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Effects of Initial Orphan Drug Act Designation on 5-Year Sales of New Prescription Drugs

Newly approved prescription drugs with orphan drug designations were as lucrative over the first 5 years as agents approved for more common conditions, according to an analysis of the SSR Health database. “Among 315 drugs, 83 (26%) were initially indicated for orphan-designated conditions,” the authors report. “Median 5-year net sales were $719 million (IQR, $298 million to $1.8 billion) for orphan-designated drugs and $812 million (IQR, $228 million to $2.4 billion) for nonorphan drugs (adjusted mean ratio, 0.6; 95% CI, 0.3-1.1; P = .09).”

A sizable fraction of orphan drugs received later approvals for more common conditions within 5 years, the investigators add: “Among orphan-designated drugs, 67 (81%) had a single indication and 16 (19%) had multiple indications within 5 years, including 6 (7%) with a secondary nonorphan indication. Compared with nonorphan drugs, revenue was not significantly different for orphan-designated drugs with 1 indication (median, $717 million [IQR, $284 million to $1.7 billion]; adjusted mean ratio, 0.6 [95% CI, 0.3-1.1]; P = .12) or multiple indications (median, $1.2 billion [IQR, $471 million to $2.4 billion]; adjusted mean ratio, 0.5 [95% CI, 0.1-1.5]; P = .20).”

Source: JAMA