A recent analysis published in JAMA highlights significant limitations in pediatric drug testing despite the Pediatric Research Equity Act (PREA) of 2003. The study reveals that a large proportion of drugs approved between 2015 and 2021 were either exempt from pediatric trials or faced substantial delays in post-approval testing, raising concerns about the availability of safety and efficacy data for children.
PREA's Limited Scope
PREA was enacted to mandate pediatric trials for new drugs, aiming to bridge the gap in pediatric drug development. However, the law includes exemptions for drugs targeting rare diseases or conditions not affecting children. Researchers, led by Ian T.T. Liu, MD, JD, MPH, MS, from Brigham and Women’s Hospital, examined post-approval testing requirements for 323 drugs approved by the FDA between 2015 and 2021.
The findings indicate that 73% of the drugs were approved for adults only. A significant 38% of the drugs had rare disease designations, exempting them from pediatric testing. An additional 22% had testing requirements waived due to impracticality. Only 10% of the drugs completed pediatric testing before approval, while 30% were required to conduct post-approval pediatric testing.
Delays in Post-Approval Testing
Of the drugs requiring post-approval pediatric studies, a concerning number faced delays. As of the study's follow-up, only 28% of pediatric trials were complete, 53% were ongoing, and 17% were terminated before completion. Furthermore, 41% of ongoing pediatric trials had exceeded their initially projected completion date, with delays averaging a median of 3 years (IQR, 1.69-3.67).
Impact of Rare Disease Designations
The study also points to the increasing prevalence of rare disease designations as a factor limiting PREA's impact. Between 1999 and 2003, 15% of drugs had rare disease designations. This proportion rose to 23% between 2003 and 2012, and nearly half (46%) of drugs approved between 2015 and 2021 had this designation.
Potential Legislative Changes
To address these limitations, legislators introduced the Innovation in Pediatric Drugs Act in 2024, which seeks to eliminate the rare disease exemption for mandatory pediatric testing under PREA. According to the authors, “This study suggests that elimination of the rare disease exemption may increase pediatric testing requirements for new drugs, although additional reforms may be necessary.” Such reforms could ensure more timely and comprehensive data on drug safety and efficacy in pediatric populations.
