The EveryLife Foundation for Rare Diseases applauds the House of Representatives for passing the Give Kids a Chance Act (H.R. 1262), legislation that includes policies that will spur progress in therapy development for rare diseases, most notably, the reauthorization of the Rare Pediatric Disease Priority Review Voucher (PRV) Program. Today’s milestone comes as a result of community-driven advocacy and dedication from rare disease patients, families, organizations, and others who shared their stories and used their voices to ensure Congress acted.
Rare Pediatric Priority Review Voucher Program Reauthorization
The Give Kids a Chance Act includes a 5-year renewal of the Rare Pediatric Disease Priority Review Voucher Program, which lapsed in December 2024. We are especially grateful to the original bill leads, Representatives McCaul (TX-10), Dingell (MI-6), Bilirakis (FL-12), Matsui (CA-7), Schrier (WA-8), Harshbarger (TN-1), Castor (FL-14), Kelly (PA-16), Crenshaw (TX-2), Trahan (MA-3), and Weber (TX-14) for their leadership and steadfast commitment to restoring the PRV Program’s authorization.
The PRV Program has led to life-altering product approvals to treat over 40 rare diseases, such as Progeria syndrome, Spinal Muscular Atrophy, Sickle Cell Disease, Rett syndrome, and other conditions that previously lacked any FDA-approved treatments.
Retaining Access and Restoring Exclusivity Act (RARE Act)
By passing the Give Kids a Chance Act, Congress also approved language from the RARE Act, clarifying that the Orphan Drug Act’s market exclusivity period should be applied based on the approved use or indication a drug is approved for, rather than applying to all uses within a disease or condition, as a court ruled in a 2021 case. If signed into law, the RARE Act would ensure that incentives to study different segments of a given patient population remain. We are grateful to the RARE Act’s lead sponsors, the House co-chairs of the Rare Disease Congressional Caucus, Representatives Doris Matsui (CA-7) and Gus Bilirakis (FL-12).
Further Action Needed
While we celebrate the House passage of the Give Kids a Chance Act in the 119th Congress, we recognize that this is only the first hurdle. Despite last year’s efforts, the Rare Pediatric Disease (PRV) Program expired in December 2024, leaving one less tool to bring treatments to the rare disease community. The impact of this is seen in clinical trial delays, not by science, but by financial limitations. With only five percent of diseases having an FDA-approved treatment, it is imperative that we continue to advance solutions to treat these devastating diseases, 70% of which start in childhood.
The EveryLife Foundation calls on the Senate to act swiftly and pass the Give Kids a Chance Act (S.932) to ensure that life-saving therapy development remains a resource for innovation and treatment.
To learn more about engaging with your Senators to support reauthorization of the PRV program, and to access shareable resources, please click here.
