The EveryLife Foundation for Rare Diseases applauds the House of Representatives for passing the Give Kids a Chance Act, legislation that includes policies aimed at spurring progress in therapy development for rare diseases. 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 took action.
Rare Pediatric Priority Review Voucher Program Reauthorization
The Give Kids a Chance Act includes a 5-year renewal of the Rare Pediatric Priority Review Voucher Program previously contained in The Creating Hope Reauthorization Act of 2024. We are especially grateful to Representatives McCaul (TX-10), Bilirakis (FL-12), Burgess (TX-26), Barragán (CA-44), Eshoo (CA-16), and Trahan (MA-3) for their leadership and steadfast commitment to a timely reauthorization of the PRV Program.
The PRV Program has led to life-altering product approvals to treat almost 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 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 the FDA can continue to approve a drug that serves a different patient population, such as a pediatric approval for a drug that has exclusivity based on an adult approval. 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 today’s action in the House, without Congressional action, the Rare Pediatric Priority Review Voucher (PRV) Program will expire on September 30, 2024, leaving one less tool to bring treatments to a rare disease community in which only five percent of diseases have 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 ensure that the life-saving therapy development resource of the Pediatric Priority Review Voucher Program (PRV) does not lapse.
To learn more about engaging with your Senators to support reauthorization of the PRV program, and to access shareable resources, please visit https://www.rareadvocates.org/prv/
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The EveryLife Foundation for Rare Diseases is a nonprofit, nonpartisan organization dedicated to advancing the development of treatment and diagnostic opportunities for rare disease patients through science-driven public policy. The Foundation works to improve the lives of the millions of Americans suffering from rare diseases by advocating for policies that foster innovation and remove barriers to life-saving treatments. Founded in 2009 and headquartered in Washington, DC, the EveryLife Foundation for Rare Diseases has programs throughout the United States. To learn more, visit EveryLifeFoundation.org.
