title: [European incentives for orphan medicinal products].
abstract: The designation as an orphan medicinal product is required in order to benefit from European Union (EU) incentives for the development of drugs for rare diseases. The European Commission decides on the designation based on the opinion of the Committee for Orphan Medicinal Products within the European Medicines Agency. Designation as an orphan drug is clearly different from marketing authorization. Criteria for the designation as an orphan drug are the low prevalence of the disease (less than 5 in 10 000 inhabitants of the EU), severity of the disease and the expected significant benefit for the patients. Incentives include fee reductions and free scientific advice during development and up to ten years market exclusivity after marketing authorization. This article describes the procedure of orphan drug designation and highlights problems regarding coverage of treatment costs and increasingly individualized medicine.
authors.affiliation: Bundesinstitut für Arzneimittel und Medizinprodukte, Bonn, BRD. h.enzmann@bfarm.de
authors.forename: H
authors.initials: H
authors.lastname: Enzmann
authors.forename: J
authors.initials: J
authors.lastname: Lütz
mesh_terms: D004345:Drug Industry / Q000191:economics* / Q000639:trends*; D005060:Europe; D006801:Humans; D009965:Orphan Drug Production / Q000191:economics* / Q000379:methods*; D035583:Rare Diseases / Q000188:drug therapy* / Q000191:economics*; D012052:Reimbursement, Incentive / Q000458:organization & administration*