ORPHAN Cures Act Enables Rare Disease Companies to Pursue Promising Research to Uncover More Treatment Options

The Rare Disease Company Coalition (RDCC) strongly supports the ORPHAN Cures Act introduced by Senators Barrasso (R-WY) and Carper (D-DE) and Representatives Joyce (R-PA-13) and Nickel (D-NC-13). The bipartisan and bicameral ORPHAN Cures Act works to encourage rare disease research and development (R&D) by addressing a critical disincentive in the Inflation Reduction Act (IRA) that impedes 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. Rare disease companies rely on this exemption as a critical incentive for R&D, but in limiting it to drugs that treat only one condition the IRA discourages rare disease companies from further exploring promising research that could lead to additional treatment options. The ORPHAN Cures Act would instead expand the Orphan Drug Exclusion to allow for the research and development of existing products that could help find treatments for the more than 95 percent of rare diseases without approved therapies.

For RDCC member companies that invest over half of their annual revenues back into research and development, improving the Orphan Drug Exclusion will have a significant impact in delivering desperately needed therapies and hope to underserved patient and caregiver communities.

Real-life impact: 1 in 5 orphan drugs are Food and Drug Administration (FDA)-approved for more than one use, and 60% of those second indications are for another rare disease. By limiting the Orphan Drug Exclusion to medicines that treat only one indication, the IRA discourages companies from pursuing development of promising therapies.

The unique needs of rare disease: The current IRA Orphan Drug Exclusion policy does not reflect the unmet need for treatments for rare diseases and the unique demands of rare disease drug development. The ORPHAN Cures Act ensures developers can continue to direct valuable resources towards rare disease research, building on the 40 years of progress enabled by the Orphan Drug Act (ODA).

The ORPHAN Cures Act strengthens the intent of the ODA: Since the enactment of the ODA in 1983, the number of orphan drugs approved by the FDA has increased from just 38 to more than 600 treatments for more than 1,000 rare diseases. While this has made a dent on the 95% of the 10,000 rare diseases that exist, continued progress requires both strengthening ODA policies and continuing to advance recognition of and support for the unique needs of rare disease R&D. The ORPHAN Cures Act achieves this by removing a disincentive for developing rare disease treatments.

The final word: The ORPHAN Cures Act addresses the concerning effects of the IRA’s Orphan Drug Exclusion and allows biopharmaceutical companies to meet the clear and urgent need for continued innovation in rare disease R&D. The RDCC thanks Senators Barrasso and Carper and Representatives Joyce and Nickel for supporting the rare disease community through this important legislation.