title: [European ordinance on orphan drugs: changes and threats].
abstract: The Orphan Drugs Act has been officially implemented in all countries of the European Union since the year 2000. The Act aims to promote the development of treatments for rare diseases (prevalence < 5:10,000). Successful therapies for such diseases are granted market-monopoly for 10 years, during which no me-too products are allowed. There are problems concerning approval and reimbursement. Drugs already in use can also be registered as orphan drugs for a specific indication. In addition to this, European approval for the market authorization of new drugs does not automatically mean that the costs of the drug are reimbursed within the Netherlands, whereas that might well be the case in other EU countries. However, a faster procedure with respect to the reimbursement of drug costs in the Netherlands may not lead to the responsibilities for the carrying out of additional trials being transferred to those handling the treatment.
authors.affiliation: Academisch Medisch Centrum, afd. Inwendige Geneeskunde, Klinische Hematologie, Meibergdreef 9, 1105 AZ Amsterdam. g.e.linthorst@amc.uva.nl
authors.forename: G E
authors.initials: GE
authors.lastname: Linthorst
authors.forename: C E M
authors.initials: CE
authors.lastname: Hollak
mesh_terms: D003365:Costs and Cost Analysis; D016527:Drug Costs; D011307:Drug Prescriptions / Q000191:economics; D005062:European Union; D006801:Humans; D007880:Legislation, Drug; D009426:Netherlands; D009965:Orphan Drug Production / Q000331:legislation & jurisprudence*; D012051:Reimbursement Mechanisms