Pediatric Drug Development: Outlook for Science‐Based Innovation

TLDR

Significant progress in pediatric drug development over the past 15 years has led to a positive outlook. Among 1,200 submitted pediatric studies, the FDA revised over 700 drug labels, established two international trial networks, and reduced pediatric efficacy trial failure rates from over 40 % to about 20 %.

Abstract

There has been significant progress in pediatric drug development during the past 15 years. Results from 1,200 pediatric studies have been submitted to the US Food and Drug Administration (FDA). Over 700 drug labels have been revised with information to guide pediatric use. Two international pediatric trial networks have been established. 1,2 The failure rate for pediatric efficacy trials has fallen from over 40% 3 to about 20%. Taken together, the outlook for pediatric drug development is positive.