title: [Orphan drugs: availability, reliability and reimbursement].
abstract: Orphan drugs are drugs used in the treatment of life-threatening or chronic diseases that affect fewer than 1 out of 2000 persons in the European Union. Since the implementation of the European Regulation on Orphan Medicinal Products in 2000, 61 orphan drugs have been brought to market. One-third of these were granted their marketing authorisations based on non-comparative clinical research. Certain orphan drugs for extramural use will be transferred to the performance-based hospital financing system within the next few years. Unapproved orphan drugs are generally not reimbursed. In so-called compassionate use programmes, unauthorised orphan drugs can still become available to patients who do not participate in clinical trials. Compassionate use drugs are made available by the manufacturer.
authors.affiliation: Academisch Medisch Centrum, afd. Inwendige Geneeskunde, Amsterdam, the Netherlands. A.R.Vegter@amc.uva.nl
authors.forename: A Rosan
authors.initials: AR
authors.lastname: Kreeftmeijer-Vegter
authors.forename: Cees K W
authors.initials: CK
authors.lastname: van Veldhuizen
authors.forename: Peter J
authors.initials: PJ
authors.lastname: de Vries
mesh_terms: D002908:Chronic Disease / Q000188:drug therapy / Q000191:economics; D057176:Compassionate Use Trials; D005062:European Union; D019317:Evidence-Based Medicine; D006801:Humans; D007349:Insurance, Health, Reimbursement; D007880:Legislation, Drug; D009965:Orphan Drug Production / Q000191:economics* / Q000331:legislation & jurisprudence / Q000592:standards*; D035583:Rare Diseases / Q000188:drug therapy* / Q000191:economics*