Orphan medicines are for diagnosing, preventing or treating life-threatening or very serious conditions that are rare and affect not more that 5 in 10 000 persons in the European Union. The legal background to the procedure in EU is set in the Regulation (EC) No 141/200 (the Orphan Regulation). Orphan designation may be obtained at any stage of development before an application for marketing authorization is made, provided proper scientific justification of the intended use is submitted. The designation procedure is free of charge. Designation as an orphan medicinal product does not indicate that the product has already satisfied the efficacy, safety and quality criteria necessary for the granting of a marketing authorization. As for any medicinal product, these criteria can only be assessed once the application for marketing authorization has been submitted.
The legislation provides incentives for pharmaceutical industry and sponsors to develop orphan medicines. The requirement for being eligible for the incentives to get the orphan drug designation is laid down in the Orphan Regulation and further in Regulation (EC) No 847/2000 and European Commission Communication 2003/C 178/02. These incentives are; market exclusivity for 10 years, protocol assistance in the form of scientific advice and guidance in preparing the dossier for marketing authorisation. In addition there are fee reductions with regards to the centralised activities.
The focus of rare diseases research in the Seventh Framework Programme (FP7; 2007-2013) is on Europe-wide studies of natural history, pathophysiology and on the development of preventive, diagnostic and therapeutic interventions.
By identifying and mobilising the critical mass of expertise the programme will: