Makady Amr, Ham Renske Ten, de Boer Anthonius, Hillege Hans, Klungel Olaf, Goettsch Wim
The National Healthcare Institute (ZIN), Diemen, The Netherlands; Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht, The Netherlands.
Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht, The Netherlands.
Value Health. 2017 Apr;20(4):520-532. doi: 10.1016/j.jval.2016.12.003. Epub 2017 Jan 27.
Randomized controlled trials provide robust data on the efficacy of interventions rather than on effectiveness. Health technology assessment (HTA) agencies worldwide are thus exploring whether real-world data (RWD) may provide alternative sources of data on effectiveness of interventions. Presently, an overview of HTA agencies' policies for RWD use in relative effectiveness assessments (REA) is lacking.
To review policies of six European HTA agencies on RWD use in REA of drugs. A literature review and stakeholder interviews were conducted to collect information on RWD policies for six agencies: the Dental and Pharmaceutical Benefits Agency (Sweden), the National Institute for Health and Care Excellence (United Kingdom), the Institute for Quality and Efficiency in Healthcare (Germany), the High Authority for Health (France), the Italian Medicines Agency (Italy), and the National Healthcare Institute (The Netherlands). The following contexts for RWD use in REA of drugs were reviewed: initial reimbursement discussions, pharmacoeconomic analyses, and conditional reimbursement schemes. We identified 13 policy documents and 9 academic publications, and conducted 6 interviews.
Policies for RWD use in REA of drugs notably differed across contexts. Moreover, policies differed between HTA agencies. Such variations might discourage the use of RWD for HTA.
To facilitate the use of RWD for HTA across Europe, more alignment of policies seems necessary. Recent articles and project proposals of the European network of HTA may provide a starting point to achieve this.
随机对照试验提供的是关于干预措施疗效而非效果的有力数据。因此，全球卫生技术评估（HTA）机构正在探索真实世界数据（RWD）是否可提供干预措施效果的替代数据来源。目前，缺乏对HTA机构在相对疗效评估（REA）中使用RWD的政策概述。
回顾六个欧洲HTA机构在药物REA中使用RWD的政策。通过文献综述和利益相关者访谈，收集了六个机构的RWD政策信息：瑞典牙科和药品福利局、英国国家卫生与临床优化研究所、德国医疗质量和效率研究所、法国卫生高级管理局、意大利药品管理局以及荷兰国家卫生保健研究所。对药物REA中RWD使用的以下情况进行了审查：初始报销讨论、药物经济学分析和有条件报销计划。我们识别出13份政策文件和9篇学术出版物，并进行了6次访谈。
药物REA中RWD使用的政策在不同情况下显著不同。此外，HTA机构之间的政策也存在差异。这种差异可能会阻碍RWD在HTA中的使用。
为促进RWD在欧洲HTA中的使用，政策上似乎需要更多的协调一致。欧洲HTA网络最近的文章和项目提案可能为此提供一个起点。
