Statistical methods and application cases examples for multiplicity issues in multiple endpoints clinical trials
10.3969/j.issn.1006-7795.2025.02.002
- VernacularTitle:多终点临床试验中多重性问题的统计学方法介绍与应用案例
- Author:
Xiudan BAI
1
;
Qin XU
1
;
Anxin WANG
1
Author Information
1. 首都医科大学附属北京天坛医院 北京市神经外科研究所流行病学研究室,北京 100070;首都医科大学附属北京天坛医院国家神经系统疾病临床医学研究中心,北京 100070;首都医科大学临床流行病学与临床试验学系,北京 100070
- Publication Type:Journal Article
- Keywords:
clinical trials;
multiple endpoints;
multiplicity;
testing strategies;
statistical methods
- From:
Journal of Capital Medical University
2025;46(2):184-190
- CountryChina
- Language:Chinese
-
Abstract:
Performing multiple tests without adjusting the test level results in a higher-than-intended overall familywise error rate(FWER).This phenomenon is known as the multiplicity problem.In this paper,we first introduced the mechanism of multiplicity problem based on the classification and characterization of clinical endpoints.Then,strategies and methods to solve the multiplicity problem were introduced,including the parallel strategy/single-step method,the sequential strategy/multistep method/stepwise method,and their combinations.The results of different analytic strategies may vary.The practical application of the above common strategies and statistical methods were introduced through the case studies of domestic and foreign investigator initiated clinical trial.Multiplicity adjustment for multiple endpoints in clinical trials can be achieved by a single strategy or a combination of strategies.Depending on the selected strategy or combination,the statistical methods and significance level(denoted as α)for each test hypothesis are determined to effectively control the multiplicity problem.
