{"id":282324,"date":"2025-12-04T12:24:36","date_gmt":"2025-12-04T17:24:36","guid":{"rendered":"https:\/\/rarediseases.org\/?p=282324"},"modified":"2025-12-22T14:15:22","modified_gmt":"2025-12-22T19:15:22","slug":"nord-urges-swift-reauthorization-of-proven-rare-pediatric-disease-voucher-program","status":"publish","type":"post","link":"https:\/\/rarediseases.org\/es\/nord-urges-swift-reauthorization-of-proven-rare-pediatric-disease-voucher-program\/","title":{"rendered":"America\u2019s Rare Children Need Congress to Act: NORD Urges Swift Reauthorization of Proven Rare Pediatric Disease Voucher Program"},"content":{"rendered":"

NORD report shows 63 FDA vouchers driving breakthroughs for 47 rare pediatric diseases, 43 with no prior treatments \u2014 unprecedented progress for children left behind<\/em><\/p>\n

\"Report<\/p>\n

WASHINGTON, Dec. 4, 2025<\/strong> \u2014 The National Organization for Rare Disorders (NORD\u00ae<\/sup>) today urged Congress to reauthorize the Rare Pediatric Disease Priority Review Voucher (RPD PRV) program by year-end, warning that continued delay threatens the future of innovation for children with rare diseases.<\/p>\n

On Dec. 1, the U.S. House of Representatives passed the Give Kids a Chance Act (H.R. 1262 \/S. 932), an important step forward in protecting incentives for rare pediatric drug development. The bill will now move to the Senate for consideration.<\/p>\n

According to NORD\u2019s original research<\/strong><\/a>, the Food and Drug Administration (FDA) has awarded 63 vouchers since the program\u2019s creation in 2012, leading to treatments for 47 rare pediatric diseases, 43 of which previously had no treatment options. More than half of all designations have occurred in just the past five years, underscoring the program\u2019s growing impact and bringing real hope to the more than 15 million children living with a rare pediatric disease, 30% of whom will not live to see their fifth birthday.<\/p>\n

Urgency to Reauthorize<\/strong><\/h2>\n

With the FDA’s authority to grant rare pediatric designations expiring in 2024, and its authority to grant priority review vouchers expiring on Sept. 30, 2026, the delay in reauthorization fuels uncertainty in the marketplace for investors and pediatric drug developers.<\/p>\n

\u201cThe PRV program has been instrumental in encouraging the development of new therapies for rare pediatric diseases. Since its inception in 2012, this program\u2019s success has resulted in more American children with rare diseases having a chance at a fuller, healthier life, and gives hope to communities still waiting for a treatment option for their condition. Congress must move on reauthorization now. \u00a0Timely restoration of the program will provide needed certainty within the U.S. pediatric treatment marketplace and prevent devastating consequences for children with rare diseases who are waiting for new treatments,\u201d said Rep. Gus Bilirakis (R-Fla.), an original cosponsor of the Give Kids a Chance Act (H.R. 1262)<\/strong>.<\/p>\n

\u201cEvery day without reauthorization creates uncertainty that disrupts research and delays innovation,\u201d said Pamela K. Gavin, NORD Chief Executive Officer<\/strong>. \u201cThat uncertainty puts both hope and progress at risk for children with life-threatening rare diseases who simply cannot afford to wait.\u00a0 Congress has a clear choice: reauthorize a program that works or allow progress to stall for kids who have no other options.\u201d<\/p>\n

\u201cThe RPD PRV is the most important incentive for developing new therapies for ultra-rare neuromuscular diseases. Already, we have seen investors and companies shy away from developing life-changing treatments for our ultra-rare community, and without rapid reauthorization, it will only get worse. MDA joins the rare disease community in calling for the program’s immediate reauthorization,” said Paul Melmeyer, EVP, Public Policy and Advocacy, Muscular Dystrophy Association<\/strong>.<\/p>\n

Proven Results<\/strong><\/h2>\n

NORD, in a white paper titled “Impact of the Rare Pediatric Disease Priority Review Voucher Program on Drug Development 2012 – 2025,”<\/a><\/strong>\u00a0confirms the RPD PRV program is a measured, effective incentive that accelerates development for rare pediatric conditions.<\/p>\n