真实世界研究：中医干预措施效果评价的新理念

解释性随机对照试验（explanatory randomized controlled trial,ERCT）得出的结论往往在真实临床环境下缺乏实际应用价值,而实用性随机对照试验（pragmatic randomized controlled trial,PRCT）在试验结果的推广上也存在一定的局限性。一些国家已开展真实世界研究（real-world study,RWS）来探讨临床干预措施或上市后药品的有效性和安全性,这是一种新的临床研究理念。通过回顾国外开展的RWS,我们总结其研究设计的基本特征,并探讨RWS和PRCT之间的区别和联系。RWS和PRCT在设计理念上具有不同之处。在RWS中,研究者倾向于在超大样本量和广泛受试人群的基础上开展长期评价,并注重有临床意义的结局指标。数据采集、管理和分析方面的严格控制对于RWS和PRCT而言都是至关重要的。鉴于中医临床干预措施及其评价的复杂性,PRCT并不完全适用于中医临床研究,将RWS运用于中医临床研究是一种很好的思路。我们可以通过PRCT来探讨中医干预措施的效力,并通过RWS来深入理解其真实临床效果。RWS将成为中医临床试验和中药上市后再评价研究的一种新方法。

Real-world study:a potential new approach to effectiveness evaluation of traditional Chinese medicine interventions

Some conclusions drawn from explanatory randomized controlled trial(ERCT) lack practical value in application under real-world clinical settings,and there are also some limitations on the generalization of pragmatic randomized controlled trial(PRCT) results.In some countries,real-world studies(RWSs) have been conducted to explore the effectiveness and safety of clinical interventions or postmarketing drugs,which is a new tendency in clinical researches.By reviewing some RWSs carried out in Western countries...