FDA Updates Guidance on Rare Pediatric Disease Priority Review Voucher Program

Drugmakers have been given more clarity on the FDA’s definition of rare pediatric disease and the process for its priority review voucher program in draft revised guidance.

The guidance addresses drug companies’ confusion over whether sponsors must receive a rare pediatric disease designation to be eligible for a voucher. At present, the designation is not required to receive a voucher.

However, FDA strongly recommends that sponsors request designation because under current law the agency “may not award any vouchers after September 30, 2020, unless the application is for a drug that was designated as a drug for a rare pediatric disease by September 30, 2020.”