#United States #Rare Pediatric Disease #Washington D.C. #NORD #Give Kids a Chance

Urgent Call to Action for Rare Children in America

The National Organization for Rare Disorders (NORD) has recently highlighted the pressing need for the U.S. Congress to reauthorize the Rare Pediatric Disease Priority Review Voucher (RPD PRV) program by the end of the year. This initiative has been critical in advancing treatments for rare diseases affecting over 15 million children nationwide. The urgency of reauthorization is underscored by the potential lapse in the program, which threatens to stall innovation and jeopardize the health of countless children who rely on groundbreaking therapies.

Significant Progress Made

A report from NORD shows that since the inception of the RPD PRV program in 2012, the FDA has awarded 63 vouchers which have facilitated treatment breakthroughs for 47 rare pediatric diseases. Alarmingly, 43 of these diseases previously had no existing treatment options. This significant progress marks an unprecedented step forward in ensuring that children afflicted with rare diseases are not left behind in the quest for viable medical solutions.

With recent legislative actions such as the passage of the Give Kids a Chance Act (H.R. 1262 /S. 932) by the U.S. House of Representatives, there is hope that the Senate will act swiftly to ensure that these vital incentives for drug development are protected. The proposed legislation aims to extend the life of the PRV program for at least another five years, crucially needed as authority for the FDA to grant these vital designations is set to expire in 2024.

Real-World Impacts

The benefits of the RPD PRV program are tangible. In 2025, four vouchers were issued for therapies addressing severely rare conditions, such as neurofibromatosis type 1 and thymidine kinase 2 deficiency (TK2d). One notable success is the first FDA-approved treatment for Friedreich's ataxia, a life-threatening genetic disorder affecting around 4,000 children in the U.S. This treatment offers real hope, enhancing coordination and stability for affected children.

Despite these advances, the clock is ticking. Delays in reauthorization threaten not only to halt further innovations but also to create uncertainty for investors and developers who are essential players in the pediatric drug development landscape. The stakes could not be higher; every moment without action places the future of countless children in jeopardy.

A Unified Call for Action

NORD's CEO, Pamela K. Gavin, has articulated the critical nature of this moment: "Every day without reauthorization creates uncertainty that disrupts research and delays innovation. This uncertainty jeopardizes the hope and progress for children with life-threatening rare diseases who simply cannot afford to wait." This sentiment echoes the urgency felt by congressional sponsors and advocates alike, underscoring the pressing need for prompt legislative action.

The fabric of support from various stakeholders, including the Muscular Dystrophy Association and families affected by conditions like Lennox-Gastaut Syndrome, emphasizes that the PRV program is the most effective strategy available to stimulate development in treatments for ultra-rare pediatric diseases. It fosters investment in therapies that would otherwise remain undeveloped due to the high costs and uncertain market return.

Conclusion

As December draws near, the call to action is clear: Congress must prioritize the reauthorization of the Rare Pediatric Disease Priority Review Voucher program. The momentum established by the House's recent vote must be translated into swift action by the Senate to preserve a vital lifeline for America’s rare children. NORD encourages advocates to reach out to their Senators, urging them to support the Give Kids a Chance Act, ensuring continued breakthroughs for children desperately awaiting treatment. With a legislative victory on the horizon, there is hope for a brighter future for those impacted by rare pediatric diseases.