title: Access to orphan drugs in western Europe: can more systematic policymaking really help to avoid different decisions about the same drug?
abstract: When the Scottish government recently launched a £21 million fund to provide patients with rare diseases access to treatment, Alex Neill, the Scottish Health Secretary, stated that 'it was only right that Scottish patients with rare conditions had access to innovative medicines which were clinically justified, and that they were not disadvantaged due to the very high cost of these treatments'. This statement raises questions about the criteria that play a role in reimbursement decisions on orphan drugs. This editorial examines the criteria that were used in the decisions about the reimbursement of an orphan drug for Pompe disease and explores methods to improve the transparency and consistency of reimbursement decisions for orphan drugs in general.
authors.affiliation: Institute for Medical Technology Assessment, Erasmus University Rotterdam, PO Box 1738, 3000DR Rotterdam, The Netherlands.
authors.forename: Tim A
authors.initials: TA
authors.lastname: Kanters
authors.forename: Leona
authors.initials: L
authors.lastname: Hakkaart
authors.forename: Maureen Pmh
authors.initials: MP
authors.lastname: Rutten-van Mölken
authors.forename: W Ken
authors.initials: WK
authors.lastname: Redekop
mesh_terms: D003657:Decision Making; D005060:Europe; D006009:Glycogen Storage Disease Type II / Q000188:drug therapy / Q000191:economics; D006297:Health Services Accessibility; D006801:Humans; D007356:Insurance, Pharmaceutical Services / Q000191:economics*; D009965:Orphan Drug Production / Q000191:economics*; D011050:Policy Making; D035583:Rare Diseases / Q000188:drug therapy* / Q000191:economics; D012051:Reimbursement Mechanisms; D012606:Scotland
keywords: Pompe disease; access; multi-criteria decision analysis; orphan drugs; reimbursement