The development of drugs for the treatment of rare diseases is usually a very costly procedure and given the small number of potential patients, the costs of R&D, authorisation and marketing of these products often cannot be recuperated under normal conditions. The European Commission, has therefore proposed a regulation to set up a procedure for EU-wide designation of medicinal products as orphan drugs (products with little or no payback). The proposal would put in place special incentives such as ten-year exclusive distribution rights throughout the EU, and a full or partial waiver of the costs of the designation procedure. Given the fact that research on rare diseases will remain a priority issue under the Fifth RTD Framework Programme, this Regulation would help overcome some of the impediments to existing research. The system proposed, in particular, the exclusive distribution rights, offers important incentives for investment in unprofitable research. The proposal also ensures that this will not prove prejudicial to the interests of patients and public health by providing for the withdrawal of the right in certain circumstances. The decision on the designation of a medicinal product as an orphan drug would be taken by the European Agency for the Evaluation of Medicinal Products who would also provide assistance in preparing a plan for the clinical trials needed to obtain marketing authorisation for a product.
Policy making and guidelines
26 April 1999