FDA’s Makary Calls for Congress to Make Rare Pediatric Disease PRVs Permanent

Rare Daily Staff

U.S. Food and Drug Administration Commissioner Martin Makary is calling on Congress to make permanent a key incentive program for rare disease drug development, arguing that greater certainty could accelerate therapies for children with life-threatening conditions.

The request appears in the FDA’s fiscal 2027 budget justification, which calls for lawmakers to permanently authorize the rare pediatric disease priority review voucher program and related user fees. The proposal comes as part of a broader $7.2 billion FDA budget request for fiscal 2027. The move was first reported by Pharmaceutical Technology.

The Rare Pediatric Disease Priority Review Voucher program allows sponsors of approved rare pediatric disease therapies to receive a transferable voucher that shortens FDA review time for another product from 10 months to six.

The vouchers are potentially lucrative because they are transferable. Most recently, Cyprium Therapeutics sold its priority review voucher for $205 million to Fortress Biotech at the end of March.

Makary framed the program as both a public health and market-driven success. “This proposal would permanently reauthorize the RPD PRV program, creating more predictability for sponsors and ensuring children living with debilitating or life-threatening conditions have access to safe and effective therapies,” the agency said in its budget document.

The program has historically required periodic reauthorization by Congress, typically every four years, and has faced lapses in recent years, including a partial expiration in 2024. It is currently scheduled to sunset again in 2029.

Supporters argue that the stop-and-go nature of the program undermines its effectiveness, particularly for companies developing therapies that can take a decade or more to reach approval. A permanent authorization, they say, would provide clearer incentives for long-term investment in rare pediatric indications.

Since its creation, the FDA has awarded 63 priority review vouchers across 47 rare pediatric diseases, according to a 2025 report from the National Organization for Rare Disorders. Prior to the program, only four of those diseases had any FDA-approved treatments.

Photo: U.S. Food and Drug Administration Commissioner Martin Makary