Skip Navigation Links-New-FDA-Study-Shows-Serious-Research-Gaps-for-Medications-Used-to-Treat-Children-with-Rare-Diseases-

aaa print

New FDA Study Shows Serious Research Gaps for Medications Used to Treat Children with Rare Diseases

8/21/2019 American Academy of Pediatrics, Elizabeth Glaser Pediatric AIDS Foundation urge FDA to act

Washington, DC—The American Academy of Pediatrics (AAP) and the Elizabeth Glaser Pediatric AIDS Foundation (EGPAF) urge the Food and Drug Administration (FDA) to act to ensure that medications used to treat rare childhood diseases are adequately studied for use in children, following the release of a new FDA study showing gaps in pediatric research for such medications.

The study, required as part of the last reauthorization of the Pediatric Research Equity Act (PREA), found that so-called "orphan drugs"—those used to treat diseases effecting less than 200,000 people—are often not studied in children, even though they treat diseases that affect children.

Specifically, the study examined all FDA-approved orphan drugs that treat rare conditions that occur in children as well as adults. Of those, 81 different indications had no pediatric labeling at all, while 46 had incomplete labeling for use in children. When a drug is labeled for children, it means that it has been appropriately studied in the pediatric population and important information on effectiveness, safety, and dosing are added to the drug's label for the benefit of providers and patients.

"Since most rare diseases are genetic, pediatricians are alarmed by the new FDA study finding that one-quarter of all medications indicated for rare diseases that occur in children have no pediatric labeling at all," said AAP President Kyle Yasuda, MD, FAAP. "Children are not just small adults; their unique developmental and physiological needs affect how medications work for them. As a result, a child may need a different dose of a certain medication than an adult, may require new safety precautions, or may benefit from a different use of the drug altogether. FDA must act to remedy this inequity so that more children can benefit from medications that are safe, effective, and tested specifically for them."

PREA requires drug companies to study medications in children, but most orphan drugs are exempted from complying with this requirement by law. The law, however, gives FDA authority to modify this exemption through rulemaking, which AAP and EGPAF urge the agency to do.

"As an organization whose mission is to end AIDS in children, EGPAF is acutely aware of the need to ensure drugs are studied specifically for safety and efficacy in children. Diseases like tuberculosis and malaria are considered rare in the United States but impact millions of children around the world each year," said EGPAF President and CEO Chip Lyons. "The FDA must take action to ensure drugs to prevent, treat, and cure diseases that affect children, regardless of prevalence, are appropriately studied to allow children equitable access to lifesaving medicines."

The proportion of new drugs approved by FDA that are orphan drugs keeps increasing, meaning that a greater proportion of new drugs are exempt from pediatric study requirements. Last year, 58 percent of new drugs approved by FDA's Center for Drug Evaluation and Research were orphan drugs. The AAP and EGPAF urge FDA to act swiftly to remove the orphan drug exemption from PREA's pediatric research requirement.

# # #

The American Academy of Pediatrics is an organization of 67,000 primary care pediatricians, pediatric medical subspecialists and pediatric surgical specialists dedicated to the health, safety and well-being of infants, children, adolescents and young adults. For more information, visit  and follow us on Twitter @AmerAcadPeds.

The Elizabeth Glaser Pediatric AIDS Foundation (EGPAF) is the global leader in the fight against pediatric HIV/AIDS and has reached nearly 30 million pregnant women with services to prevent transmission of HIV to their babies. Founded in 1988, EGPAF today supports activities in 19 countries and over 5,000 sites to implement prevention, care, and treatment services; to further advance innovative research; and to execute global advocacy activities that bring dramatic change to the lives of millions of women, children, and families worldwide. For more information, visit