1. Optimal promising zone designs
- Author
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Cyrus R. Mehta, Lingyun Liu, and Samuel T. Hsiao
- Subjects
Statistics and Probability ,Optimal design ,Mathematical optimization ,Biometry ,Computer science ,01 natural sciences ,gold standard sample size reassessment rule ,promising zone design ,sample size reassessment ,010104 statistics & probability ,03 medical and health sciences ,0302 clinical medicine ,Interim ,Test statistic ,adaptive design ,power comparisons of adaptive versus nonadaptive ,Humans ,030212 general & internal medicine ,group sequential design ,0101 mathematics ,trial optimization ,Expected utility hypothesis ,Clinical Trials as Topic ,optimal adaptive design ,General Medicine ,Decision rule ,Class (biology) ,Research Papers ,Pancreatic Neoplasms ,Sample size determination ,Statistics, Probability and Uncertainty ,Construct (philosophy) ,Research Paper - Abstract
Clinical trials with adaptive sample size reassessment based on an unblinded analysis of interim results are perhaps the most popular class of adaptive designs (see Elsäßer et al., 2007). Such trials are typically designed by prespecifying a zone for the interim test statistic, termed the promising zone, along with a decision rule for increasing the sample size within that zone. Mehta and Pocock (2011) provided some examples of promising zone designs and discussed several procedures for controlling their type‐1 error. They did not, however, address how to choose the promising zone or the corresponding sample size reassessment rule, and proposed instead that the operating characteristics of alternative promising zone designs could be compared by simulation. Jennison and Turnbull (2015) developed an approach based on maximizing expected utility whereby one could evaluate alternative promising zone designs relative to a gold‐standard optimal design. In this paper, we show how, by eliciting a few preferences from the trial sponsor, one can construct promising zone designs that are both intuitive and achieve the Jennison and Turnbull (2015) gold‐standard for optimality.
- Published
- 2018