Washington, D.C. — The Rare Disease Company Coalition (RDCC) strongly supports the ORPHAN Cures Act (H.R.946) introduced by Representatives John Joyce (R-PA-13) and Don Davis (D-NC-01). The bipartisan legislation will help to encourage critical rare disease research and development (R&D) by addressing a disincentive within the Inflation Reduction Act (IRA) that has hindered rare disease innovation and investment.
Currently, the IRA’s Orphan Drug Exclusion offers a price negotiation exemption for orphan drugs that treat only one orphan condition, thereby discouraging rare disease companies from further exploring promising research that could lead to additional treatment options. In practice, however, 1 in 5 orphan drugs are FDA-approved for more than one use, and 60 percent of those second indications are for another rare disease.
The current limits imposed by the IRA have created a roadblock to rare disease innovation, and in turn, progress towards developing treatments for the more than 95 percent of rare diseases without approved therapies. The ORPHAN Cures Act would open the door for the research and development of existing products that could help find treatments by expanding the single-orphan exclusion.
“The ORPHAN Cures Act is common sense legislation that will remove unnecessary hurdles towards developing treatments for individuals with rare diseases. RDCC member companies understand the importance of investing back into research and development for rare diseases therapies, but in order to maximize the pursuit of promising research we must expand the IRA’s single-orphan exclusion,” said Stacey Frisk, Executive Director of the RDCC. “We thank Representatives Joyce and Davis for understanding the importance of delivering these desperately needed therapies to underserved patient and caregiver communities by introducing this legislation.”
Rare disease drug development is uniquely challenging, and the current policy does not reflect the unmet need for rare disease treatments. By ensuring developers can continue to direct much needed resources towards rare disease research, the ORPHAN Cures Act will build upon the 40 years of progress enabled by the Orphan Drug Act (ODA).
One in ten people live with a rare disease, and the ORPHAN Cures Act provides an opportunity to address the urgent need for innovation in rare disease R&D by breaking down barriers that are restricting biopharmaceutical companies from meeting the moment. The RDCC thanks Representatives John Joyce and Don Davis for leading the charge and Representatives Kevin Hern (R-OK-1), Scott H. Peters (D-CA-50), Mariannette Miller-Meeks (R-IA-1), William R. Keating (D-MA-9), Richard Hudson (R-NC-9), Shri Thanedar (D-MI-13), Gus M. Bilirakis (R-FL-12) and Josh Gottheimer (D-NJ-5) for co-sponsoring this legislation and supporting the rare disease community.
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