Orphan Drug Act of 1983

Orphan Drug Act of 1983
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
	Introduced in the House as H.R. 5238 by Henry Waxman (D-CA) on December 15, 1981; Committee consideration by House Energy and Commerce, House Ways and Means, Senate Labor and Human Resources; Passed the House on September 28, 1982 (passed voice vote); Passed the Senate on October 1, 1982 (passed voice vote) with amendment; House agreed to Senate amendment on December 14, 1982 (agreed voice vote) with further amendment; Senate agreed to House amendment on December 17, 1982 (agreed voice vote); Signed into law by President Ronald Reagan on January 4, 1983;

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.

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. 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.