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Data Brief: How Often Are High-Expenditure Orphan Drugs Being Used for Their Orphan Indications in Medicare

The Orphan Drug Act was enacted in 1983 to provide financial incentives and marketing exclusivity for manufacturers to develop drugs for rare diseases that occur so infrequently that there would be no reasonable expectation to generate a profit. In recent years, an increasing number of orphan designations have been granted for existing drugs that already have high sales and utilization for non-orphan indications. For example, 7 of the top 10 drugs in 2017 global sales have been granted orphan designations, with all but one having Food and Drug Administration approval for non-orphan indications as well. OIG will determine (1) how many high-expenditure Medicare drugs have orphan designations and (2) the extent to which these drugs are utilized for their orphan designated indications in Medicare.

Announced or Revised Agency Title Component Report Number(s) Expected Issue Date (FY)
May 2020 Food & Drug Administration Data Brief: How Often Are High-Expenditure Orphan Drugs Being Used for Their Orphan Indications in Medicare Office of Evaluation and Inspections OEI-BL-20-00080 2021