Kaiser Permanente Washington Health Research Institute.
Harvard Pilgrim Health Care Institute, Harvard Medical School.
Clin Pharmacol Ther. 2022 Jan;111(1):30-34. doi: 10.1002/cpt.2253. Epub 2021 May 19.
Concerns regarding both the limited generalizability and the slow pace of traditional randomized trials have led to calls for greater use of real-world evidence (RWE) in the evaluation of new treatments or products. The RWE label has been used to refer to a variety of departures from the methods of traditional randomized controlled trials. Recognizing this complexity and potential confusion, the National Academies of Science, Engineering, and Medicine convened a series of workshops to clarify and address questions regarding the use of RWE to evaluate new medical treatments. Those workshops identified three specific dimensions in which RWE studies might differ from traditional clinical trials: use of real-world data (data extracted from health system records or data captured by mobile devices), delivery of real-world treatment (open-label treatments delivered in community settings by community practitioners), and real-world treatment assignment (including nonrandomized comparisons and variations on random assignment such as before-after or stepped-wedge designs). For any RWE study, decisions regarding each of these dimensions depends on the specific research question, characteristics of the potential study settings, and characteristics of the settings where study results would be applied.
人们对传统随机试验的普遍适用性有限和进展缓慢感到担忧，因此呼吁在评估新治疗方法或产品时更多地使用真实世界证据 (RWE)。RWE 这一标签已被用于指代各种偏离传统随机对照试验方法的情况。为了厘清并解决与使用 RWE 评估新医疗方法相关的问题，美国国家科学院、工程院和医学院召集了一系列研讨会。这些研讨会确定了 RWE 研究可能与传统临床试验存在差异的三个具体方面：真实世界数据的使用（从健康系统记录中提取的数据或移动设备捕获的数据）、真实世界治疗的实施（社区医生在社区环境中提供开放性标签治疗）以及真实世界治疗的分配（包括非随机对照和随机分配的变化，如前后或逐步楔形设计）。对于任何 RWE 研究，对于这些方面的每一个方面的决策都取决于具体的研究问题、潜在研究环境的特征以及研究结果将应用的环境的特征。
