Congressman Don Davis Introduces Bipartisan Legislation to Encourage Life-Saving Innovation

Congressman Don Davis (NC-01) and Congressman John Joyce, M.D. (PA-13) introduced H.R. 946, the Optimizing Research Progress Hope And New (ORPHAN) Cures Act, legislation that would accelerate the development of new life-saving cures and provide hope to millions of Americans affected by rare diseases.

“We must empower our innovators to continue developing lifesaving rare disease treatments,” said Congressman Don Davis. “By cutting red tape for researchers and scientists, Congress can help lay the foundation for the next generation of cures.”

Under current federal law, a drug or treatment that receives approval from the U.S. Food and Drug Administration (FDA) to exclusively treat one rare disease – commonly known as an “orphan drug” – is eligible for certain incentives, including an exemption from Medicare’s drug negotiation program.  Unfortunately, those same incentives do not exist if an orphan drug receives FDA approval to treat two or more rare diseases.  The result is a disincentive for American innovators to invest in the expensive and time-intensive research necessary to determine if an orphan drug could cure or treat additional rare diseases.

“Over 30 million Americans are affected by nearly 10,000 rare diseases– yet 95% of these rare diseases lack an FDA-approved treatment.  We need to be doing more – not less – to bring new FDA-approved treatments to market for rare disease patients,” said Congressman John Joyce, M.D. “The ORPHAN Cures Act ensures that proven, critical R&D incentives are in place so the millions of Americans with rare diseases can continue to have hope for the future.”

The ORPHAN Cures Act would remedy these harmful, unintended consequences by honoring the intent of the Orphan Drug Act of 1983 and restoring proven, time-tested incentives to encourage the discovery of new cures for the narrow patient populations affected by rare diseases.

“NCLifeSci, the trade association for the NC life sciences industry, is grateful to Congressman Don Davis for leading on legislation that will ensure companies can continue to innovate for patients currently living with rare diseases. This legislation would address unintended consequences related to the IRA’s harmful orphan drug exclusion and align incentives to enable follow-on investment into rare diseases. Patients living with one of more than 7000 currently identified rare diseases are relying on our companies and their ability to innovate to provide hope and cures, and much of this development is happening in our backyard right here in North Carolina,” Laura Gunter, President, North Carolina Life Sciences Organization.

"At Tigerlily Foundation, we stand firmly in support of the ORPHAN Cures Act, a vital step toward ensuring access to life-saving treatments for rare disease patients, including those needing care for rare diseases as a result of their anti-cancer treatments. This legislation not only accelerates the development of innovative therapies but also addresses the unique challenges faced by patients who have long been overlooked. We believe every individual deserves the right to hope, healing, and health, and the ORPHAN Cures Act brings us closer to that vision. Together, we can create a future where no one is left behind in the fight for better care and cures," saidMaimah Karmo, President & CEO, Tigerlily Foundation.

“With the growing role of genetics and genomics in cancer and other diseases, we are seeing more rare patient communities of under 200,000 who may benefit from a targeted treatment. Passage of the ORPHAN Cures Act is essential to encourage therapeutic innovation for these patients. Without it, the incentives established under the Orphan Drug Act are undermined, and some of our most vulnerable patients will suffer,” said Lisa Schlager, Vice President, Public Policy, FORCE: Facing Our Risk of Cancer Empowered. 

“The Save Rare Treatments Task Force thanks Congressmen Joyce and Davis for their bipartisan leadership in introducing the ORPHAN Cures Act. This vital legislation corrects an unintended consequence in law to ensure strong incentives for research and development of new medical treatments for rare diseases,” said the Save Rare Treatments Task Force.