非劣性/等效性试验的样本含量估计及把握度分析

目的介绍以标准治疗为对照的非劣性/等效性试验样本含量估计及把握度分析.方法方法主要参考国际上近年有关非劣性/等效性试验设计和分析的进展,对涉及到的统计事项和相关问题进行探讨和应用.结果针对非劣性/等效性试验的特定目的,详细阐明了样本含量估计各相关要素的意义及设定方法,给出了样本含量估计及把握度计算的通用公式,结合临床试验的实际案例对样本含量和把握度进行了应用分析.结论随着我国药物临床试验与国际的接轨,应该按照非劣性/等效性来设计试验的情形会有所增多,本文所介绍的方法将为实际工作提供有效的参考.

Sample Size Determination and Power Analysis for Noninferiority/Equivalence Trials

Objective To introduce some statistical procedures including sample size determination and power analysis in therapeutic noinferiority/equivalence trials designed to compare experimental treatment with standard treatment.Methods Reviewing some development of this type of trial, the statistical issues were involved and summed up.Results According to the special consideration, meaning of the key factors which were used to estimate sample size was detailed to clarify. The general formulae of sample size and power were given and applied to the real examples.Conclusion Along with the more and more conducting of noinferiority/equivalence trials in our country, the authors were confident that these methods introduced in the paper would provide to consult effectively for the trial design and evaluation.