House Vote Marks Critical Step Toward Reauthorizing Lifesaving Incentive Program for Rare Pediatric Treatments
Washington, D.C. — The Rare Disease Company Coalition (RDCC) commends the U.S. House of Representatives for passing the Mikaela Naylon Give Kids a Chance Act (H.R. 1262), a pivotal bill that would reauthorize the rare pediatric disease Priority Review Voucher (PRV) program. This bipartisan vote brings the nation one step closer to restoring a vital initiative that helps drive urgently needed treatments for children living with rare, life-threatening diseases.
The PRV program, which lapsed in December 2024, has been instrumental in catalyzing innovation for patients who too often have no therapeutic options. Since its inception, the program has supported treatments across 47 rare pediatric disease indications and benefited more than 200,000 children. With 30 percent of U.S. children with a rare disease not expected to reach their fifth birthday, reinstating this program is essential to sustaining research and investment in therapies that can save young lives.
RDCC is now calling on the U.S. Senate to take up the bill without delay.
“Today’s House passage of the Mikaela Naylon Give Kids a Chance Act is a massive step forward for the rare disease community,” said Stacey Frisk, Executive Director of the Rare Disease Company Coalition. “We applaud every member who voted for this bill and thank the sponsors for their dedication to advancing solutions for children who cannot wait. With House approval secured, we are urging the Senate to prioritize this legislation and ensure the PRV program is reinstated as quickly as possible. Children living with rare, devastating diseases need action, not uncertainty.”
Reauthorizing the PRV program will safeguard continued momentum in rare pediatric drug development and reaffirm the nation’s commitment to the hundreds of thousands of families affected by rare childhood illnesses.
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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.