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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. |
|---|---|
| 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 created | 21 U.S.C. ch. 9, subch. V §§ 360aa-360ee |
| U.S.C. sections amended | 21 U.S.C. ch. 9 § 301 et seq. |
| Legislative history | |
| |
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 or muscular dystrophy which affect small numbers of individuals residing in the United States.[1]
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 market exclusivity and 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.[2] They succeeded in getting the United States Congress to pass the Orphan Drug Act (ODA) in early 1983.[2][3][4] Only thirty-eight orphan drugs had been approved prior to the 1983 Act; by 2014 "468 indication designations covering 373 drugs have been approved."[5] Partly as a result of the 1983 US Orphan Drug Act, Japan adopted it in 1993 as did the European Union in 2000.[5]
- ^ "Orphan Drug Act of 1983" (PDF). US Food and Drug Administration. 4 January 1983. Retrieved 27 October 2015.
- ^ a b Parisse-Brassens, Jerome (June 2007). "Abbey Meyers, President of NORD, announces her retirement (July 07)". European Organization for Rare Disorders. Archived from the original on 2007-10-09. Retrieved 2024-04-10.
- ^ "Millions Around World to Observe Rare Disease Day". PR Newswire. 13 February 2009. Retrieved 14 February 2009.[permanent dead link]
- ^ Henkel, John (1999). Orphan Drug Law Matures into Medical Mainstay. U.S. Food and Drug Administration. ISBN 978-1-4223-2677-0. Retrieved 14 February 2009.
{{cite book}}:|work=ignored (help) - ^ a b Hadjivasiliou, Andreas (October 2014), "Orphan Drug Report 2014" (PDF), EvaluatePharma, retrieved 28 June 2015