Progress in methodological research on bridging the efficacy-effectiveness gap of clinical interventions (1): to improve the validity of real-world evidence
10.3760/cma.j.cn112338-20230925-00189
- VernacularTitle: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
- Author:
Zuoxiang LIU
1
;
Zilin LONG
;
Zhirong YANG
;
Shuyuan SHI
;
Xinran XU
;
Houyu ZHAO
;
Zuyao YANG
;
Zhu FU
;
Haibo SONG
;
Tengfei LIN
;
Siyan ZHAN
;
Feng SUN
Author Information
1. 北京大学公共卫生学院流行病与卫生统计学系，北京 100191
- Keywords:
Randomized controlled trial;
Real-world evidence;
Target trial;
Efficacy;
Effectiveness
- From:
Chinese Journal of Epidemiology
2024;45(2):286-293
- CountryChina
- Language:Chinese
-
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 31 st 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.
