NORD report shows 63 FDA vouchers driving breakthroughs for 47 rare pediatric diseases, 43 with no prior treatments—unprecedented progress for children left behind

WASHINGTON, Dec. 4, 2025 /PRNewswire/ -- The National Organization for Rare Disorders (NORD®) 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.

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. 

According to NORD's original research, the FDA has awarded 63 vouchers since the program's 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's 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. 

Urgency to Reauthorize
With the Food and Drug Administration's (FDA) authority to grant rare pediatric designations expiring in 2024, and FDA's 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.

"The PRV program has been instrumental in encouraging the development of new therapies for rare pediatric diseases. Since its inception in 2012, this program's 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. Timely 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," said Rep. Gus Bilirakis (R-Fla.), an original cosponsor of the Give Kids a Chance Act (H.R. 1262). 

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

"The 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. 

Proven Results:
NORD, in a white paper titled "Impact of the Rare Pediatric Disease Priority Review Voucher Program on Drug Development 2012 - 2025," confirms the RPD PRV program is a measured, effective incentive that accelerates development for rare pediatric conditions. 

• In 2025 alone, four vouchers were granted for life-saving therapies addressing severe rare diseases such as neurofibromatosis type 1, diffuse midline glioma with H3 K27M mutation, Barth syndrome and thymidine kinase 2 deficiency (TK2d). 

•  29 RPD PRVs have been redeemed for priority review of 26 different drugs for a variety of diseases, including eight drugs to treat rare diseases. 

• Of the top 50 Medicare-spend drugs in 2023, only three were approved through a redeemed voucher and fewer than half were among the top 100 in Medicare Parts B or D spend. 

"In 2023, the Friedreich's ataxia (FA) community saw a long-awaited breakthrough: the first and only FDA-approved treatment for a progressive, life-shortening disease that begins in childhood and affects about 4,000 people in the U.S. This therapy offers meaningful improvements in stability, coordination, and fatigue, bringing families hope where there had been none," said Jennifer Farmer, Friedreich's Ataxia Research Alliance, Chief Executive Officer. "This milestone may not have been possible without the Rare Pediatric Disease Priority Review Voucher program," added Farmer. 

A Bipartisan Opportunity
NORD is calling on lawmakers to pass the bipartisan Give Kids a Chance Act before year-end, extending the program for at least another five years. "A year-end reauthorization would be a bipartisan victory," said NORD's Gavin. "America's rare children cannot afford another month of inaction." 

"Lennox-Gastaut Syndrome (LGS) is a devastating childhood epilepsy, and for years, families had no options. The PRV program finally drove investment in treatments that would otherwise never have been developed," said Tracy Dixon-Salazar, LGS Foundation Executive Director. "These therapies don't just improve quality of life; they help prevent lifelong disability, reduce medical crises, and ease enormous emotional and financial strain," added Dixon-Salazar. 

The Give Kids a Chance Act would extend the Rare Pediatric Disease Priority Review Voucher program for at least five more years, protecting one of the few incentives proven to drive new treatments for children with rare diseases. The House passed its version on Dec. 1, but the Senate must act quickly to keep this lifesaving program alive. 

Urge your Senators to support the Give Kids a Chance Act and help ensure continued breakthroughs for kids who urgently need them. Act with a NORD ACTION ALERT HERE. 

Learn More 

• Full NORD RPD PRV Report: HERE 
• One-Pager Summary: HERE 
• NORD ACTION ALERT: HERE 

About NORD 
Founded in 1983, the National Organization for Rare Disorders (NORD®) is the leading independent nonprofit dedicated to improving the health and lives of the 30+ million Americans living with rare diseases. In partnership with more than 350 member organizations, NORD drives policy, research, education, and patient support nationwide. Learn more at rarediseases.org. 

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SOURCE National Organization for Rare Disorders (NORD®)