Abstract
刘佐相,龙子临,杨智荣,石舒原,许欣然,赵厚宇,杨祖耀,符祝,宋海波,林腾飞,詹思延,孙凤.临床干预措施效力-效果差距弥合的方法学研究进展(一):改善真实世界研究的效果估计[J].Chinese journal of Epidemiology,2024,45(2):286-293
临床干预措施效力-效果差距弥合的方法学研究进展(一):改善真实世界研究的效果估计
Progress in methodological research on bridging the efficacy-effectiveness gap of clinical interventions (1): to improve the validity of real-world evidence
Received:September 25, 2023  
DOI:10.3760/cma.j.cn112338-20230925-00189
KeyWord: 随机对照试验  真实世界证据  目标试验  效力  效果
English Key Word: Randomized controlled trial  Real-world evidence  Target trial  Efficacy  Effectiveness
FundProject:国家自然科学基金(72074011);中国药品监管科学行动计划第二批重点项目([2021]37-10);海南省博鳌乐城国际医疗旅游先行区管理局真实世界研究专项计划(HNLC2022RWS012)
Author NameAffiliationE-mail
Liu Zuoxiang Department of Epidemiology and Biostatistics, School of Public Health, Peking University, Beijing 100191, China
Key Laboratory of Epidemiology of Major Diseases(Peking University), Ministry of Education, Beijing 100191, China 
 
Long Zilin Department of Epidemiology and Biostatistics, School of Public Health, Peking University, Beijing 100191, China
Key Laboratory of Epidemiology of Major Diseases(Peking University), Ministry of Education, Beijing 100191, China 
 
Yang Zhirong School of Computer Science and Control Engineering, Shenzhen Institute of Advanced Technology, Chinese Academy of Sciences, Shenzhen 518055, China  
Shi Shuyuan China Rehabilitation Science Institute, China Disability Control and Prevention Center, China Disable Persons'Federation, Beijing 100068, China  
Xu Xinran Department of Epidemiology and Biostatistics, School of Public Health, Peking University, Beijing 100191, China
Key Laboratory of Epidemiology of Major Diseases(Peking University), Ministry of Education, Beijing 100191, China 
 
Zhao Houyu Department of Epidemiology and Biostatistics, School of Public Health, Peking University, Beijing 100191, China
Key Laboratory of Epidemiology of Major Diseases(Peking University), Ministry of Education, Beijing 100191, China 
 
Yang Zuyao School of Public Health and Primary Care, the Chinese University of Hong Kong, Hong Kong 999077, China  
Fu Zhu Administration of Hainan Boao Lecheng International Medical Tourism Pilot Zone, Hainan Institute of Real World Data, Haikou 571437, China  
Song Haibo Department of Traditional Chinese Medicine Monitoring and Evaluation, Center for Drug Reevalaution, National Medical Products Administration, Beijing 100076, China
Key Laboratory for Research and Evaluation of Pharmacovigilance, National Medical Products Administration, Beijing 100076, China 
 
Lin Tengfei Biomedical Information Technology Research Center, Institute of Advanced Computing and Digital Engineering, Shenzhen Institute of Advanced Technology, Chinese Academy of Sciences, Shenzhen 518055, China  
Zhan Siyan Department of Epidemiology and Biostatistics, School of Public Health, Peking University, Beijing 100191, China
Key Laboratory of Epidemiology of Major Diseases(Peking University), Ministry of Education, Beijing 100191, China
Clinical Epidemiology Research Center, Peking University Third Hospital, Beijing 100191, China 
 
Sun Feng Department of Epidemiology and Biostatistics, School of Public Health, Peking University, Beijing 100191, China
Key Laboratory of Epidemiology of Major Diseases(Peking University), Ministry of Education, Beijing 100191, China
Administration of Hainan Boao Lecheng International Medical Tourism Pilot Zone, Hainan Institute of Real World Data, Haikou 571437, China 
sunfeng@bjmu.edu.cn 
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Abstract:
      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。
English Abstract:
      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.
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