The Regulation of Orphan Medicines in the EU: Objectives reached and main challenges when facing the future

摘要: “Whereas some conditions occur so infrequently that the cost of developing and bringing to market a medicinal product . would not be recovered by expected sales product; pharmaceutical industry unwilling develop under normal conditions; these products are called ‘orphan’; Patients suffering from rare should entitled same quality treatment as other patients; it is therefore necessary stimulate development ; such action best taken at Community level in order take advantage widest possible avoid dispersion limited sources, with deserve quality, safety efficacy orphan submitted evaluation process; sponsors have possibility obtaining authorisation”. From Regulation (EC) No 141/2000 European Parliament Council implementing Commission regulation 847/2000. To date, more than 7 years experience has been gained. Following adoption regulation, EMEA its Committee Orphan Medicinal Products (COMP) an important new role stimulating (OMPs). Between April 2000 2007 (up March 31, 2007), 673 applications received EMEA, 443 which had positive opinions for designation COMP resulting thirty-five on (Tables 1 2). Compared before when few drugs were developed patients diseases, incentives available Union reached goal well beyond initial expectations. This article reviews far i.e. impact benefits seen date areas where future