Szegedi Márta, Molnár Mária Judit, Boncz Imre, Kosztolányi György
Emberi Erőforrások Minisztériuma Egészségügyért Felelős Államtitkárság Budapest Arany János u. 6-8. 1051.
Semmelweis Egyetem, Általános Orvostudományi Kar Genomikai Medicina és Ritka Betegségek Intézete Budapest.
Orv Hetil. 2014 Nov 2;155(44):1735-41. doi: 10.1556/OH.2014.30031.
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.
着眼于罕见病患者的利益,作者分析了2012年匈牙利社会保险体系框架内资助的孤儿药情况,以使消费更加合理、透明和可预测。大多数孤儿药是通过报销系统在同情用药框架内获得资助的。因此,与这种非常规补贴方式相关出现了大量关键问题,尤其是在成本最高的酶替代疗法方面。基于这些发现,作者针对获取孤儿药提出了建议,以契合医疗保健系统这一独特领域的特定专业、经济和伦理方面。首要目标是为治疗有未满足医疗需求的罕见病患者提供合适的补贴方式。在匈牙利,孤儿药的财务调整变得不可或缺。众多从事罕见病患者护理领域的专业人士在这个问题上达成了共识。已根据国际通用原则启动了孤儿药财务结构的改革。