de Lusignan Simon, Crawford Laura, Munro Neil
University of Surrey, Guildford, UK.
J Innov Health Inform. 2015 Nov 4;22(3):368-73. doi: 10.14236/jhi.v22i3.177.
New forms of evidence are needed to complement evidence generated from randomised controlled trials (RCTs). Real-World Evidence (RWE) is a potential new form of evidence, but remains undefined. This paper sets to fill that gap by defining RWE as the output from a rigorous research process which: (1) includes a clear a priori statement of a hypothesis to be tested or research question to be answered; (2) defines the data sources that will be used and critically appraises their strengths and weaknesses; and (3) applies appropriate methods, including advanced analytics. These elements should be set out in advance of the study commencing, ideally in a published protocol.The strengths of RWE studies are that they are more inclusive than RCTs and can enable an evidence base to be developed around real-world effectiveness and to start to address the complications of managing other real-world problems such as multimorbidity. Computerised medical record systems and big data provide a rich source of data for RWE studies. However, guidance is needed to help assess the rigour of RWE studies so that the strength of recommendations based on their output can be determined. Additionally, RWE advanced analytics methods need better categorisation and validation.We predict that the core role of RCTs will shift towards assessing safety and achieving regulatory compliance. RWE studies, notwithstanding their limitations, may become established as the best vehicle to assess efficacy.
需要新的证据形式来补充随机对照试验（RCT）产生的证据。真实世界证据（RWE）是一种潜在的新证据形式，但仍未明确界定。本文旨在通过将RWE定义为严格研究过程的产出，来填补这一空白，该过程包括：（1）对要检验的假设或要回答的研究问题有明确的先验陈述；（2）定义将使用的数据来源，并严格评估其优缺点；（3）应用适当的方法，包括高级分析。这些要素应在研究开始前确定，理想情况下应在已发表的方案中列出。RWE研究的优势在于，它们比RCT更具包容性，能够围绕真实世界的有效性建立证据基础，并开始解决管理其他真实世界问题（如多种疾病并存）的复杂性。计算机化医疗记录系统和大数据为RWE研究提供了丰富的数据来源。然而，需要指导来帮助评估RWE研究的严谨性，以便确定基于其产出的推荐强度。此外，RWE高级分析方法需要更好的分类和验证。我们预测，RCT的核心作用将转向评估安全性和实现监管合规。RWE研究尽管有其局限性，但可能会成为评估疗效的最佳手段。
