Bipartisan Bill Enables Rare Disease Companies to Pursue Promising Research, Development of Treatment Options
Washington, D.C. — The Rare Disease Company Coalition (RDCC) strongly supports the ORPHAN Cures Act, led by Senators Barrasso (R-WY) and Heinrich (D-NM) on May 22. The bipartisan legislation 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.
The IRA’s current Orphan Drug Exclusion provides a price negotiation exemption for orphan drugs indicated for a single rare condition. This framework has inadvertently created a disincentive for the further investigation of existing therapies for additional rare disease applications. Notably, a significant proportion of orphan drugs approved by the FDA have received authorization for more than one indication, with a substantial number of these subsequent approvals targeting other rare diseases.
The existing limitations within the IRA have presented an impediment to the progress of rare disease innovation, thereby affecting the development of treatments for the over 95 percent of rare diseases that currently have no approved therapies. The ORPHAN Cures Act aims to facilitate the exploration of existing products for potential new treatments by broadening the scope of the single-orphan exclusion.
“Rare disease drug development is uniquely challenging, and a one-size-fits-all approach to policy can stymie innovation for the 30 million Americans living with a rare disease,” said Stacey Frisk, Executive Director of the Rare Disease Company Coalition. “The ORPHAN Cures Act removes harmful barriers to innovation and opens the door for promising research, providing much-needed hope to the rare disease community. We thank Senators Barrasso and Heinrich for recognizing the needs of rare disease patients and championing this crucial bill.”
The development of therapies for rare diseases presents unique challenges, and current policy does not reflect the unmet need for rare disease treatments. By ensuring that developers can allocate necessary resources towards rare disease research, the ORPHAN Cures Act has the potential to build upon the established progress facilitated by the Orphan Drug Act (ODA) over the past four decades.
The potential benefits of the ORPHAN Cures Act for rare disease patients are significant. By fostering a more robust environment for research and development, the legislation aims to accelerate the discovery of new therapies and expand the availability of treatment options.
Approximately one in ten people are living with a rare disease, and the introduction of the ORPHAN Cures Act offers an opportunity to address the critical need for sustained innovation in rare disease R&D by mitigating unnecessary barriers that may restrict biopharmaceutical companies in their efforts to meet this demand.
The RDCC thanks both Senators Barrasso and Heinrich for leading the charge and introducing the ORPHAN Cures Act. We urge Congress to support the ORPHAN Cures Act by advocating for its continued inclusion in the reconciliation package.
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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