Dr. Joyce Introduces Bipartisan Legislation to Encourage Life-Saving Innovation
Washington, D.C. –Congressman John Joyce, M.D. (PA-13) and Congressman Don Davis (NC-01) have 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 Americans affected by rare diseases.
Under current federal law, a drug or treatment that receives approval from the U.S. Food and Drug Administration (FDA) to treat exclusively 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. This has the unintended effect of discouraging and disincentivizing American innovators from engaging in the expensive and time-intensive research necessary to determine if an orphan drug could cure or treat additional rare diseases.
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.
“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.”
“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.”
“Life Sciences Pennsylvania applauds Congressman Joyce on the introduction of the ORPHAN CURES Act in the 119th Congress,” said Christopher P. Molineaux, President and CEO of Life Sciences Pennsylvania. “Dr. Joyce understands that the process of taking a rare disease medicine from research through development and approval for patients has many unique challenges. With small patient populations, the development of medicines for rare diseases is significantly more difficult, costly, and risky than typical drug research and development. The ORPHAN CURES Act creates hope for the millions of patients living with a rare disease.”
"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 results 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," said Maimah Karmo, President & CEO, Tigerlily Foundation
"On behalf of our LGMD2I/R9 community, CureLGMD2i fully supports the Orphan Cures Act (OCA), which will maintain existing incentives and boost research into new treatments for the 30 million Americans currently suffering from rare diseases. LGMD2I/R9 is an ultra rare and progressive muscle wasting disease that currently has no approved treatment. The OCA provides hope to our patient community by protecting the incentives for drug developers to continue working on a potential treatment for rare diseases like the LGMDs," saidKelly Brazzo, Co-Founder and CEO of CureLGMD2i Foundation
“Rare disease medications are often brought to market as a second indication, because research is just too expensive for our small populations to do the initial expansive testing. Eliminating limiting the exemption to one rare disease, hope and treatment is unnecessarily taken away. This, and the removal of incentives for investing in rare disease research are devastating to the rare disease community. While we believe these were oversights in crafting the law, they must be corrected immediately. People's lives are at stake. Eosinophilic & Rare Disease Cooperative strongly support the passage of the Orphan Cures Act. We are here to help in any way we can to move this legislation forward,” said Sarah Jones, Community Engagement, Eosinophilic & Rare Disease Cooperative
“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 disease,” said the Save Rare Treatments Task Force
For more information, you can find a one-pager here.
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