Orphan medicines

Bosentan (Tracleer ) Actelion, Switzerland ETA/ETB receptor Grade III and IV PAH and chronic thromboembolic pulmonary hypertension (European approval in 2002 as orphan medicine) Program for CHF was terminated in phase III... 

TBC11251 (Sitaxsentan) Texas Biotechnology, USA ETA receptor Grade III PAH (designated orphan medicine in 2004)... 

Regulation (EC) No. 141/2000 ofthe European Parliament and of the Council ofl6 December 1999 on orphan medicinal products Commission Regulation (EC) No. 847/2000 of 27 April 2000 laying down the provisions for implementation of the criteria for designation of a medicinal product as an orphan medicinal product and definitions of the concepts similar medicinal product and clinical superiority ... 

A medicinal product shall be designated as an orphan medicinal product il its sponsor can establish ... 

Pre-authorization evaluation of medicines for human use unit. This unit provides scientific advice relating to quality, safety and efficacy issues, as well as relating to orphan drugs. 

Synold, T.W., Dussault, I. and Forman, B.M. (2001) The orphan nuclear receptor SXR coordinately regulates drug metabolism and efflux. Nature Medicine, 7, 584-590. 

Orphan medicines (medicines used for rare diseases)... 

In order to encourage pharmaceutical companies to invest in orphan drug development, legislation provides for a number of incentives. These include application fee waiver (the extent of reduction varies with the region of the world, and in the European Union it is 50% for all fees since 2002), market exclusivity and protocol assistance. In the European Union, there is 100% reduction in the fee applicable to the provision of any scientific advice. The fund made available by the Community for fee exemptions for orphan medicinal products amounts to= 3 700000 in 2005. 

The authors are most grateful to Dr Agnes Saint Raymond, Head of Sector Scientific Advice and Orphan Drugs and Acting Head of Sector Safety Efficacy, European Medicines Agency, London for her valuable and constructive comments during the preparation of this chapter. Any errors, however, are entirely the responsibility of the authors. 

EU Orphan Drug designation. European Medicines Agency (EMEA). INTERNET SITE, 2005 August 26. 

Milne, C., K. Kaitin, and E. Ronchi. 2004. Orphan Drug Laws in Europe and the US Incentives for the Research and Development of Medicines for Diseases of Poverty. WHO Commission on Macroeconomics and Health, CMH Working Paper Series, Paper No.WG2 9. Available at http //www.cmhealth.org/docs/wg2 paper9.pdf [Accessed May 10, 2005]. 

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