Closing the Evidence Gap for Drugs in Children — Measures to Strengthen the Pediatric Research Equity Act

The Pediatric Research Equity Act (PREA), the primary US law requiring that new drugs be tested in children, has been undermined by persistent delays in pediatric trial initiation, weak enforcement mechanisms, and statutory gaps that exempt roughly 20% of new drugs and the majority of orphan-designated drugs from pediatric study requirements. The authors recommend that Congress strengthen PREA by requiring pediatric trials to be initiated before adult drug approval, improving enforcement of study deadlines, and expanding the FDA’s authority to require pediatric studies beyond proposed adult indications and for orphan-designated products.