Pediatric Drug Policies Supporting Safe And Effective Use Of Therapeutics In Children: A Systematic Analysis

Several policies have been implemented in the US to promote the evidence-based use of therapeutics in pediatric populations. Under the Best Pharmaceuticals for Children Act of 2002 and the Pediatric Research Equity Act of 2003, the Food and Drug Administration (FDA) can request pediatric studies for new drug and biologic indications. The acts have been credited with generating pediatric information for hundreds of drugs. However, concerns have been raised that delays and noncompliance with study requests contribute to high rates of off-label and potentially unsafe or ineffective medication use in children. We used publicly available FDA documents to analyze all indications for new drugs and biologics approved by the FDA from 2002 through 2018. During this time the FDA issued 389 pediatric study requests (141 under the Best Pharmaceuticals for Children Act and 248 under the Pediatric Research Equity Act) for 274 new drugs and biologics, representing 320 indications. We found that as of December 31, 2018, fewer than a third of these study requests had been completed. Overall, 64 percent of new drug and biologic indications deemed relevant to pediatric patients lacked pediatric prescribing information at five years after FDA approval. Enforcement of pediatric drug study policies should be strengthened to reduce non-evidence-based medication use in pediatric patients.