The U.S. House of Representatives narrowly passed a spending bill that reauthorizes the FDA’s rare pediatric disease priority review voucher (PRV) program, which had lapsed at the end of 2024. President Donald Trump signed the legislation on Tuesday, ending a partial government shutdown and providing clarity for rare disease drug developers.
The House approved the continuing resolution by a margin of 217-214. The measure separates funding for agencies such as the Department of Homeland Security and Immigration and Customs Enforcement from other government divisions, following an agreement reached between Trump and Senate Democrats last week.
The PRV program, established in 2012, awards vouchers to companies whose therapies for rare diseases receive approval. These vouchers can be used to speed up the FDA review process for another product or sold to generate revenue. The typical value of these vouchers is around $150 million.
Stacey Frisk, executive director of the Rare Disease Company Coalition (RDCC), highlighted what was at stake if Congress had not acted: more than $4 billion in reinvestment dollars could have been lost. “This milestone brings renewed hope to families awaiting lifesaving treatments for children with rare diseases and reaffirms a bipartisan commitment to advancing innovation for pediatric patients who urgently need therapeutic breakthroughs,” Frisk said in an email statement to BioSpace on Tuesday. She noted that the PRV program has enabled therapies across 47 rare pediatric indications.
A BioSpace analysis found that 2024 saw record activity in priority review vouchers across all three FDA programs offering them, with companies disclosing $513 million spent on vouchers earned during that year.
The new spending bill also increases funding for the National Institutes of Health by $400 million. Kendalle Burlin O’Connell, president and CEO of the Massachusetts Biotechnology Council, commented on this development: “It sends a clear signal that Congress recognizes the essential role federal research investment plays in driving discovery and, ultimately, delivering cures.” O’Connell added, “We are also encouraged to see Congress act on PBM reform and the reauthorization of the Rare Pediatric Disease Priority Review Voucher, which incentivizes the development of treatments for children facing the rarest and most devastating diseases.”
In addition to PRV reauthorization, the legislation allocates $190 million to help Centers for Medicare and Medicaid Services (CMS) implement reforms related to pharmacy benefit managers (PBMs). These measures will give CMS authority over contract terms in Medicare Part D plans and increase transparency regarding payments to pharmacies.
President Trump emphasized PBM reform as part of his Most Favored Nation drug pricing plan: “We’re going to cut out the middlemen and facilitate the direct sale of drugs at the most favored nation price directly to the American citizen,” he said when signing an executive order related to this initiative in May.
With these budget issues resolved, lawmakers are expected to focus next on immigration reform negotiations over the coming weeks.
