Division of Cardiology, Duke Clinical Research Institute, Duke University, 2400 Pratt Street, Durham, NC USA.
Division of Cardiology, University Heart Center Zürich, Rämistrasse 100, Zürich, Switzerland.
Eur Heart J. 2018 Aug 21;39(32):2932-2941. doi: 10.1093/eurheartj/ehy236.
Real-world data (RWD) has been defined as data generated outside of traditional randomized clinical trials (RCTs). Though RWD has received increasing attention from regulatory authorities and professional societies, dividing evidence into that derived from 'real-world' vs. 'non-real-world' sources provides only one element of a much larger framework for evidence evaluation. Evidence should be evaluated on the source of the data, the method of treatment allocation (whether any intervention being evaluated was assigned or simply observed as used in practice) and the context in which the evidence was generated (overall study design). Under this framework, RWD refers only to data source, and a study incorporates RWD when it primarily uses data collected for non-research purposes, such as insurance claims data or the electronic health record, regardless of study design. Separation of study design, data source, and context enables parallel evaluation of two critical elements: (i) whether a study can support claims of causal inference, which can be assured with a high degree of confidence only in studies where patients are assigned treatments by protocol; and (ii) whether the study population and clinical context mirror clinical practice, a strength of observational studies using data from clinical practice or administrative claims. In this review, we describe the strengths and weaknesses of observational and non-observational studies, and studies involving RWD and non-RWD, through the lens of anticoagulation for atrial fibrillation (AF). Observational studies employing RWD are useful for describing how oral anticoagulants are used in clinical practice, but generally cannot be used to make claims regarding comparative treatment effects. Questions regarding treatment effect generally are best answered through an RCT, and additional pragmatic RCTs are needed to compare different antithrombotic agents for the prevention of thrombotic events in AF.
真实世界数据（RWD）被定义为在传统随机临床试验（RCT）之外产生的数据。尽管 RWD 受到监管机构和专业协会的越来越多的关注，但将证据分为“真实世界”和“非真实世界”来源提供的证据仅为证据评估的更大框架的一个要素。应该根据数据来源、治疗分配方法（正在评估的任何干预措施是分配的还是在实践中作为常规使用的）以及生成证据的背景（总体研究设计）来评估证据。在这个框架下，RWD 仅指数据源，当一项研究主要使用收集用于非研究目的的数据时，例如保险索赔数据或电子健康记录，则该研究包含 RWD，无论研究设计如何。研究设计、数据源和背景的分离使对两个关键要素的并行评估成为可能：（i）研究是否能够支持因果推断的主张，只有在患者按照方案分配治疗的研究中，才能高度确信地保证这一点；（ii）研究人群和临床背景是否反映了临床实践，这是利用临床实践或行政索赔数据的观察性研究的优势。在这篇综述中，我们通过心房颤动（AF）的抗凝治疗，从观察性和非观察性研究、涉及 RWD 和非 RWD 的研究的角度，描述了这些研究的优缺点。使用 RWD 的观察性研究对于描述口服抗凝剂在临床实践中的使用情况非常有用，但通常不能用于就比较治疗效果提出主张。关于治疗效果的问题通常最好通过 RCT 来回答，需要进行更多的实用 RCT 来比较不同抗血栓药物在 AF 中预防血栓事件的效果。
