Kosztolányi György, Boncz Imre, Molnár Mária Judit
Ideggyogy Sz. 2016 Jan 30;69(1-2):37-45. doi: 10.18071/isz.69.0037.
Nervous system involvement is expected up to 60-70% in case of rare diseases. This article aims to present the financial methods and expenditures of rare neurological diseases' orphan medicinal products being financed in the frame of Hungarian social insurance system in 2012.
The subsidized orphan medicines were selected on the Orphanet portal 2012 while orphans financed by compessionate use were provided by the Hungarian National Insurance Fund Administration (OEP) database. Three products exist without orphan designation, however those are intended for the treatment of rare neurological ailments. The medicines were categorized by financial methods and determined by costs.
Numerically, out of 36 pieces of subsidized orphan or orphan criteria fulfilled medicines 17 were authorized for the treatments of rare neurological diseases in the year of 2012. Most of the drugs (14 pieces) were to be financed in the frame of compassionate use by the reimbursement system. The cost amount of social insurance for 387 rare neurological disease patients reached more than 4.5 billion HUF (1.4% of the total pharmaceutical budget in outpatient care).
In Hungary half of the subsidized orphans are intended for the treatments of rare neurological ailments. 30% of the total amount of social insurance for rare diseases' medicinal treatments were used to subsidizing rare neurological disease patients in 2012. Most of the orphan medicines were to be financed in the frame of compassionate use by the reimbursement system for outpatient care. Consequently, a great deal of crucial problems occurred in relation with the unconventional subsidizing method. At the end of 2012 new financial methods have been elaborated and introduced in a pilot phase from 1 January 2013. In spite of the high cost commitment, nearly the entire diagnosed rare disease subpopulation has been provided with subsidized treatments in Hungary. In order to facilitate the access to orphan drugs, collaboration is needed between the financing agencies and the professional representatives for identifying the optimal form of financial subsidy.
在罕见病病例中，预计高达60 - 70%会出现神经系统受累情况。本文旨在介绍2012年在匈牙利社会保险体系框架内获得资助的罕见神经疾病孤儿药品的资助方式和支出情况。
2012年在“孤儿药网”门户上选取了获得补贴的孤儿药，而通过同情用药获得资助的孤儿药则由匈牙利国家健康保险基金管理局（OEP）数据库提供。有三种产品没有孤儿药指定，但它们旨在治疗罕见神经疾病。药品按资助方式分类并确定成本。
从数量上看，在36种获得补贴的孤儿药或符合孤儿药标准的药品中，2012年有17种被批准用于治疗罕见神经疾病。大多数药品（14种）将通过报销系统在同情用药框架内获得资助。387名罕见神经疾病患者的社会保险费用超过45亿匈牙利福林（占门诊护理药品总预算的1.4%）。
在匈牙利，一半的补贴孤儿药用于治疗罕见神经疾病。2012年，罕见病药物治疗社会保险总额的30%用于补贴罕见神经疾病患者。大多数孤儿药将通过门诊护理报销系统在同情用药框架内获得资助。因此，与这种非常规资助方式相关出现了许多关键问题。2012年底制定了新的资助方法，并于2013年1月1日进入试点阶段。尽管成本投入很高，但在匈牙利几乎所有确诊的罕见病亚群体都获得了补贴治疗。为了便于获得孤儿药，资助机构和专业代表之间需要合作，以确定最佳的财政补贴形式。
