Congress Extends Rare Pediatric Disease Priority Review Voucher Program to 2029

The Consolidated Appropriations Act, 2026 became law on February 3, 2026 and extends the Food and Drug Administration’s authority to issue rare pediatric disease priority review vouchers through September 30, 2029. The FDA initiative incentivizes drug development for rare pediatric conditions by awarding priority review vouchers to sponsors of approved therapies, and these vouchers allow expedited review of future drug applications.

Section 6604 of the law extends the FDA’s authority to issue priority review vouchers to encourage treatments for rare pediatric diseases through September 30, 2029. While the extension provides certainty to this program for the next few years, newly enacted reporting provisions included with the extension also set the stage for continued assessments of the impact of this program.

Section 6604 requires the Government Accountability Office to study and submit a report to Congress on the effectiveness of awarding rare pediatric disease priority vouchers in the development of products that treat or prevent rare pediatric diseases not later than five years after enactment of this provision. The analysis is likely to be submitted after the current program authority extension is set to expire.

The program, extended until September 30, 2029, has already made a significant impact. During a 2025 FDA roundtable on cell and gene therapies, the director of the Center for Biologics Evaluation and Research emphasized the agency’s commitment to flexibility for therapies targeting rare diseases.

The current Prescription Drug User Fee Act program is set to expire on September 30, 2027. Rare disease issues have been a prominent part of prior PDUFA reauthorizations, and policymakers continue to examine how to spur the timely development of safe and effective therapies for rare disease patients as they address unique development challenges, urgent patient needs and advances in innovation.