We need the ORPHAN Cures Act to incentivize the development of orphan drugs for rare diseases, says a letter from the Council of State Bioscience Associations (CSBA) to congressional leaders.
Why it matters: Roughly 7,000 rare diseases afflict 30 million Americans, says CSBA, the confederation of state-based affiliates of BIO. But 95% of rare diseases lack an approved treatment.
The newest challenge: The Inflation Reduction Act exempts orphan drugs for rare diseases from price controls—but only if they treat a single indication. However, of the 280+ orphan drugs approved since 2003, nearly a quarter were approved for additional indications.
The solution: The proposed ORPHAN Cures Act would amend the IRA, exempting rare disease treatments that address multiple indications from price controls. This would incentivize R&D for new treatments.
What the people want: 94% of American voters think it’s important for scientists to search for rare disease treatments, and 85% want the government to “expand incentives for rare disease drug development,” a recent poll conducted by Morning Consult for BIO finds.
BIO’s view: “If enacted, the ORPHAN Cures Act would support existing incentives and boost research into new treatments by incentivizing research in follow-on indications,” says the letter, signed by Michele Oshman, CSBA Executive Director and BIO VP for External Affairs.
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