The future of the rare pediatric disease priority review voucher (PRV) program remains uncertain as it is tied to a $1.2 trillion appropriations bill in Congress. The Mikaela Naylon Give Kids a Chance Act of 2025, which would reauthorize the PRV program, faces challenges due to its inclusion with other contentious proposals.
The PRV program began in 2012 and awards vouchers for approved therapies targeting rare pediatric diseases. These vouchers can be used to speed up future FDA reviews or sold by companies for additional revenue. Although there was unanimous support for renewal among members of the 118th Congress, health programs including the PRV were removed from a continuing resolution at the last minute.
Stacey Frisk, executive director of the Rare Disease Company Coalition (RDCC), told BioSpace last year that “the program—which began in 2012 and provides a voucher upon approval of certain therapies for rare pediatric therapies that can either be used to expedite the FDA review of a future program or sold for much needed revenue—had unanimous renewal support among the 118th Congress, which ended on Jan. 3, 2025.” However, she noted that health programs were stripped from legislative negotiations unexpectedly.
Industry leaders argue that losing access to these vouchers could threaten many small biotech companies focused on rare diseases. Bo Cumbo, CEO of Solid Biosciences, said: “That review voucher was worked into many people’s budgets to think about how much money you have to raise. So . . . [its removal] could be the final straw that breaks many rare disease companies’ backs.”
Efforts to revive the PRV program faced setbacks last month due to disagreements among senators over how best to reauthorize it. Senators Markwayne Mullin (R-OK), Bill Cassidy (R-LA), and Maggie Hassan (D-NH) called for immediate action, but Senator Bernie Sanders (I-VT) sought broader healthcare reforms within the same legislation, leading to its collapse.
Executives in the rare disease sector are calling not only for reauthorization but also for longer-term extensions aligned with drug development timelines. Dan Williams, cofounder and CEO of SynaptixBio—which develops treatments for TUBB4a-related leukodystrophy—explained: “I think that’s one of the big issues with the current system, is that they sunset and then renew far too early and far too quickly for biotech companies to really pick up and start a program and see it right the way through and gain those financial incentives promised through the voucher.”
Under current law, only therapies approved by September 30, 2026 qualify under a temporary extension; proposed legislation would extend this until September 30, 2029. Williams argued this period should stretch further—to at least 2030 or 2031—to accommodate typical clinical development cycles in rare diseases.
Williams added: “It needs to be a time that fits rare disease development,” suggesting an ideal window is seven to ten years rather than three.
Justin To from BridgeBio agreed: “Every few years, you’re kind of back to the starting point, and for a variety of different reasons, sometimes things get stalled.” He said longer authorization cycles would provide predictability within biopharma focused on rare conditions.
Cumbo concluded: “Many of us have children that suffer from a rare disease or a genetic disorder. This should be a no-brainer for both Republicans and Democrats.”
Congress did not renew authorization at end-2024 despite advocacy efforts; industry observers say this has already affected investment decisions across biotechnology firms working on treatments for rare diseases.
