FDA Issues Draft Guidance on Rare Pediatric Diseases

FDA has issued the draft guidance, Rare Pediatric Disease Priority Review Vouchers, Guidance for Industry, which provides information on the implementation of section 908 of the Food and Drug Administration Safety and Innovation Act, which added section 529 to the Federal Food, Drug, and Cosmetic Act.  According to the guidance, FDA will award priority review vouchers to sponsors of certain rare pediatric disease product applications that meet the criteria.

The draft guidance provides information for developers of rare pediatric disease products so they may assess whether their product may be eligible for rare pediatric disease designation and a rare pediatric disease priority review voucher. The draft guidance also clarifies the process for requesting rare pediatric disease designation and vouchers. It details sponsor responsibilities upon approval of a rare pediatric disease product application and the parameters for using and transferring a rare pediatric disease priority review voucher.