经典的随机对照试验(RCT)是评价理论疗效的金标准,但其结果外推到实际的临床患者时常常受限。 为了帮助临床医师、患者和管理者更好地进行诊疗决策,仅有理论疗效是不够的,还需要提供这些疗法在“真实世界”中的效果,实效研究应运而生。 本文概述了实效研究产生的背景、相关的概念、可以采用的方法,以及面临的挑战。

While classic randomized controlled trials (RCT) is traditionally viewed as a research gold-standard for evaluation of the theoretical efficacy,the results are often limited when applied to the actual clinical patients. To help health care providers, patients, and policymakers make better health care decisions, only theoretical efficacy given is not enough, as effects of these RCT therapies in the “real world”should also be provided, which has made the outcome research emerge. This article is aimed to briefly introduce the background, relevant concepts, methodologies, and challenges faced in the development of clinical outcome research.