Crucial PRV Program Lapsed in December, Will Sunset Without Congressional Action. Give Kids a Chance Act Would Revive Program, Provide Necessary Incentives to Spur Research and Development of Rare Disease Treatments
Washington, DC – The Rare Disease Company Coalition (RDCC) applauds the introduction of the Give Kids a Chance Act, which would reauthorize the rare pediatric disease priority review voucher (PRV) program. The program began to lapse on December 20, 2024 and without urgent Congressional action, hope for millions of children may vanish for good. We thank Representative Michael McCaul (R-TX) for his leadership on this bill, and original sponsors, Representatives Gus Bilirakis (R-FL), Debbie Dingell (D-MI), Kim Schrier (D-WA), Diana Harshbarger (R-TN.), Kathy Castor (D-FL), Mike Kelly (R-PA), Doris Matsui (D-CA), Dan Crenshaw (R-TX), Lori Trahan (D-MA) and Randy Weber (R-TX), for their support.
The rare pediatric disease PRV program is critical to providing hope for the millions of children in the United States living with a rare disease, 30 percent of whom will not live to see their fifth birthday. The program has led to new innovations benefiting over 200,000 patients and addressing high unmet needs across 47 rare pediatric indications, at little to no cost to taxpayers.
“Since just before Christmas last year, millions of children and their families have faced uncertainty about the future of the lifesaving rare pediatric disease PRV program, a proven tool that has led to groundbreaking innovations in rare disease therapies,” said Stacey Frisk, Executive Director of the Rare Disease Company Coalition. “Small, emerging biopharmaceutical companies rely on the PRV program to stabilize investment in new treatments. Every minute the PRV program remains expired jeopardizes investment in critical innovations that could transform the lives of children living with rare, often fatal and progressively debilitating, diseases.”
Passage of the Give Kids a Chance Act would ensure that the rare pediatric disease PRV program will continue to benefit hundreds of thousands of patients living with a rare disease. The program has received broad, bipartisan, and bicameral support since its inception in 2012. Most recently in the 118th Congress, the program enjoyed unanimous passage through the House of Representatives and was included in the health title of the end-of-year package. Further, the Food and Drug Administration (FDA) has supported the reauthorization of the program via inclusion in their anomalies list. A diverse array of stakeholders, from Congress to the Administration, from patient advocates to academia, and from the investor community to rare disease innovators, understand that the PRV program is a proven innovation-driving and cost-effective policy that spurs research & development in rare pediatric diseases.
The Rare Disease Company Coalition applauds Congressman McCaul and all cosponsors for their leadership on the Give Kids a Chance Act, and urges Congress to act quickly to restore the rare pediatric disease priority review voucher program and ensure children can get access to the treatment they desperately need.
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About the Rare Disease Company Coalition (RDCC): Founded in May 2021, the Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes so that critical innovation can continue and positive changes can be enacted for the rare disease community. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com