The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates have been fighting to get it back ever since.
In our uniquely acrimonious political climate, it’s somewhat ironic—and quite sad—that one of the few policy proposals that enjoys bipartisan support is caught up in a dicey spending bill. The Mikaela Naylon Give Kids a Chance Act of 2025, designed to reauthorize the rare pediatric disease priority review voucher (PRV) program that got lost in the shuffle of changing timelines and priorities 16 months ago, could fail to receive Congress’ approval simply because it’s packaged within a massive $1.2 trillion appropriations bill with far less harmonious proposals.
That’s government red tape for you, but the tape rips a little harder when it’s kids who will suffer.
And because the PRV program never needed to lapse in the first place.
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, Stacey Frisk, executive director of the Rare Disease Company Coalition (RDCC), told BioSpace in March last year. However, health programs—including the PRV program—were stripped from a continuing resolution at the last minute.
Ever since, Frisk and other rare disease leaders have been fighting for reauthorization of the program—which they say is vital to the survival of the sector.
“That review voucher was worked into many people’s budgets to think about how much money you have to raise,” Bo Cumbo, CEO of Solid Biosciences, told me. “So . . . [its removal] could be the final straw that breaks many rare disease companies’ backs.”
The PRV program had a chance to be back up and running last month, but instead, it was held back by disagreements between senators. Sens. Markwayne Mullin (R-OK), along with Bill Cassidy (R-LA) and Maggie Hassan (D-NH), implored the Senate body in late December to reauthorize the program, but Sen. Bernie Sanders (I-VT) wanted more, according to reporting by Endpoints News. The perennially outspoken senator sought to package a voucher provision into a broader healthcare bill that Mullin and other Republicans did not support, and the bill collapsed.
Rare disease executives I spoke with this week are eager first to secure reauthorization through the current spending bill but also noted a potential solution to avoid future lapses: Extend the lifespan of the PRV program to match the rare disease drug development lifecycle.
“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,” said Dan Williams, cofounder and CEO of SynaptixBio.
SynaptixBio launched in 2021 to take on one of the world’s rarest diseases: TUBB4a-related leukodystrophy. Usually affecting infants and young children, leukodystrophies are life-limiting genetic disorders that affect the brain’s white matter, affecting movement and cognitive development, among other functions.
Under a temporary extension, rare disease therapies approved by Sept. 30, 2026 still qualify for a PRV, but SynaptixBio’s programs are too early-stage for that. A renewal of the current legislation would keep the vouchers going for another three years, until Sept. 30, 2029. But Williams said the program would need to extend through 2030 or 2031 to enable the British biotech to complete clinical development and move through an accelerated approval.
“It needs to be a time that fits rare disease development,” Williams said, noting that for rare disease therapies, with the accelerated approval pathways that are available, this would be approximately 7–10 years, not three. For comparison, the Prescription Drug User Fee Act (PDUFA), which allows the FDA to collect fees from pharma companies filing product applications for review, is renewed every five years.
Justin To, CEO of Skeletal Dysplasia programs at 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. A longer authorization cycle would provide predictability to the rare disease ecosystem, To added.
It would also have the added benefit of keeping this critical program out of the political pork barrel for longer. And that can only be a good thing—for biotech, for innovation and for our children.
“Many of us have children that suffer from a rare disease or a genetic disorder,” Cumbo said. “This should be a no-brainer for both Republicans and Democrats.”
