STAT+: The latest orphan drug exemptions may not be warranted, analysis finds

Ed’s stories explore prescription drug pricing, affordability and access, as well issues surrounding patents, litigation, and legislation. He is also the author of the morning Pharmalittle newsletter and the afternoon Pharmalot newsletter.A controversial provision in the One Big Beautiful Bill Act, which exempts more so-called orphan drugs from Medicare pricing negotiations in order to encourage further development, may not warrant that protection, according to a new analysis. At issue is a portion of the Inflation Reduction Act that went into effect three years ago and allows Medicare to negotiate prices for certain drugs. Under that law, orphan drugs, which are used to treat rare diseases, were exempt in the case of drugs granted just one orphan designation and approved by the Food and Drug Administration. However, the Orphan Cures Act, which was part of the Big Beautiful Bill Act that became law last year, broadened the exception by allowing medicines with multiple orphan indications to remain exempt. The law also delayed the timelines for which an otherwise eligible orphan drug may be considered for price negotiations. STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus in-depth analysis, newsletters, premium events, and news alerts. Already have an account? Log in View All Plans To read the rest of this story subscribe to STAT+. Subscribe