Rare Disease Company Coalition (RDCC) Executive Director Stacey Frisk released the following statement:
Washington, D.C. — “The Rare Disease Company Coalition is pleased to celebrate the passage of the Mikaela Naylon Give Kids a Chance Act, which reauthorizes the lifesaving Pediatric Rare Disease Priority Review Voucher (PRV) program. The pediatric PRV program supports the development of new treatments and cures for the millions of American children living with rare diseases.
“This milestone brings renewed hope to families awaiting lifesaving treatments for children with rare diseases and reaffirms a bipartisan commitment to advancing innovation for pediatric patients who urgently need therapeutic breakthroughs. The PRV program has already helped enable therapies across 47 rare pediatric indications.
“The RDCC is deeply grateful to the policymakers, advocates, and innovators who worked to restore this essential program.”
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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.