The rare disease priority review voucher (PRV) program remains stalled in Congress, creating uncertainty for biotechnology companies that depend on it to support the development of treatments for rare diseases. The program's reauthorization has been delayed as part of a larger dispute over funding for the Department of Homeland Security (DHS), which includes Immigration and Customs Enforcement (ICE) operations. The Senate recently voted 55–45 against a spending bill that included the PRV program, with DHS funding being a point of contention.
A temporary extension allows rare disease drugs approved by September 30, 2026, to still qualify for a PRV. However, biotech leaders have expressed concern about the ongoing delay. Brett Monia, CEO of Ionis, told BioSpace at the J.P. Morgan Healthcare Conference: “We think it’s absolutely critical to support innovative research, to support pediatric, rare disease medicines coming forward.”
Monia said his company is working quickly to submit zilganersen for FDA review to help patients with Alexander disease. He emphasized that while getting treatments to patients is the main goal, obtaining a PRV is also important: “Getting the drug to patients, of course, is number one. But not losing out on a potential PRV is one of our other objectives,” he said. “Our team has worked through the holidays to get the NDA submitted as quickly as possible to meet that deadline.”
Priority review vouchers can be sold or used by companies; recent transactions have valued them at around $150 million, with Jazz Pharmaceuticals reportedly selling one for $200 million earlier this month.
An analysis by the Rare Disease Company Coalition suggests that if the PRV program is not reinstated soon, up to 200 therapies could lose eligibility for these vouchers—a scenario that could result in an estimated $4 billion in lost revenue for biotechs focused on rare diseases.
Justin To, CEO overseeing skeletal dysplasia programs at BridgeBio, said: “The PRV directly impacts how many of our rare-disease shots on goal we can fund and move into the clinic.”
Catherine Owen Adams, CEO of Acadia Pharmaceuticals, has also advocated for reauthorizing the program and highlighted its impact on investment: “Since that’s come into being, we’ve seen an acceleration of rare drug approvals because the innovation has been worthwhile in terms of the large investment for small patient populations,” she said.
Both Monia and Owen Adams noted their belief that Congress must resolve this issue rather than the FDA. Monia mentioned meeting with FDA Commissioner Marty Makary last year and reported receiving positive signals regarding agency support for continuing the program.
Owen Adams described broad bipartisan understanding and support during her lobbying efforts but acknowledged continued uncertainty: “There does seem to be widespread understanding and support that this is a good thing for rare disease patients, as well as American innovation,” she stated. “I feel vaguely positive that it is going to be renewed. But, you know, stranger things have happened.”
As lawmakers work toward separating DHS funding from other budget items—an effort likely delaying any immediate resolution—biopharma companies are preparing themselves for further waiting amid ongoing concerns about missed opportunities in developing treatments for rare diseases.
