Orphan Drug Act of 1983
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| Long title | An Act to amend the Federal Food, Drug, and Cosmetic Act to facilitate the development of drugs for rare diseases and conditions, and for other purposes. |
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| Enacted by | the 97th United States Congress |
| Effective | January 4, 1983 |
| Citations | |
| Public law | 97-414 |
| Statutes at Large | 96 Stat. 2049 |
| Codification | |
| Acts amended | Federal Food, Drug, and Cosmetic Act |
| Titles amended | 21 U.S.C.: Food and Drugs |
| U.S.C. sections amended | 21 U.S.C. ch. 9 §§ 301 et seq., 360aa |
| Legislative history | |
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The Orphan Drug Act of 1983 is a law passed in the United States to facilitate development of orphan drugs — drugs for rare diseases such as Huntington's Disease, myoclonus, ALS, Tourette syndrome and muscular dystrophy which affect small numbers of individuals residing in the United States.
Orphan drug designation does not indicate that the therapeutic is either safe and effective or legal to manufacture and market in the United States. That process is handled through other offices in the US Food and Drug Administration. Instead, the designation means only that the sponsor qualifies for certain benefits from the federal government, such as reduced taxes.
In 1982 an informal coalition of supporters and families of patients with rare diseases who formed National Organization for Rare Disorders (NORD) and others, called for change to legislation to support development of orphan drugs, or drugs for treating rare diseases. They succeeded in getting the United States Congress to pass the Orphan Drug Act (ODA) in early 1983. Only thirty-eight orphan drugs had been approved prior to the 1983 Act; by 2014 "468 indication designations covering 373 drugs have been approved." Partly as a result of the 1983 US Orphan Drug Act, Japan adopted it in 1993 as did the European Union in 2000.
In response to incidents such as difficulties with thalidomide the Kefauver-Harris Amendment was passed. Kefauver-Harris was passed in order to require that all drugs approved for sale be proven safe and effective via rigorous scientific studies. While this legislation improved drug safety, it also dramatically increased the costs associated with developing new medicines. Pharmaceutical companies responded by focusing on developing treatments for common diseases in order to maximize the possibility of recouping research and development costs and generating significant profits. As a result, rare diseases were largely ignored due to poor economic potential and were thus said to be "orphaned." The gap between drugs for common versus rare diseases eventually widened to the point where few or no treatments were available for some rare conditions such as Crohn's disease, Hansen's disease, etc.
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