Pharmacoepidemiology in the era of real-world evidence.
Pharmacoepidemiology in the era of real-world evidence.
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DOI：
10.1007/s40471-017-0123-y
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发表时间：
2017-12
影响因子：
3.3
通讯作者：
Toh S
中科院分区：
文献类型：
--
作者：
Toh S
早在真实世界数据(RWD)和真实世界证据(RWE)这两个术语被创造出来之前，研究人员一直在使用收集的数据作为常规医疗保健提供的一部分，以生成关于医疗产品的利用、好处和风险的证据[1-4]。RWD和RWE的定义有几种变体，但大多数与美国食品和药物管理局(FDA)使用的定义相似，FDA将RWD定义为“与患者健康状况和/或卫生保健提供有关的数据，这些数据通常从各种来源收集”，RWE定义为“关于RWD分析得出的医疗产品的使用和潜在益处或风险的临床证据”[5]。用更现代的描述符重新标记现有的结构并不少见。RWD一词提供了一个更统一的框架，以广泛地获取传统随机对照试验之外收集的各种类型的数据。RWD的例子包括电子健康记录数据、行政索赔数据、产品和疾病登记数据，以及通过智能设备或社交媒体从患者那里收集的数据。但更重要的是，RWD和RWE的引入标志着证据生成范式的转变，这可能对研究、监管科学和公共卫生产生重大影响。美国国会2016年通过的《21世纪治疗法案》指示FDA评估如何使用RWE来支持批准批准的药物的新适应症，或支持或满足批准后研究的要求[6]。欧洲药品管理局也在考虑将RWE作为适应性许可的一部分[7]。换句话说，利用常规收集的电子健康数据的观察性研究或实用试验产生的RWE可能很快就会被用来补充传统随机对照试验的结果，用于批准医疗产品[8，9]。基于RWD的上市后研究的安全性发现，最近已更正式地纳入美国的监管审查[10]，可能会获得额外的监管重要性。未来几年，监管机构和研究界将如何反应和定位，这将是一件有趣的事情。药物流行病学是一个研究领域，涉及对常规收集的电子健康数据的分析。该领域制定了开展和报告基于可持续发展的研究的良好做法[11-15]。建立在其坚实的基础上，药物流行病学可以在RWD和RWE时代的几个领域取得进展。
Long before the terms real-world data (RWD) and real-world evidence (RWE) were coined, researchers had been using data collected as part of routine healthcare delivery to generate evidence about the utilization, benefits, and risks of medical products [1–4]. There are several variations to the definitions of RWD and RWE, but most are similar to the ones used by the US Food and Drug Administration (FDA), which defines RWD as “data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources” and RWE as “the clinical evidence regarding the usage, and potential benefits or risks, of a medical product derived from analysis of RWD”[5]. It is not uncommon to re-label an existing construct with a more contemporary descriptor. The term RWD provides a more unified framework to broadly capture the various types of data collected outside of traditional randomized controlled trials. Examples of RWD include electronic health record data, administrative claims data, product and disease registry data, and data collected from patients through smart devices or social media. But more important, the introduction of RWD and RWE signifies a shift in the paradigm of evidence generation that could have major impacts on research, regulatory science, and public health. The 21st Century Cures Act, passed by the US Congress in 2016, directed the FDA to evaluate how RWE may be used to support approval of new indications for approved drugs or to support or satisfy post-approval study requirements [6]. The European Medicines Agency is also considering the inclusion of RWE as part of adaptive licensing [7]. In other words, RWE generated from observational studies or pragmatic trials that leverage routinely collected electronic health data may soon be used to supplement findings from traditional randomized controlled trials in the approval of medical products [8, 9]. Safety findings from postmarketing RWD-based studies, which have been more formally integrated into regulatory review in the USA recently [10], may gain additional regulatory importance. It will be interesting to see how regulatory agencies and the research community react and position themselves in the coming years. Pharmacoepidemiology is a research field that involves analysis of routinely collected electronic health data. The field has developed good practices on the conduct and report of RWD-based studies [11–15]. Building upon its solid foundation, pharmacoepidemiology can move forward in several domains in the era of RWD and RWE.