FRIEDREICH'S ATAXIA RESEARCH ALLIANCE

Rare Pediatric Disease Voucher Program Faces Uncertain Future as Congressional Reauthorization Stalls

- The FDA has awarded 63 rare pediatric disease priority review vouchers since 2012, leading to treatments for 47 rare pediatric diseases, with 43 having no prior treatment options. - Senator Bernie Sanders blocked renewal of the program due to concerns about pharmaceutical industry profits and pricing practices, causing uncertainty for future rare disease drug development. - The House passed the Give Kids a Chance Act in December 2025, but Senate action is needed to reauthorize the program that provides expedited FDA review incentives for rare pediatric treatments. - More than half of all voucher designations have occurred in the past five years, demonstrating the program's growing impact for over 15 million children with rare diseases.