临床试验中的无效停止、最优性和实际考虑。
Futility stopping in clinical trials, optimality and practical considerations.
机构信息
Department of Biostatistics, The University of North Carolina, Chapel Hill, North Carolina, USA.
出版信息
J Biopharm Stat. 2020 Nov 1;30(6):1050-1059. doi: 10.1080/10543406.2020.1818253. Epub 2020 Sep 14.
Abstract
Stopping for futility is a useful tool in a clinical trial. It is widely used in single-arm trials in oncology and in many two-arm trials. We review three stopping rules for futility. We give recommendations for the optimal timing of futility looks in two-stage trials in terms of the information fraction and the probability of stopping under the alternative hypothesis. We discuss futility stopping in trials with substantial uncertainty about the variability of the outcome and in crossover trials.
摘要
在临床试验中，停止无效治疗是一种有用的工具。它在肿瘤学的单臂试验和许多双臂试验中都得到了广泛应用。我们回顾了三种无效性停止规则。我们根据信息分数和替代假设下停止的概率，为两阶段试验中最佳的无效性观察时机提出了建议。我们还讨论了在结果变异性存在较大不确定性的试验中和交叉试验中无效性停止的问题。
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