Optimal unplanned design modification in adaptive two-stage trials

被引:0
|
作者
Pilz, Maximilian [1 ]
Herrmann, Carolin [2 ,3 ,4 ]
Rauch, Geraldine [2 ,3 ,4 ]
Kieser, Meinhard [1 ]
机构
[1] Heidelberg Univ, Univ Med Ctr, Inst Med Biometry, Neuenheimer Feld 130-3, D-69120 Heidelberg, Germany
[2] Charite Univ Med Berlin, Inst Biometry & Clin Epidemiol, Berlin, Germany
[3] Free Univ Berlin, Berlin, Germany
[4] Humboldt Univ, Berlin, Germany
关键词
adaptive design; clinical trial; conditional error principle; optimal design; sample size calculation; SAMPLE-SIZE MODIFICATION; CLINICAL-TRIALS;
D O I
10.1002/pst.2228
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
Adaptive planning of clinical trials allows modifying the entire trial design at any time point mid-course. In this paper, we consider the case when a trial-external update of the planning assumptions during the ongoing trial makes an unforeseen design adaptation necessary. We take up the idea to construct adaptive designs with defined features by solving an optimization problem and apply it to the situation of unplanned design reassessment. By using the conditional error principle, we present an approach on how to optimally modify the trial design at an unplanned interim analysis while at the same time strictly protecting the type I error rate. This linking of optimal design planning and the conditional error principle allows sound reactions to unforeseen events that make a design reassessment necessary.
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页码:1121 / 1137
页数:17
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