Urges Congress to support rare disease patients & support the bill
WASHINGTON, D.C. (February 5, 2025) – John F. Crowley, President & CEO of the Biotechnology Innovation Organization (BIO), today released the following statement after introduction of the ORPHAN Cures Act (H.R. 946), a bipartisan bill that removes barriers to treating and curing rare disease:
“For more than 40 years, the Orphan Drug Act has provided hope to 30 million Americans living with a rare disease. This bipartisan law has helped spur hundreds of new treatments and transformed the standard of care for these patients and their families.
“Despite notable advances, today more than 6,000 rare diseases are still without a proven treatment option. Unfortunately, provisions in the Inflation Reduction Act disincentivize companies to test whether existing treatments for rare diseases also benefit other rare diseases, undermining decades of bipartisan agreement and biomedical progress for the thousands of individuals living with these diseases who are still waiting for any treatment or cure.
“That’s why BIO strongly supports the ORPHAN Cures Act, which would amend the IRA and ensure the continuation of essential research and development of new medicines for patients with rare diseases. I’d like to thank Representatives John Joyce, M.D., (R-PA) and Don Davis (D-NC) for their bipartisan support for this important legislation.
We would also like to thank Reps. Kevin Hern (R-OK), Scott Peters (D-CA), Mariannette Miller-Meeks, M.D. (R-IA), Bill Keating (D-MA), Richard Hudson (R-NC), Shri Thanedar (D-MI), Gus Bilirakis (R-FL), and Josh Gottheimer (D-NJ).
“I urge Congress to stand with the millions of patients and families impacted by rare disease by supporting this bipartisan legislation. Let’s pass this bill as soon as possible and clear the way for medical discoveries that patients can’t live without.”
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