随机和非随机干预研究的证据整合研究进展

Research progress on evidence synthesis of randomized and non-randomized studies of interventions

证据整合是连接临床实践和最佳证据的桥梁,基于高质量随机对照试验的证据整合通常被认为是最高等级的证据,但其外部真实性受限。在某些情况下,证据整合中纳入非随机干预研究可进一步补充、递补甚至替代随机对照试验证据,如在更广泛人群中评估干预效果和罕见事件,为医疗卫生决策提供更多信息。随着真实世界数据的快速发展和统计分析方法的逐步完善,真实世界证据作为非随机干预研究的重要证据来源,加速了高质量非随机干预研究的发展。然而,由于非随机干预研究缺少随机化会导致潜在选择偏倚和混杂偏倚等,开展基于随机和非随机干预研究的证据整合仍面临诸多挑战。本文基于既往研究,对整合随机和非随机干预研究的研究现状,包括整合前提、整合时机、整合方法、整合结果解释等,进行系统梳理,旨在为研究者和决策者正确使用非随机研究证据提供参考,以期进一步推动最佳证据的产生和临床实践转化。

Evidence synthesis serves as a bridge between clinical practice and the best available evidence.Evidence synthesis based on high-quality randomized controlled trials is generally considered the highest level of evidence,but its external validity is limited.In some scenarios,the inclusion of non-randomized intervention studies(NRSI)in evidence synthesis may further supplement or even replace randomized controlled trial evidence,such as assessing intervention effectiveness and rare events in a broader population to provide more information for health care decision-making.With the rapid development of real-world data and the improvement of statistical analysis methods,real-world evidence,as an important source of evidence for NRSI,has accelerated the development of high-quality NRSI.However,there are numerous challenges in integrating evidence from randomized and non-randomized intervention studies due to selection and confounding biases caused by the lack of randomization.Based on previous studies,this paper systematically examines the current status of integrated randomized and non-randomized intervention studies,including integration premise,timing,methods,and result interpretation,in order to provide references for researchers and policy-makers to correctly use nonrandomized research evidence and further promote optimal evidence generation and clinical practice translation.