Bayesian sample‐size determination methods considering both worthwhileness and unpromisingness for exploratory two‐arm randomized clinical trials with binary endpoints
Bayesian sample‐size determination methods considering both worthwhileness and unpromisingness for exploratory two‐arm randomized clinical trials with binary endpoints
复制标题
考虑具有二元终点的探索性双臂随机临床试验的价值和前景的贝叶斯样本量确定方法
DOI：
10.1002/pst.1971
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发表时间：
2019
影响因子：
1.5
通讯作者：
Daimon Takashi
中科院分区：
文献类型：
--
作者：
Kakizume Tomoyuki;Zhang Fanghong;Kawasaki Yohei;Daimon Takashi
通常进行一项随机探索性临床试验，比较试验性治疗与对照治疗的二元终点，以做出进行或不进行的决定。这种探索性试验需要有足够的样本量，以便提供令人信服的证据，证明实验治疗相对于对照治疗是值得的或没有希望的。在本文中，我们提出了三种新的探索性试验的样本量确定方法，这些方法利用根据预定义的有效性和无效性标准计算的后验概率，从而宣布实验治疗的可行性或不可行性。包括数值研究在内的模拟研究表明，当实验治疗组的真实反应概率分别高于或低于对照治疗组时，这三种方法都可以以很高的概率宣布实验治疗是值得的或没有希望的。
A randomized exploratory clinical trial comparing an experimental treatment with a control treatment on a binary endpoint is often conducted to make a go or no‐go decision. Such an exploratory trial needs to have an adequate sample size such that it will provide convincing evidence that the experimental treatment is either worthwhile or unpromising relative to the control treatment. In this paper, we propose three new sample‐size determination methods for an exploratory trial, which utilize the posterior probabilities calculated from predefined efficacy and inefficacy criteria leading to a declaration of the worthwhileness or unpromisingness of the experimental treatment. Simulation studies, including numerical investigation, showed that all three methods could declare the experimental treatment as worthwhile or unpromising with a high probability when the true response probability of the experimental treatment group is higher or lower, respectively, than that of the control treatment group.