Posted by Michael Wonder on 06 Jul 2024
Four decades of orphan drugs and priorities for the future
6 July 2024 - The Orphan Drug Act was enacted in the United States in 1983 in response to growing awareness of rare diseases, coupled with concerns about tepid interest in developing therapies that were unlikely to generate large profits.
By many measures, the Orphan Drug Act has been a resounding success, with more than 800 orphan indications approved and nearly 7,000 orphan drug designations granted in the past four decades. Yet many life-threatening rare diseases still lack treatments. Furthermore, some manufacturers have used the law in ways that diverge from its original intent.
