The House Committee on Energy and Commerce has cleared proposed legislation that could bring back the FDA’s rare pediatric priority review voucher program, which allows for expedited drug reviews.
The FDA’s rare pediatric priority review vouchers are one step closer to becoming available to drugmakers again, after the House Committee on Energy and Commerce advanced a bill that would restart the program.
Lawmakers on Wednesday ushered the proposed legislation out of committee on Wednesday, according to reporting from Endpoints News. If it passes full House and Senate votes, this bill, dubbed the Give Kids a Chance Act of 2025, would keep the program alive through Sept. 30, 2029. The proposal will now move forward to be discussed and voted on by the entire House.
First enacted in 2012, the FDA’s rare pediatric priority review voucher (PRV) program is designed to incentivize drugmakers to work on rare diseases afflicting children. Companies that successfully secure an approval for a therapy treating a rare disease are then awarded a voucher that, if claimed, will qualify another of their products for priority review. Sponsors can also sell their vouchers to another sponsor for often much-needed revenue.
In September 2024, however, Congress failed to authorize the renewal of the program, forcing the FDA to “sunset” the system after Dec. 20, 2024. Speaking to BioSpace in March this year, Matthew Winton, chief operating officer of Inozyme Pharma, said the program had bipartisan support, but that it “got caught” in between timelines and changing priorities.
The consequences of this failure to keep the program running were immediate and far-reaching. According to a BioSpace analysis in March, pediatric rare disease vouchers accounted for the vast majority of these fast-track tickets that the FDA hands out in a year. In 2024, for instance, the agency issued 11 vouchers—the most of any year since the program was launched—10 of which were connected to pediatric rare disease development.
After the program went offline, many companies specializing in rare disease were forced to warn investors that they may never receive their vouchers. Among these are OS Therapies, Amylyx Pharmaceuticals and Solid Biosciences.
These rare disease PRVs, according to Winton, “feed the cycle of innovation and bringing drugs to patients in the U.S. who have no other options.”
Correction (Sept. 22): A previous version of this article stated that Xenon Pharmaceuticals warned investors it could be impacted after the rare pediatric PRV program went offline. In fact, it was Xenon’s collaborator, Neurocrine Biosciences, that was mentioned in Xenon’s SEC filing. BioSpace regrets the error.
