tools such as ChatGPT and similar large language models can be a valuable tool in your clinical practice, but be aware of potential pitfalls such as patient privacy, medical inaccuracies, [and] intrinsic biases in the tools,” Mr. Huang told this news organization. “As these technologies continue to advance, it is essential for all of us as medical clinicians to gain familiarity and stay abreast of new developments, just as we adapted to electronic health records and the use of the Internet.”
Maria Buethe, MD, PhD, a pediatric dermatology fellow at Rady Children’s Hospital–San Diego, who was asked to comment on the study, said she found it “interesting” that ChatGPT’s version 4.0 started to produce comparable results to clinician responses in some of the tested scenarios.
“The authors propose a set of best practices for pediatric dermatology clinicians using ChatGPT and other AI tools,” said Dr. Buethe, who was senior author of a recent literature review on AI and its application to pediatric dermatology. It was published in SKIN The Journal of Cutaneous Medicine. “One interesting recommended use for AI tools is to utilize it to generate differential diagnosis, which can broaden the list of pathologies previously considered.”
Asked to comment on the study, Erum Ilyas, MD, who practices dermatology in King of Prussia, Pennsylvania, and is a member of the Society for Pediatric Dermatology, said she was not surprised that ChatGPT “can perform fairly well on multiple-choice questions as we find available in testing circumstances,” as presented in the study. “Just as board questions only support testing a base of medical knowledge and facts for clinicians to master, they do not necessarily provide real-life circumstances that apply to caring for patients, which is inherently nuanced.”
In addition, the study “highlights that ChatGPT can be an aid to support thinking through differentials based on data entered by a clinician who understands how to phrase queries, especially if provided with enough data while respecting patient privacy, in the context of fact checking responses,” Dr. Ilyas said. “This underscores the fact that AI tools can be helpful to clinicians in assimilating various data points entered. However, ultimately, the tool is only able to support an output based on the information it has access to.” She added, “ChatGPT cannot be relied on to provide a single diagnosis with the clinician still responsible for making a final diagnosis. The tool is not definitive and cannot assimilate data that is not entered correctly.”
The study was not funded, and the study authors reported having no disclosures. Dr. Buethe and Dr. Ilyas, who were not involved with the study, had no disclosures.
A version of this article appeared on Medscape.com .
Global Analysis Identifies Drugs Associated With SJS-TEN in Children
TOPLINE:
METHODOLOGY:
- SJS and TEN are rare, life-threatening mucocutaneous reactions mainly associated with medications, but large pharmacovigilance studies of drugs associated with SJS-TEN in the pediatric population are still lacking.
- Using the WHO’s pharmacovigilance database (VigiBase) containing individual case safety reports from January 1967 to July 2022, researchers identified 7342 adverse drug reaction reports of SJS-TEN in children (younger than 18 years; median age, 9 years) in all six continents. Median onset was 5 days, and 3.2% were fatal.
- They analyzed drugs reported as suspected treatments, and for each molecule, they performed a case–non-case study to assess a potential pharmacovigilance signal by computing the information component (IC).
- A positive IC value suggested more frequent reporting of a specific drug-adverse reaction pair. A positive IC025, a traditional threshold for statistical signal detection, is suggestive of a potential pharmacovigilance signal.
TAKEAWAY:
- Overall, 165 drugs were associated with a diagnosis of SJS-TEN; antiepileptic and anti-infectious drugs were the most common drug classes represented.
- The five most frequently reported drugs were carbamazepine (11.7%), lamotrigine (10.6%), sulfamethoxazole-trimethoprim (9%), acetaminophen (8.4%), and phenytoin (6.6%). The five drugs with the highest IC025 were lamotrigine, carbamazepine, phenobarbital, phenytoin, and nimesulide.
- All antiepileptics, many antibiotic families, dapsone, antiretroviral drugs, some antifungal drugs, and nonsteroidal anti-inflammatory drugs were identified in reports, with penicillins the most frequently reported antibiotic family and sulfonamides having the strongest pharmacovigilance signal.
- Vaccines were not associated with significant signals.
IN PRACTICE:
The study provides an update on “the spectrum of drugs potentially associated with SJS-TEN in the pediatric population,” the authors concluded, and “underlines the importance of reporting to pharmacovigilance the suspicion of this severe side effect of drugs with the most precise and detailed clinical description possible.”
SOURCE:
The study, led by Pauline Bataille, MD, of the Department of Pediatric Dermatology, Hôpital Necker-Enfants Malades, Paris City University, France, was published online in the Journal of the European Academy of Dermatology and Venereology.
LIMITATIONS:
Limitations include the possibility that some cases could have had an infectious or idiopathic cause not related to a drug and the lack of detailed clinical data in the database.
DISCLOSURES:
This study did not receive any funding. The authors declared no conflict of interest.
A version of this article first appeared on Medscape.com.
TOPLINE:
METHODOLOGY:
- SJS and TEN are rare, life-threatening mucocutaneous reactions mainly associated with medications, but large pharmacovigilance studies of drugs associated with SJS-TEN in the pediatric population are still lacking.
- Using the WHO’s pharmacovigilance database (VigiBase) containing individual case safety reports from January 1967 to July 2022, researchers identified 7342 adverse drug reaction reports of SJS-TEN in children (younger than 18 years; median age, 9 years) in all six continents. Median onset was 5 days, and 3.2% were fatal.
- They analyzed drugs reported as suspected treatments, and for each molecule, they performed a case–non-case study to assess a potential pharmacovigilance signal by computing the information component (IC).
- A positive IC value suggested more frequent reporting of a specific drug-adverse reaction pair. A positive IC025, a traditional threshold for statistical signal detection, is suggestive of a potential pharmacovigilance signal.
TAKEAWAY:
- Overall, 165 drugs were associated with a diagnosis of SJS-TEN; antiepileptic and anti-infectious drugs were the most common drug classes represented.
- The five most frequently reported drugs were carbamazepine (11.7%), lamotrigine (10.6%), sulfamethoxazole-trimethoprim (9%), acetaminophen (8.4%), and phenytoin (6.6%). The five drugs with the highest IC025 were lamotrigine, carbamazepine, phenobarbital, phenytoin, and nimesulide.
- All antiepileptics, many antibiotic families, dapsone, antiretroviral drugs, some antifungal drugs, and nonsteroidal anti-inflammatory drugs were identified in reports, with penicillins the most frequently reported antibiotic family and sulfonamides having the strongest pharmacovigilance signal.
- Vaccines were not associated with significant signals.
IN PRACTICE:
The study provides an update on “the spectrum of drugs potentially associated with SJS-TEN in the pediatric population,” the authors concluded, and “underlines the importance of reporting to pharmacovigilance the suspicion of this severe side effect of drugs with the most precise and detailed clinical description possible.”
SOURCE:
The study, led by Pauline Bataille, MD, of the Department of Pediatric Dermatology, Hôpital Necker-Enfants Malades, Paris City University, France, was published online in the Journal of the European Academy of Dermatology and Venereology.
LIMITATIONS:
Limitations include the possibility that some cases could have had an infectious or idiopathic cause not related to a drug and the lack of detailed clinical data in the database.
DISCLOSURES:
This study did not receive any funding. The authors declared no conflict of interest.
A version of this article first appeared on Medscape.com.
TOPLINE:
METHODOLOGY:
- SJS and TEN are rare, life-threatening mucocutaneous reactions mainly associated with medications, but large pharmacovigilance studies of drugs associated with SJS-TEN in the pediatric population are still lacking.
- Using the WHO’s pharmacovigilance database (VigiBase) containing individual case safety reports from January 1967 to July 2022, researchers identified 7342 adverse drug reaction reports of SJS-TEN in children (younger than 18 years; median age, 9 years) in all six continents. Median onset was 5 days, and 3.2% were fatal.
- They analyzed drugs reported as suspected treatments, and for each molecule, they performed a case–non-case study to assess a potential pharmacovigilance signal by computing the information component (IC).
- A positive IC value suggested more frequent reporting of a specific drug-adverse reaction pair. A positive IC025, a traditional threshold for statistical signal detection, is suggestive of a potential pharmacovigilance signal.
TAKEAWAY:
- Overall, 165 drugs were associated with a diagnosis of SJS-TEN; antiepileptic and anti-infectious drugs were the most common drug classes represented.
- The five most frequently reported drugs were carbamazepine (11.7%), lamotrigine (10.6%), sulfamethoxazole-trimethoprim (9%), acetaminophen (8.4%), and phenytoin (6.6%). The five drugs with the highest IC025 were lamotrigine, carbamazepine, phenobarbital, phenytoin, and nimesulide.
- All antiepileptics, many antibiotic families, dapsone, antiretroviral drugs, some antifungal drugs, and nonsteroidal anti-inflammatory drugs were identified in reports, with penicillins the most frequently reported antibiotic family and sulfonamides having the strongest pharmacovigilance signal.
- Vaccines were not associated with significant signals.
IN PRACTICE:
The study provides an update on “the spectrum of drugs potentially associated with SJS-TEN in the pediatric population,” the authors concluded, and “underlines the importance of reporting to pharmacovigilance the suspicion of this severe side effect of drugs with the most precise and detailed clinical description possible.”
SOURCE:
The study, led by Pauline Bataille, MD, of the Department of Pediatric Dermatology, Hôpital Necker-Enfants Malades, Paris City University, France, was published online in the Journal of the European Academy of Dermatology and Venereology.
LIMITATIONS:
Limitations include the possibility that some cases could have had an infectious or idiopathic cause not related to a drug and the lack of detailed clinical data in the database.
DISCLOSURES:
This study did not receive any funding. The authors declared no conflict of interest.
A version of this article first appeared on Medscape.com.