Innovation

We believe clinical trials for rare disease treatments must account for the unique circumstances of developing a treatment for small patient population sizes. We are focused on advancing consensus around the modernization of clinical trials for rare disease treatments while maintaining support for expedited programs, including Accelerated Approval, Priority Review Voucher, Fast Track, Breakthrough Designation, and Regenerative Medicine Advanced Therapy Designation. In addition, we believe that research incentives established by the Orphan Drug Act (ODA), such as the Orphan Drug Tax Credit, must be preserved to continue to attract and maintain long-term investment in the development of advanced treatments and diagnostics for rare diseases.

In the News

Hopes, Fears for Orphan Drug Act
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Celebrating 40 Years of the Orphan Drug Act: Renewing a Commitment to Advancing Innovation for Rare Disease Patients
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The State of the Biotech Industry
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RARECast Podcast: Examining the Legislative Landscape for Rare Disease Drug Development
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Recognizing the 40th Anniversary of the Orphan Drug Act, The Rare Disease Company Coalition Calls on Policymakers to Renew Commitment to Advancing Innovation for Rare Disease Patients
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Recent Updates

Learn more about how the Orphan Drug Act enables researchers to discover breakthrough cures for rare diseases
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RDCC Joins in Call for Repeal of R&D Tax Policy that Hurts Rare Disease Innovation
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Rare Disease Drugmakers to Congress: Don’t Gut the Accelerated Approval Pathway
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Featured Policy Positions

Orphan Drug Tax Credit

The Orphan Drug Tax Credit (ODTC) is a vital lifeline for rare disease patients and must be protected to preserve hope for the rare disease community. Given that about 95% of rare diseases lack any FDA-approved treatment, proposals to reduce the ODTC would have a devastating impact on orphan drug development in the U.S.. To foster hope for millions of Americans living with rare disease, we must continue to support policies and incentives like the ODTC and preserve the gains that have been made since the passage of the ODA.

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image of RDCC Letter - EEOC Draft FY22-26 Strategic Plan for Public Comment

Research & Development

Research and development (R&D) is at the heart of rare disease innovation. Due to the complex nature of rare diseases, a comparatively higher percentage of operating expenses is dedicated to R&D. Considering that over 95% of rare diseases are without an FDA-approved treatment, policymakers should be compelled to protect incentives for R&D to ensure that companies can continue to innovate and advance ambitious research and development programs that  bring treatments to patients.

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Priority Review Voucher

FDA’s priority review vouchers (PRV) provide a critical and cost-effective incentive to spur investment in high-risk development. By building upon this model and making PRVs permanent for use in rare disease, Congress can further offset the high cost and risk of rare disease drug development

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What They Said

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The Rare Disease Company Coalition helps educate key policy stakeholders on the unique considerations of life science companies when developing and bringing to market therapies for small and differentiated patient populations. If you are a life science company dedicated to rare diseases and interested in joining us, please contact info@rarecoalition.com.

If you are a member of the media interested in learning more about the Rare Disease Company Coalition, please contact us at media@rarecoalition.com.

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