Rare paediatric disease voucher programme makes long-awaited US return

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Source: Pharmaceutical Technology

Original: https://www.pharmaceutical-technology.com/news/rare-paediatric-disease-voucher-p...

Published: Thu, 05 Feb 2026 19:37:34 +0000

The Rare Pediatric Disease Priority Review Voucher (RPD PRV) program provides sponsors of approved drugs and biologics for the treatment of rare pediatric diseases a priority review voucher to expedite FDA review of future applications.[1][3] The sponsor can use the voucher for a priority review of another product or sell it to another entity.[3][5] The program ended in December 2024, but the law allows for vouchers for applications designated as rare pediatric diseases until December 20, 2024 and approved until September 30, 2026.[1][3] The Mikaela Naylon Give Kids a Chance Act (S.932/H.R.1262), included in the Continuing Appropriations Act of 2026, reauthorizes the program and extends the vouchers until September 30, 2029.[1][4] Since its launch in 2012, 63 vouchers have been awarded for 47 rare pediatric diseases, only 4 of which had FDA-approved treatments before the program.[4][6] The program supports the development of therapies, including cell and gene therapies, for children without available treatment options.[4][6]