Abstract:A245 Objectives: The market for orphan drugs presents challenges in balancing incentives for innovation with accessibility for patients. We assessed availability and prices of United States (US) Food and Drug Administration (FDA) orphan-designated drugs approved for treatment of leukemia in the US and England. MethOds: Regulatory information for leukemia drugs with FDA orphan designation was collected from the FDA, the European Medicines Agency, and the Medicines and Healthcare Products Regulatory Agency. Gene… Show more
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