USA – Rare Pediatric PRV Program Not Linked to Uptick in New Drugs Starting Trials, Study Finds

The rare pediatric priority review voucher (PRV) program was not found to be associated with an increase in the number or rate of new rare pediatric disease drugs that began clinical trials, a Health Affairs study found.

But the researchers from Harvard Medical School, working on what they said is the first study on the impact of the rare disease PRV program on drug development, also found that their data do provide some encouraging news about rare pediatric disease drug development.

“Such [rare pediatric] drugs were more likely to advance from Phase I to Phase II trials, compared to drugs for rare adult diseases, after the PRV program’s creation, although the same difference was not observed for progress in later stages of development,” they write.

The authors also found that compared to drugs for rare adult diseases, drugs for rare pediatric diseases progressed more quickly through all phases of clinical testing and were more likely to be first-in-class…