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.
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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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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New White Paper Examines the Impact of Policy Uncertainty on Investment in Rare Disease Drug Development
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RDCC Applauds Reintroduction of Cameron’s Law and the Rare Disease Clinical Trial Pandemic Disruption Act
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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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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.
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.
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.
“As Co-Chair of the Rare Disease Caucus, I’ve proudly fought to accelerate the development of and improve access to treatments for the rare disease community. This is largely possible due to the enactment of the Orphan Drug Act in 1983. On the 40th anniversary of this transformational law, I renew my commitment to continue the mission of incentivizing the speedy approval of new therapies and cures for millions of rare disease patients across the nation.”
U.S. Representative Gus Bilirakis (R-FL)
“Limiting the Orphan Drug Tax Credit to the first approval would fundamentally alter the rare disease pipeline and discourage companies from pursuing further clinical programs to test approved therapies in new disease areas. Investment in further research improves the lives of the most vulnerable rare disease patients and offers clinicians and insurers the evidence needed to guide treatment recommendations that wouldn’t otherwise be available.”
The EveryLife Foundation
“The Orphan Drug Act created a framework whereby industry can focus on long-term investments in R&D, but there is much work to be done. Policymakers must understand that rare disease patients and families have unique needs. Working together for the unified purpose of delivering treatments for rare disease patients is a public health imperative.”
Giacomo Chiesi, Head of Chiesi Global Rare Diseases
“Time is precious for patients and families affected by rare diseases. Fulcrum is proud to have launched the first Phase 3 clinical trial for facioscapulohumeral muscular dystrophy (FSHD) – a devastating, progressive neuromuscular disease for which there are no approved treatments – less than three years after becoming a public company. The incentives provided by the Orphan Drug Tax Credit are essential for enabling young companies like ours to rapidly research and develop promising new therapies for the communities that are waiting.”
Santiago Arroyo, M.D., Ph.D. Chief Medical Officer, Fulcrum Therapeutics
“We believe the speed and agility with which we were able to bring these transformative medicines to patients who are living with some of the world’s rarest diseases, such as primary hyperoxaluria type 1 and acute hepatic porphyria, would not have been possible without the provisions of the Orphan Drug Act.”
Pushkal Garg, MD, Chief Medical Officer and EVP, Development & Medical Affairs, Alnylam Pharmaceuticals
“When you look beyond rare diseases that are currently being treated or have the potential for new therapies, you see millions of people who now have hope thanks to the ODA. While the disease may be rare, the people are not, and without the ODA, the incentive to research and develop new therapies for patients in need would simply not be there.”
Nevan Charles Elam, JD, Chief Executive Officer & Founder, Rezolute Bio
“The Orphan Drug Act (ODA) makes it possible for us to invest in the research and development necessary to address the unmet needs of rare disease patients. Since the passage of the ODA, all of BioMarin’s FDA-approved therapies have received orphan designation. As such, BioMarin has been able to transform the lives of children across the globe who have rare diseases such as CLN2 (a form of Batten disease) and enzyme deficiency diseases, among others.” By protecting the ODA, we are protecting the future of innovation and sustaining hope for children and adults alike with rare diseases.“
Jean-Jacques Bienaime, Chairman and Chief Executive Officer, BioMarin
“The incentive provided by the Orphan Drug Tax Credit allowed Ultragenyx to study Crysvita® for tumor-induced osteomalacia (TIO), an ultra-rare disease that most companies would never have studied, after Ultragenyx previously obtained initial FDA approval of Crysvita® for the treatment of X-linked hypophosphatemia (XLH). We support the continued existence of the Orphan Drug Tax Credit as a critical incentive for rare disease companies.”
Camille Bedrosian, Chief Medical Officer, Ultragenyx
“The Orphan Drug Act spurred tremendous progress in the development of rare diseases treatments not only in the United States, but worldwide. The law has served as a catalyst for rare disease innovation and regulatory approval pathways in many other countries. Now is the time to build on that progress and promote sound policies that will improve patients’ lives.”
Del Lebel, Head, U.S. Government Affairs and Policy, Alexion, AstraZeneca Rare Disease
“We’re incredibly tired of the erosion of the ODA. We’re grateful for the progress that’s come since the passage of that landmark legislation, but 40 years later, I think it’s really time that we start to think about how we expand on that great work.”
Annie Kennedy, chief of policy, advocacy & patient engagement, EveryLife Foundation for Rare Diseases
“NORD is strongly advocating for the tax credit to be increased to 50%, and we have fought ferociously against subsequent efforts to further reduce the credit.”
Heidi Ross, VP for policy and regulatory affairs, National Organization for Rare Diseases
“It’s likely that there will be reduction in innovation in rare disease because follow-on indications are now potentially penalized under the IRA. It’s also the case more generally there will be reductions in incentives to innovate. It’s notable that rare disease often features very high unmet need for patients. As an economist, I can tell you that means that the value of any given health improvement is greater, because patients have so little health that even a given relatively modest improvement of health can be quite valuable.”
Dr. Darius Lakdawalla, Professor of Pharmaceutical Economics and Public Policy, USC Leonard D. Schaeffer Center for Health Policy & Economics
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 firstname.lastname@example.org.
If you are a member of the media interested in learning more about the Rare Disease Company Coalition, please contact us at email@example.com.