Impact of the Priority Review Voucher Program on Drug Development for Rare Pediatric Diseases

Drugs for rare pediatric diseases progressed faster through all phases of clinical testing and were more likely to be first-in-class compared to drugs for rare adult diseases. The pediatric rare disease priority review voucher program was not associated with a change in the rate of new pediatric drugs starting or completing clinical testing, but there was a significant increase in the rate of progress from Phase I to Phase II clinical trials after the program was implemented, suggesting that new policies may be needed to further advance the pipeline of therapies for rare pediatric diseases.