目的 干预措施在临床实践中的实际干预效果与随机对照试验(RCT)中表现的效力存在差异,即效力-效果差距.RCT结果与真实世界研究(RWS)结果的差异可能无法代表真实的效力-效果差距,这是因为当RWS与RCT在研究设计上有较大差异,或RWS结果估计存在偏倚时,效力-效果的估计可能是有偏的.其次,当发现干预措施存在效力-效果差距,不能对所有患者实行一刀切的临床决策,而需要进一步评估影响干预措施效果的真实世界因素,识别可能取得期望效用的患者群体.方法 检索PubMed、Embase、Web of Science、万方数据知识服务平台、维普数据库、中国知网6个数据库从建库至2022年12月31日的中英文文献,采用概括性综述的方法,对如何改进RWS设计从而弥合效力-效果差距的方法进行归纳整合和定性描述.结果 共纳入10篇文献,探讨如何以RCT研究方案为模板,制定相应的RWS方案,在正确估计效力-效果差距的基础上,进一步评估干预措施在患者亚群中的效果,选取能获得预期收益风险比的患者亚群,从而弥合效力-效果差距.结论 使用医疗大数据,模拟目标试验方案关键特征,可以提高研究结果的真实性和有效性,弥合效力-效果差距.
Progress in methodological research on bridging the efficacy-effectiveness gap of clinical interventions(1):to improve the validity of real-world evidence
Objective Differences between randomized controlled trial(RCT)results and real world study(RWS)results may not represent a true efficacy-effectiveness gap because efficacy-effectiveness gap estimates may be biased when RWS and RCT differ significantly in study design or when there is bias in RWS result estimation.Secondly,when there is an efficacy-effectiveness gap,it should not treat every patient the same way but assess the real-world factors influencing the intervention's effectiveness and identify the subgroup likely to achieve the desired effect.Methods Six databases(PubMed,Embase,Web of Science,CNKI,Wanfang Data,and VIP)were searched up to 31st December 2022 with detailed search strategies.A scoping review method was used to integrate and qualitatively describe the included literature inductively.Results Ten articles were included to discuss how to use the RCT research protocol as a template to develop the corresponding RWS research protocol.Moreover,based on correctly estimating the efficacy-effectiveness gap,evaluate the intervention effect in the patient subgroup to confirm the subgroup that can achieve the expected benefit-risk ratio to bridge the efficacy-effectiveness gap.Conclusion Using real-world data to simulate key features of randomized controlled clinical trial study design can improve the authenticity and effectiveness of study results and bridge the efficacy-effectiveness gap.
NETL
NSTL
万方数据
