Optimal sample size allocation for two-arm superiority and non-inferiority trials with binary endpoints

被引:0
|
作者
Kirchner, Marietta [1 ]
Schuepke, Stefanie [2 ,3 ,4 ]
Kieser, Meinhard [1 ,5 ]
机构
[1] Heidelberg Univ, Inst Med Biometry, Heidelberg, Germany
[2] Privatpraxis Kardiol, Frankfurt, Germany
[3] Deutsch Herzzentrum Munich, ISARes Ctr, Munich, Germany
[4] DZHK German Ctr Cardiovasc Res, Partner Site Munich Heart Alliance, Berlin, Germany
[5] Heidelberg Univ, Inst Med Biometry, Im Neuenheimer Feld 130 3, D-69120 Heidelberg, Germany
关键词
allocation ratio; clinical trial; power; sample size; UNEQUAL RANDOMIZATION; CLINICAL-TRIALS;
D O I
10.1002/pst.2375
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
The sample size of a clinical trial has to be large enough to ensure sufficient power for achieving the aim the study. On the other side, for ethical and economical reasons it should not be larger than necessary. The sample size allocation is one of the parameters that influences the required total sample size. For two-arm superiority and non-inferiority trials with binary endpoints, we performed extensive computations over a wide range of scenarios to determine the optimal allocation ratio that minimizes the total sample size if all other parameters are fixed. The results demonstrate, that for both superiority and non-inferiority trials the optimal allocation may deviate considerably from the case of equal sample size in both groups. However, the saving in sample size when allocating the total sample size optimally as compared to balanced allocation is typically small.
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页数:9
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