Journal of the Korean Statistical Society ( IF 0.6 ) Pub Date : 2020-02-18 , DOI: 10.1007/s42952-020-00053-w Junjiang Zhong , Miin-Jye Wen , Siu Hung Cheung
Non-inferiority (NI) trials have gained recognition as an effective tool with which to search for substitutes for a standard treatment that is associated with certain undesirable features, such as adverse side-effects, an exorbitant cost, or an extremely complicated therapeutic regimen. Statistical methods have been developed for NI studies with multiple experimental treatments. However, experimental treatments sometimes represent a new treatment with various dose levels, and in such circumstances, identification of the minimum non-inferior dose is a reasonable objective of the trial. Thus, we examine several potential testing procedures with respect to this testing objective. A simulation study is conducted to evaluate their performance, and determination of the sample size is discussed. Two clinical examples are provided to illustrate the proposed method.
中文翻译:
在三臂非劣效性试验中确定最小非劣效剂量
非劣效性(NI)试验已被公认为一种有效的工具,可用于寻找与某些不良特征(例如不良副作用,高昂的费用或极其复杂的治疗方案)相关的标准治疗方法。已经为具有多种实验方法的NI研究开发了统计方法。但是,实验治疗有时代表具有不同剂量水平的新治疗,在这种情况下,确定最低非劣效剂量是该试验的合理目标。因此,我们针对该测试目标检查了几种潜在的测试程序。进行了仿真研究以评估其性能,并讨论了样本量的确定。