Rare Pediatric Disease Priority Review Voucher (PPRV) Program

What Is the Rare Pediatric Disease Priority Review Voucher Program?

The Rare Pediatric Disease Priority Review Voucher (PPRV) program is an FDAincentive that encourages pharmaceutical companies to develop treatments for rare diseases affecting children. Under the program, when FDA approves a drug for a rare pediatric disease, the sponsor receives a Priority Review Voucher — a transferable voucher that can be used or sold to receive faster FDA review (within 6 months instead of the standard 10 months) on a future drug application. This creates a financial incentive for companies to invest in rare pediatric disease treatments, which typically have small patient populations and limited commercial return. The PRV program was originally established through the Creating Hope Act in 2012. Since its creation, FDA has awarded more than 60 priority review vouchers for treatments addressing nearly 40 rare pediatric diseases, benefiting more than 200,000 children.

Reauthorization: The Mikaela Naylon Give Kids a Chance Act

The PRV program lapsed at the end of 2024 after Congress failed to include its reauthorization in end-of-year legislation. After a sustained bipartisan advocacy campaign, the program was reauthorized in February 2026 through the Mikaela Naylon Give Kids a Chance Act (H.R. 1262), which was signed into law as part of a continuing resolution. The Act extends PRV eligibility for medicines achieving FDA approval through September 30, 2029. The legislation also authorizes FDA to direct companies to study combinations of cancer drugs and therapies in pediatric trials — addressing the gap where thousands of combination therapies are being developed for adult cancers but not for children. It is named in honor of Mikaela Naylon, a 16-year-old osteosarcoma patient who advocated for the bill until her passing. The Save Rare Treatments Task Force supported reauthorization of the PRV program and continues to advocate for its protection.