Issues in Pediatric Accelerated Approvals Highlighted

International researchers say their review of 26 drugs for 35 pediatric indications given accelerated approval by FDA between 2000 and 2020 suggests there are persistent challenges in generating timely, high-quality pediatric evidence under the accelerated approval pathway and emphasizes the need for innovative data-driven strategies to strengthen confirmatory research. Writing in an open-source JAMA article, the researchers say that of the 35 pediatric indications, seven were pediatric only, six were pediatric extensions, and 22 were combined pediatric and adult indications.

“Approvals frequently relied on surrogate endpoints and extrapolation with pharmacokinetic bridging studies, with limited pediatric enrollment,” the study says. “Although confirmatory studies typically verified efficacy within four years, pediatric-only indications experienced persistent delays, underscoring ongoing challenges in timely pediatric evidence generation.”

The study concludes that the findings “underscored the importance of appropriately addressing pediatric-specific uncertainties through appropriate evidence-generation strategies, including optimizing extrapolation when scientifically justified, model-informed development and innovative clinical data–driven designs to ensure timely confirmation of clinical benefit for children.”