The European Regulation on Orphan Medicinal Products

Society cannot accept that certain individuals be denied the benefits of medical progress simply because the affliction from which they suffer affects only a small number of people. Therefore, public authorities need to provide the necessary incentives and to adapt their administrative procedures so as to make it easier to provide these patients with medicinal products which are just as safe, effective and meet the same quality standards as other medicinal products. In 1983, the US introduced the Orphan Drug Act as an incentive system for the development of these less required drugs. At the end of 1997, 152 orphan drug products had obtained marketing approval and the products are being used by over 7 million patients. The success of the US programme has stimulated many other countries into emulating the orphan drug programme. In the European Union in the 1990s, a number of member states adopted specific measures to increase our knowledge of rare diseases.
The Commission has proposed a Decision of the European Parliament and Council adopting a programme of community action 1999-2003 on rare diseases, including actions to provide information, to deal with clusters of rare diseases in a population and to support relevant patient organizations.