FDA Oops: Two Orphan Drugs Approved for Same Disease or Condition
An orphan drug treats a rare disease or condition that occurs so infrequently in the United States that there is no reasonable expectation that the cost of making the drug will ever be recovered by the manufacturer. The Orphan Drug Act of 1983 incentivizes pharmaceutical manufacturers to investigate and develop drugs for rare diseases with a low probability of profitability. Orphan drugs have been approved and used to treat various cancers, Huntington’s disease, Fragile X syndrome, pulmonary fibrosis, myelomas, carcinomas, and other rare and unfortunate ailments that impact people’s lives. According to the National Organization for Rare Disorders, the number of approved drugs for treating rare diseases soared from 38 drugs before the act to 6,583 orphan-drug designations by the Food & Drug Administration (FDA) today. Undoubtedly, the Orphan Drug Act has had a positive impact on both patients suffering from rare conditions and the manufacturers that utilized the law.