The ORPHAN Cures Act, a bipartisan bill that removes barriers to treating and curing rare diseases, was reintroduced in the House on Tuesday.
What it does: The ORPHAN Cures Act is bipartisan legislation that would amend the Inflation Reduction Act’s (IRA) orphan drug exclusion and incentivize critical follow-on investment into rare disease drug development.
Why it matters: Despite notable advances, today more than 6,000 rare diseases are still without a proven treatment option. Unfortunately, provisions in the IRA 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. The ORPHAN Cures Act would fix this.
Who’s behind it: The ORPHAN Cures Act was reintroduced in the 119th Congress by Reps. John Joyce, M.D., (R-PA) and Don Davis (D-NC), with support from 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), demonstrating strong bipartisan support for the policy.
What they’re saying: “We need to be doing more—not less—to bring new FDA-approved treatments to market for rare disease patients,” according to Rep. Joyce. "We must empower our innovators to continue developing lifesaving rare disease treatments,” said Rep. Davis.
BIO’s view: “BIO strongly supports the ORPHAN Cures Act,”
says BIO President & CEO John F. Crowley. “I urge Congress to stand with the millions of patients and families impacted by rare disease by supporting this bipartisan legislation.”
