Definition of Orphan Drug

orphan drugOrphan Drug is a drug to treat a rare condition.

The US Orphan Drug Act of 1983 (ODA) established criteria in the United States to encourage pharmaceutical manufacturers to investigate new drugs and continue to produce approved drugs to treat conditions, such as Huntington’s disease and some forms of muscular dystrophy, that affect fewer than 200,000 people.

The underlying premise of an orphan drug is that its sales will not generate enough revenue for its manufacturer to recover the costs of its development and testing, a circumstance that makes research and production unappealing to pharmaceutical manufacturers.

The ODA establishes mechanisms of financial support for pharmaceutical manufacturers through grants and tax relief, in return for which the manufacturer agrees to produce and market the drug.

Additional grants are available to support research about rare diseases. In 2005 there were approximately 1,400 drugs with orphan drug status. The US Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) oversees orphan drug research.


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