论文标题
样本量在第2阶段剂量找到的重新估计：条件功率与贝叶斯预测能力
Sample size re-estimation in Phase 2 Dose-Finding: Conditional power vs. Bayesian predictive power
论文作者
论文摘要
当对真实治疗效果存在很大的不确定性时，通常会计划在临床试验中计划未盲的样本量重估算（SSR）。对于II期剂量发现研究中的概念证明（POC），可以采用对比度测试来利用所有治疗组的信息。在本文中，我们提出了使用频繁的条件功率和贝叶斯后验预测能力的两阶段SSR设计，用于单个和多个对比度测试。贝叶斯SSR可以在广泛的先前设置下实施，以融合不同的先验知识。考虑到适应性，本文中所有最终分析的I型错误都受到严格保护。进行了仿真研究以证明在多臂试验中未盲的SSR的优势。
Unblinded sample size re-estimation (SSR) is often planned in a clinical trial when there is large uncertainty about the true treatment effect. For Proof-of Concept (PoC) in a Phase II dose finding study, contrast test can be adopted to leverage information from all treatment groups. In this article, we propose two-stage SSR designs using frequentist conditional power and Bayesian posterior predictive power for both single and multiple contrast tests. The Bayesian SSR can be implemented under a wide range of prior settings to incorporate different prior knowledge. Taking the adaptivity into account, all type I errors of final analysis in this paper are rigorously protected. Simulation studies are carried out to demonstrate the advantages of unblinded SSR in multi-arm trials.
