Bipartisan House Bill Seeks Permanent Rare Pediatric Disease Voucher Program

Rare Daily Staff

A bipartisan group of House lawmakers has introduced legislation that would permanently authorize the FDA’s Rare Pediatric Disease Priority Review Voucher program, seeking to give drug developers greater long-term certainty to invest in treatments for children with rare illnesses.

Reps. Gus Bilirakis, R-Fla.; Nanette Barragán, D-Calif.; Kat Cammack, R-Fla.; Jake Auchincloss, D-Mass.; Tom Kean Jr., R-N.J.; and Kevin Mullin, D-Calif., introduced the legislation. The bill would eliminate the program’s current 2029 expiration date.

The Rare Pediatric Disease Priority Review Voucher program gives a company that receives FDA approval for a qualifying therapy a transferable voucher for priority review of a future product application. The company can use the voucher itself or sell it to another drugmaker.

Supporters argue that the incentive helps offset the financial challenges of pursuing therapies for diseases affecting relatively small patient populations. The sponsors said rare-disease drug development can take as long as 15 years, making a 2029 sunset date insufficient for companies deciding now whether to begin or continue lengthy clinical-development programs.

“Parents of children battling rare diseases should never have to hear that promising research was abandoned because there wasn’t enough incentive to pursue a treatment,” Bilirakis said.

Since the program was created in 2012, sponsors said it has helped bring treatments to market for 47 rare pediatric diseases, only four of which had an FDA-approved treatment before the program began. FDA has awarded 63 vouchers to date, according to the release, and more than 90 percent went to therapies for indications that previously had no approved treatment, the lawmakers said.

The proposal follows a recent lapse in the program, which was unavailable from December 2024 through February 2026. The Rare Disease Company Coalition said about 200 therapies risked losing voucher eligibility during that interval and estimated that the disruption put more than $4 billion in potential reinvestment capacity at risk.

The legislation has support from the National Organization for Rare Disorders, the Rare Foundation, the Biotechnology Innovation Organization and the Rare Disease Company Coalition.