US Orphan Drug Act

In 1983 the US Congress enacted the Orphan Drug Act, establishing financial incentives for companies to develop new drugs and biologics for rare diseases, including a partial tax credit for clinical trial expenditures, waived user fees, and eligibility for seven years of marketing exclusivity.1 Administered by the Office of Orphan Products Development at the Food and Drug Administration, the act enables companies developing therapies for clinical indications affecting fewer than 200 000 people in the US to request an orphan designation.1 The legislation wholly changed drug development for rare diseases: before 1983 the FDA had approved only 10 drugs that would have qualified for an orphan designation.2 By 2002 the number of products with orphan designations reached nearly 1100, of which 232 were eventually approved.3However, concerns began to arise as increasingly narrow disease definitions, enabled by precision medicine and advances in genetics, were used to qualify increasing numbers of drugs…
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