配对二项数据等效性/非劣效性评价的样本含量估计和假设检验

新药及医疗器械临床试验中, 有时会涉及到两比较组采用配对设计获得的二项反应数据(配对二项数据) 的等效性 非劣效性问题。两独立组率之间等效性/非劣效试验的样本含量估计及假设检验方法已较为成熟, 但对于配对二项数据两组率之间的等效性/非劣效性试验的样本含量估计及假设检验方法还应用不多。本文介绍了一种渐进的基于约束极大似然估计的方法用于配对二项数据两组率之间的等效性 非劣效性试验的样本含量估计和假设检验, 借助一个超声诊断仪临床试验的例子阐明了本方法的应用, 还就有关实际问题进行了讨论。

Sample size estimation and hypothesis testing of assessing equivalence/noninferiority for paired binary data

Sometimes assessment of equivalence / noninferiority between two new drugs and medical devices involves comparisons of the response rates between paired binary endpoints in clinical trials.Statistical procedures have been developed for inferring equivalence / noninferiority often concentrated on comparing the rates of two independent binary responses in the past few years.Procedures for assessing equivalence/ noninferiority between the paired binary endpoints have not been well studied.In this paper, we introduce the determination of sample size and hypothesis testing of equivalence / noninferiority for paired binary data based on restricted maximum likelihood estimation.An equivalence / noninferiority clinical trial of ultrasound diagnostic device is used to illustrate the proposed methods.Discussions on some important issues are provided to help understand and conduct these trials.