U.S. President Donald Trump signed a spending package into law Tuesday that reauthorizes the FDA’s previously stalled rare pediatric disease priority review voucher program, among other initiatives, while ending a three-day partial government shutdown.
President Donald Trump signed a pared down spending bill on Tuesday that will end a partial government shutdown—and a long year of uncertainty for rare disease drug developers. The FDA’s rare pediatric disease priority review voucher program, which lapsed at the end of 2024, was reauthorized in the legislation.
The vote in the U.S. House of Representatives, upon which the legislation hinged, could scarcely have been tighter at 217-214. The continuing resolution is a stopgap measure that separates funding for the Department of Homeland Security (DHS) and Immigration and Customs Enforcement (ICE) from funding for other government divisions. The vote came after Trump and Senate Democrats reached the new deal last week.
For biopharma companies, specifically those developing therapies for rare diseases, Tuesday’s vote ends a long fight to reauthorize the rare pediatric priority review voucher (PRV) program—through a bill known as the Mikaela Naylon Give Kids a Chance Act—after Congress failed to re-up the program over a year ago.
The program, which began in 2012, provides a PRV to a company or other drug sponsor upon approval of a rare disease therapy. Companies can use the PRV to expedite the drug review timeline for another product, down to around six months from the usual 12 months, or they can also sell the voucher to gain much-needed revenue in a space that often doesn’t quite fit the typical pharmaceutical revenue model. PRVs go for around $150 million or more.
More than $4 billion in reinvestment dollars would have been left on the table if the bill had failed to pass in this legislative round, Stacey Frisk, executive director of the Rare Disease Company Coalition (RDCC), told BioSpace last month.
“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 added that the PRV program has already helped enable therapies across 47 rare pediatric indications.
The newly approved spending bill also allows for a $400 million increase in funding for the National Institutes of Health, which “sends a clear signal that Congress recognizes the essential role federal research investment plays in driving discovery and, ultimately, delivering cures,” Kendalle Burlin O’Connell, president and CEO of the Massachusetts Biotechnology Council said in a statement, also sent to BioSpace Tuesday evening.
“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,” O’Connell added.
With regard to PBM reform, the new legislation provides the Centers for Medicare and Medicaid Services (CMS) with $190 million to ensure these pharmaceutical “middlemen” comply with several new provisions, Endpoints News reported Tuesday. These include allowing CMS to define and enforce “reasonable and relevant” Medicare Part D contract terms and increasing transparency by allowing CMS to track payment trends to pharmacies and pharmacy inclusion in PBM networks.
PBM reform is a core tenet of President Donald Trump’s Most Favored Nation drug pricing scheme. “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,” the president said during the signing of an executive order related to the program in May.
Now, Democrats and Republicans will spend the next two weeks working to hammer out an agreement on the hot-button issue of immigration reform.
