Legislation would permanently reauthorize the Rare Pediatric Disease Priority Review Voucher program and provide long-term certainty for rare disease innovation
Washington, D.C. — The Rare Disease Company Coalition (RDCC) applauds the introduction of the bipartisan Priority for Pediatric Cures Act, led by Congressman Gus Bilirakis alongside original co-leads Representatives Nanette Barragán, Kat Cammack, Jake Auchincloss, Tom Kean Jr., and Kevin Mullin.
The legislation would permanently reauthorize the Rare Pediatric Disease Priority Review Voucher (PPRV) program, providing long-term certainty for an incentive that has helped support the development of therapies for children living with rare diseases.
“The Rare Pediatric Disease Priority Review Voucher program is a proven, budget-neutral incentive that spurs innovation and supports the development of new therapies for children living with rare diseases,” said Chris Porter, Chair, Rare Disease Company Coalition. “During the program’s lapse from December 2024 to February 2026, more than 200 pipeline assets were at risk of not receiving a priority review voucher, representing more than $4 billion in potential lost reinvestment capacity. Permanently reauthorizing the program will provide the certainty and predictability rare disease innovators need to continue investing in new treatments for patients with few or no existing options. The Rare Disease Company Coalition applauds Representatives Bilirakis, Auchincloss, and Barragán for their leadership and for taking action to make the PPRV program a permanent part of our nation’s rare disease innovation ecosystem.”
For companies developing therapies for rare pediatric diseases, predictability is especially important given the significant time, investment, and uncertainty involved in bringing a new treatment from development to children in need. Permanent authorization of the PPRV program would help remove the recurring uncertainty surrounding the program’s future and allow innovators to make longer-term research and development decisions with greater confidence.
RDCC looks forward to working with bipartisan leaders in Congress to advance the Priority for Pediatric Cures Act and support policies that encourage continued investment in treatments for children and families affected by rare diseases.
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