Post-Approval Pediatric Use of Drugs Granted Waivers from Pediatric Testing

The Pediatric Research Equity Act requires new drugs to be tested in children before approval but allows waivers, often on the grounds that pediatric studies are “impossible or highly impracticable.” Among 291 drugs granted complete waivers from 2007 to 2022, 51% nonetheless had observable pediatric use within 3 years of approval. This off-label use suggests some waived drugs had sufficient uptake to support pediatric trials, and that routine clinical-use data should be monitored to update labeling and inform safe prescribing.