样本量测定
可解释性
贝叶斯概率
临床试验
I类和II类错误
观察研究
人口
计算机科学
后验概率
稳健性(进化)
医学
统计
医学物理学
计量经济学
数学
机器学习
内科学
生物化学
化学
环境卫生
基因
作者
Ziyu Ji,Junjing Lin,Jianchang Lin
标识
DOI:10.1080/10543406.2022.2058529
摘要
In many therapeutic areas with unmet medical needs, such as pediatric oncology and rare diseases, one of the deterrent factors for clinical trial interpretability is the limited sample size with less-than-ideal operating characteristics. Single arm is usually the only viable design due to feasibility and ethical concerns. For the trial results to be more interpretable and conclusive, the evaluation of operating characteristics, such as type I error rate and power, and the appropriate utilization of prior information for study design, shall be prespecified and fully investigated during the trial planning phase. So far, very few existing literature addressed optimal sample size determination issues for the planning of pediatric and rare population trials, with majority of research focusing on analysis perspective with focus on Bayesian borrowing. In practice, when a single-arm trial is designed for rare population, it is not uncommon that the only information available is from an earlier trial and/or a few clinical publications based on observational studies, often constituting mixed or uncertain conclusions. In light of this, an optimal Bayesian sample size determination method for single-arm trial with binary or continuous endpoint is proposed, where conflicting prior beliefs can be readily incorporated. Prior effective sample size can be calculated to assess the robustness as well as the prior information borrowed. Moreover, due to the lack of closed-form posterior distributions in general, an alternative approach for calculating Bayesian power is described. Simulation studies are provided to demonstrate the utility of the proposed methods. In addition, a case study in pediatric patients with leukemia is included to illustrate the proposed method with the existing approaches.
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