Innovation

“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