Pediatric Law Gains, Challenges
In its latest five-year report, covering 7/1/2020 to 6/30/2025, FDA says the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) have “fundamentally transformed the landscape of pediatric drug development, delivering substantial benefits to children and families across the United States and globally.” The report notes that since the enactment of the two laws, there have been over 1,364 pediatric labeling changes, providing evidence-based prescribing information for conditions ranging from common childhood ailments to rare diseases and pediatric cancers, with 429 of the changes occurring in the reporting period.
It says that the average time between adult approval and pediatric labeling decreased from about six to five years for the current reporting period, “suggesting some progress toward more timely pediatric drug development.” But it also says that improvement must be seen in the context of broader completion data showing that while 90% of drugs are expected to complete pediatric studies by 10 years post-approval, only 53% had any labeling changes reflecting PREA-mandated study data at that time point.
FDA acknowledges that despite overall progress in pediatric labeling, significant gaps remain in neonatal therapeutics development, with only 12% of the 1,364 pediatric labeling changes including neonatal information, and some 40% of drugs with new neonatal indications relying on extrapolated data rather than clinical studies conducted in neonates.
“The achievements documented in this report underscore that BPCA and PREA have been highly successful in increasing the availability of approved drugs and biological products for children,” the report concludes. “However, addressing persistent challenges in rare disease and neonatal therapeutics development, improving compliance with PREA requirements, and enhancing international alignment will require continued collaboration among all stakeholders. As we look toward the future, the foundation established by BPCA and PREA provides a strong platform for continued innovation in pediatric therapeutics, with the ultimate goal of ensuring safe, effective, and appropriately studied medications for all children who need them.”
The agency says it is examining new enforcement authority for certain PREA violations occurring on or after 8/2.