比较欧洲罕见病药物的可及性。
Comparing access to orphan medicinal products in Europe.
机构信息
Office of Health Economics, Southside, 7th Floor, 105 Victoria Street, London, SW1E 6QT, UK.
出版信息
Orphanet J Rare Dis. 2019 May 3;14(1):95. doi: 10.1186/s13023-019-1078-5.
OBJECTIVES
The primary objective of this study was to compare the availability and access of orphan medicinal products (OMPs) in the devolved nations in the United Kingdom (UK), France, Germany, Italy and Spain. Availability is defined as the possibility to prescribe OMPs. Access refers to their full or partial reimbursement by the public health service.
METHODS
Data were collated on: marketing authorisations, Health Technology Assessment (HTA) decisions, commissioning, and reimbursement decisions, and respective dates of these events for all the OMPs centrally authorised. Indicators of availability of and access to OMPs were calculated in each country and compared.
RESULTS
We found that since the implementation of the OMPs Regulation in 2000 to end of May 2016, 143 OMPs obtained a marketing authorisation in the European Union. These OMPs are most widely accessible in Germany and France. In the other countries between 30 and 60% of OMPs are reimbursed. In particular in England, less than 50% of centrally authorised OMPs are routinely funded by the NHS, with one-third of these recommended by NICE. In Germany reimbursement is automatically granted to all medicines which receive a marketing authorisation, immediately after authorisation - but since 2011, there is an evaluation and potentially a pricing negotiation between companies and sickness funds (third party payers). In the other countries, the shortest time from authorisation to a reimbursement decision is observed in Italy and France where it takes 18.6 and 19.5 months respectively on average.
CONCLUSIONS
Marketing authorisation granted to OMPs is only the first step, as medicines reach patients when reimbursement decisions are implemented by national health systems (this applies to non-OMPs too). We found that more than a half of centrally authorised OMPs were available in the five selected countries, but that access to patients was further restricted by different national reimbursement policies, especially in the UK, Italy and Spain.
目的
本研究的主要目的是比较英国（UK）、法国、德国、意大利和西班牙等分散国家的孤儿药（OMP）的可及性和可获得性。可及性定义为开处 OMP 的可能性。可获得性是指公共医疗服务对其的全额或部分报销。
方法
我们收集了所有中央授权 OMP 的营销授权、卫生技术评估（HTA）决策、委托和报销决策，以及这些事件的各自日期的数据。在每个国家计算了 OMP 的可及性和可获得性的指标，并进行了比较。
结果
我们发现，自 2000 年 OMP 法规实施至 2016 年 5 月底，有 143 种 OMP 在欧盟获得了营销授权。这些 OMP 在德国和法国最广泛可用。在其他国家，30%至 60%的 OMP 得到报销。特别是在英格兰，不到 50%的中央授权 OMP 由国民保健制度（NHS）常规资助，其中三分之一由 NICE 推荐。在德国，所有获得营销授权的药物在授权后立即自动获得报销——但自 2011 年以来，公司和疾病基金（第三方付款人）之间进行了评估和潜在的定价谈判。在其他国家，从授权到报销决定的最短时间在意大利和法国观察到，平均分别为 18.6 和 19.5 个月。
结论
授予 OMP 的营销授权仅是第一步，因为当国家卫生系统实施报销决定时，药物才能惠及患者（这也适用于非 OMP）。我们发现，在五个选定国家中，超过一半的中央授权 OMP 是可用的，但由于不同的国家报销政策，特别是在英国、意大利和西班牙，患者获得 OMP 的途径受到了进一步限制。
Zotero 插件