Washington, D.C. — In response to passage of the Senate’s reconciliation bill, which included the Optimizing Research Progress Hope And New Cures (ORPHAN Cures) Act, a vital provision that would ensure continued innovation and progress in the development of rare disease treatments, Stacey Frisk, Executive Director of the Rare Disease Company Coalition (RDCC), released the following statement:
“We are grateful the Senate included in its reconciliation package the ORPHAN Cures Act, an essential policy that would provide hope for the 1 in 10 Americans living with a rare disease. This provision will spur tremendous innovation and progress for rare disease research by addressing a flaw in the IRA that offers a price negotiation exemption for orphan drugs that treat only one rare condition, hindering further research of additional rare disease applications.
“The RDCC particularly commends Senator Barasso, a longtime champion for the rare disease community, for his key role in leading the ORPHAN Cures Act. We are also grateful to Majority Leader John Thune and Chairman Mike Crapo for prioritizing policies that pave the way for innovation that gives hope to individuals living with rare disease and their families.
“Since the IRA’s passage less than three years ago, studies show we are already seeing fewer investments towards expanded uses of FDA-approved drugs to treat rare diseases, with second orphan designations down 48 percent. If action isn’t taken, we can expect a reduction in research and development spending by $663 billion, resulting in 135 fewer new drugs being approved through 2039.
“With 95% of rare diseases lacking an FDA-approved treatment, it’s imperative that members of Congress work together to ensure that Americans living with rare diseases are not left behind. We urge the Senate and House to work together to ensure the ORPHAN Cures Act remains in the reconciliation package and deliver a final bill that safeguards innovation and progress in rare disease research and treatment.”
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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.