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Utility of Adaptive Sample Size designs and A Review Example

Journal of Statistical Science and Application 2017年第1期5卷 1-15页

作者： Kohei Uemura Yuki Ando Yutaka Matsuyama Biostatistics group Center for Product Evaluation Pharmaceuticals and Medical Devices Agency Tokyo Japan Department of Biostatistics School of Public Health University of Tokyo Tokyo Japan

Sample size can be a key design feature that not only affects the probability of a trial＇s success but also determines the duration and feasibility of a trial. If an investigational drug is expected to be effective and address unmet medical needs of an orphan disease, where the accrual period may require many years with a large sample size to detect a minimal clinically relevant treatment effect, a minimum sample size may be set to maintain nominal power. In limited situations such as this, there may be a need for flexibility in the initial and final sample sizes; thus, it is useful to consider the utility of adaptive sample size designs that use sample size re-estimation or group sequential design. In this paper, we propose a new adaptive performance measure to consider the utility of an adaptive sample size design in a trial simulation. Considering that previously proposed sample size re-estimation methods do not take into account errors in estimation based on interim results, we propose Bayesian sample size re-estimation criteria that take into account prior information on treatment effect, and then, we assess its operating characteristics in a simulation study. We also present a review example of sample size re-estimation mainly based on published paper and review report in Pharmaceuticals and Medical Devices Agency （PMDA）.

关键词： adaptive design sample size re-estimation group sequential design utility Bayesian method regulatorydiscussion review example

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