Testing of non-inferiority and superiority for three-arm clinical studies with multiple experimental treatments

被引:9
作者
Zhong, Junjiang [1 ,2 ]
Wen, Miin-Jye [2 ]
Kwong, Koon Shing [3 ]
Cheung, Siu Hung [2 ,4 ]
机构
[1] Xiamen Univ Technol, Sch Appl Math, Xiamen, Peoples R China
[2] Natl Cheng Kung Univ, Dept Stat, Tainan, Taiwan
[3] Singapore Management Univ, Sch Econ, Singapore, Singapore
[4] Chinese Univ Hong Kong, Dept Stat, Shatin, Hong Kong, Peoples R China
关键词
Non-inferiority; superiority; three-arm trial; familywise error rate; assay sensitivity; PLACEBO-CONTROLLED TRIALS; ESTABLISH SUPERIORITY/EQUIVALENCE; NONINFERIORITY TRIALS; STANDARD TREATMENTS; RANDOMIZED-TRIAL; HYPOTHESES; METFORMIN; EFFICACY; DESIGN; SAFETY;
D O I
10.1177/0962280216668913
中图分类号
R19 [保健组织与事业(卫生事业管理)];
学科分类号
摘要
The purpose of a non-inferiority trial is to assert the efficacy of an experimental treatment compared with a reference treatment by showing that the experimental treatment retains a substantial proportion of the efficacy of the reference treatment. Statistical methods have been developed to test multiple experimental treatments in three-arm non-inferiority trials. In this paper, we report the development of procedures that simultaneously test the non-inferiority and the superiority of experimental treatments after the assay sensitivity has been established. The advantage of the proposed test procedures is the additional ability to identify superior treatments while retaining an non-inferiority testing power comparable to that of existing testing procedures. Single-step and stepwise procedures are derived and then compared with each other to determine their relative testing power and testing error in a simulation study. Finally, the suggested procedures are illustrated with two clinical examples.
引用
收藏
页码:1751 / 1765
页数:15
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