title: [Shift of focus in the financing of Hungarian drugs. Reimbursement for orphan drugs for treating rare diseases: financing of enzyme replacement therapy in Hungary].
abstract: Focusing on the benefits of patients with rare disease the authors analysed the aspects of orphan medicines financed in the frame of the Hungarian social insurance system in 2012 in order to make the consumption more rational, transparent and predictable. Most of the orphan drugs were financed in the frame of compassionate use by the reimbursement system. Consequently, a great deal of crucial problems occurred in relation to the unconventional subsidized method, especially in the case of the highest cost enzyme replacement therapies. On the base of the findings, proposals of the authors are presented for access to orphan drugs, fitting to the specific professional, economical and ethical aspects of this unique field of the health care system. The primary goal is to provide a suitable subsidized method for the treatment of rare disease patients with unmet medical needs. The financial modification of orphans became indispensible in Hungary. Professionals from numerous fields dealing with rare disease patients' care expressed agreement on the issue. Transforming the orphan medicines' financial structure has been initiated according to internationally shared principles.
authors.affiliation: Emberi Erőforrások Minisztériuma Egészségügyért Felelős Államtitkárság Budapest Arany János u. 6-8. 1051.
authors.forename: Márta
authors.initials: M
authors.lastname: Szegedi
authors.affiliation: Semmelweis Egyetem, Általános Orvostudományi Kar Genomikai Medicina és Ritka Betegségek Intézete Budapest.
authors.forename: Mária Judit
authors.initials: MJ
authors.lastname: Molnár
authors.affiliation: Pécsi Tudományegyetem, Egészségtudományi Kar Egészségbiztosítási Intézet Pécs.
authors.forename: Imre
authors.initials: I
authors.lastname: Boncz
authors.affiliation: Pécsi Tudományegyetem, Egészségtudományi Kar Orvosi Genetikai Intézet Pécs.
authors.forename: György
authors.initials: G
authors.lastname: Kosztolányi
mesh_terms: D057176:Compassionate Use Trials / Q000191:economics; D016527:Drug Costs; D056947:Enzyme Replacement Therapy / Q000191:economics*; D000795:Fabry Disease / Q000188:drug therapy / Q000191:economics; D005380:Financing, Government / Q000331:legislation & jurisprudence / Q000379:methods / Q000458:organization & administration; D005776:Gaucher Disease / Q000188:drug therapy / Q000191:economics; D006009:Glycogen Storage Disease Type II / Q000188:drug therapy / Q000191:economics; D006301:Health Services Needs and Demand; D006801:Humans; D006814:Hungary; D019458:Insurance Coverage; D007348:Insurance, Health; D008059:Mucopolysaccharidosis I / Q000188:drug therapy / Q000191:economics; D016532:Mucopolysaccharidosis II / Q000188:drug therapy / Q000191:economics; D009965:Orphan Drug Production / Q000191:economics*; D035583:Rare Diseases / Q000188:drug therapy* / Q000191:economics*
keywords: enzimpótló terápia; enzyme replacement therapies; financing; finanszírozás; orphan drug; racionalizálás; rare disease; rationalization; ritka betegség; árva gyógyszer