Orphan drug
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The term orphan drug refers to a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease. The assignment of orphan status to a disease and to any drugs developed to treat it is a matter of public policy in many countries, and has resulted in medical breakthroughs that would not have otherwise been achieved due to the economics of drug research and development.
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[edit] Orphan drug law
The Orphan Drug Act (ODA) of January 1983, passed in the United States with lobbying from the National Organization for Rare Disorders,[1] is meant to encourage pharmaceutical companies to develop drugs for diseases that have a small market: under the law, companies that develop such a drug (a drug for a disorder affecting fewer than 200,000 people in the United States) may sell it without competition for seven years,[2] and may get clinical trial tax incentives.[2] The European Union (EU) has enacted similar legislation, in which pharmaceuticals developed to treat rare diseases are referred to as "orphan medicinal products." The EU's definition of an orphan condition is broader than that of the USA, in that it also covers some tropical diseases that are primarily found in developing nations.[3] The EU's legislation is administered by the Committee on Orphan Medicinal Products of the European Medicines Agency (EMEA). In an effort to reduce the burden on manufacturers applying for orphan drug status, the FDA and EMEA agreed in late 2007 to utilize a common application process for both agencies. However, the two agencies will continue to maintain separate approval processes.[4] Orphan drug legislation also exists in Australia and Japan.[1]
Under the ODA and EU legislation, many orphan drugs have been developed, including drugs to treat glioma, multiple myeloma, cystic fibrosis, phenylketonuria, snake venom and idiopathic thrombocytopenic purpura. In the USA, from January 1983 to June 2004, a total of 1,129 different orphan drug designations have been granted by the Office of Orphan Products Development (OOPD) and 249 orphan drugs have received marketing authorization. In contrast, the decade prior to 1983 saw fewer than ten such products come to market. Nevertheless, some critics have questioned whether orphan drug legislation was the real cause of this increase (claiming that many of the new drugs were for disorders that were already being researched anyway, and would have had drugs developed regardless of the legislation), and whether the ODA has really stimulated the production of truly non-profitable drugs; the act also received some criticism for allowing some pharmaceutical companies to make a large profit off of drugs that have a small market but still sell for a high price.[2]
In 2003, the leading orphan drug by worldwide sales revenue was Amgen’s Erythropoietin (Epogen), with sales of $2.4bn.[citation needed]
[edit] Orphan drug development
Orphan drugs generally follow the same regulatory development path as any other pharmaceutical product, in which testing focuses on pharmacokinetics and pharmacodynamics, dosing, stability, safety and efficacy. However, some statistical burdens are lessened in an effort to maintain development momentum. For example, orphan drug regulations generally acknowledge the fact that it may not be possible to test 1,000 patients in a phase III clinical trial, as fewer than that number may be afflicted with the disease in question.
Since the market for any drug with such a limited application scope would, by definition, be small and thus largely unprofitable, government intervention is often required to motivate a manufacturer to address the need for an orphan drug. Critics of free market enterprise often cite this as a failure of free market economic systems. Free market advocates often respond that without government-mandated minimum safety and efficacy requirements, drug development costs would be considerably lower.
The intervention by government on behalf of orphan drug development can take a variety of forms:
- Tax incentives.
- Enhanced patent protection and marketing rights.
- Clinical research financial subsidization.
- Creating a government-run enterprise to engage in research and development (see Crown corporation).
[edit] See also
- Pharmaceutical company
- Supplementary protection certificate
- Orphan disease
- European Organization for Rare Diseases
[edit] References
- ^ a b Henkel, John (1999). "Orphan Drug Law Matures into Medical Mainstay". FDA Consumer. U.S. Food and Drug Administration. http://www.fda.gov/Fdac/features/1999/399_orph.html. Retrieved on 14 February 2009.
- ^ a b c Pollack, Andrew (30 April 1990). "Orphan Drug Law Spurs Debate". The New York Times. http://query.nytimes.com/gst/fullpage.html?sec=health&res=9C0CE4DF1F3EF933A05757C0A966958260. Retrieved on 15 February 2009.
- ^ http://www.medterms.com/script/main/art.asp?articlekey=11418
- ^ Donna Young (2007-11-28). "U.S., EU Will Use Same Orphan Drug Application.". BioWorld News (Washington). Archived from the original on 2007-12-11. http://www.pharmasentry.com/news/newsletter.cfm?linkid=CE077395-1372-54C2-61CFB42D06A278FB. Retrieved on 2008-01-06. "In an attempt to simplify the process for obtaining orphan status for medications targeting rare diseases, the FDA and the European Medicines Agency (EMEA) have created a common application. ... U.S. and European regulators still will conduct independent reviews of application submissions to ensure the data submitted meet the legal and scientific requirements of their respective jurisdictions, the agencies said."
