Researchers suggest FDA needs authority to require pediatric studies for orphan drugs

The US Food and Drug Administration (FDA) may need greater authority to require pediatric clinical studies for drugs, including orphan drugs, according to a recent research letter published in JAMA Pediatrics.

While the Pediatric Research Equity Act (PREA) grants FDA the authority to require drugmakers to conduct pediatric studies for certain drugs that are approved for adults, most orphan indications are exempt from this requirement, which the authors of the letter say restricts public understanding of how these treatments affect children.

The authors suggest that FDA may need additional authority to require clinical studies on these drugs in pediatric populations, citing a report by FDA that calls on Congress to consider removing the orphan exemption under PREA, as well as a recent but stalled legislative effort to do so.

“Drug treatment for rare diseases is particularly relevant to pediatric populations, as many of these diseases manifest in childhood. However, except for certain oncology products, drugs designated by the FDA to treat orphan conditions are exempt from PREA study requirements,” wrote Apoorva Kakkilaya of the John Sealy of the School of Medicine at the University of Texas in Galveston, and colleagues, in the letter.

The researchers examined drugs approved by the FDA over a 13-year period, from January 2011 to December 2023. They found that orphan indications were more likely to receive approval for pediatric use than non-orphan indications, even though the studies for orphan drugs were exempt from PREA requirements.

During this period, 78 pediatric orphan indications were approved without the support of a pediatric study, while 81 pediatric non-orphan indications were supported by a pediatric study program.

“While most orphan indications received pediatric approval without pediatric-specific regulatory programs, three quarters of nonorphan indications were supported by one of the programs,” the authors wrote.

In addition, the study found that pediatric orphan drug indications were more likely to include approvals for younger children, with 49 orphan indications approved for those younger than 1 year, compared to 20 non-orphan indications.

FDA has reported that as many as 36% of drugs intended for rare diseases affecting children are approved without comprehensive pediatric information. The authors of the study suggest that granting authorities enhanced powers to mandate pediatric studies for these drugs could be beneficial.

JAMA Letter

Researchers suggest FDA needs authority to require pediatric studies for orphan drugs

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