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Non-inferiority tests for binary endpoints with variable margins.
Journal of Biopharmaceutical Statistics ( IF 1.1 ) Pub Date : 2019-09-05 , DOI: 10.1080/10543406.2019.1657136
Yixin Ren 1 , Chao Wang 2 , Meiyu Shen 2 , Yi Tsong 2
Affiliation  

Non-inferiority comparison between binary response rates of test and reference treatments is often performed in clinical studies. The most common approach to assess non-inferiority is to compare the difference between the estimated response rates with some margin. Previous methods use a variety of margins, including fixed margin, step-wise constant margin, and piece-wise smooth margin, where the latter two are functions of the reference response rate. The fixed margin approach assumes that the margin can be determined from historical trials with the consistent difference between the reference treatment and placebo, which may not be available. The step-wise constant margin approach suffers discontinuity in the power function which can cause trouble in sample size determination. Furthermore, many methods ignore the variability in margins dependent on the estimated reference response rate, leading to poor type I error control and power function approximation. In this study, we propose a variable margin approach to overcome the difficulties in fixed and step-wise constant margin approaches. We discuss several test statistics and evaluate their performance through simulation studies.



中文翻译:

具有可变边距的二进制端点的非劣等性测试。

在临床研究中经常进行试验和参考疗法的二元反应率之间的非劣效性比较。评估非自卑性的最常见方法是比较估计的响应率与一定幅度之间的差异。先前的方法使用各种边距,包括固定边距,逐步恒定边距和分段平滑边距,其中后两个是参考响应率的函数。固定边距方法假设可以从历史试验中确定边距,而参考疗法与安慰剂之间的差异始终是一致的,这可能是不可用的。逐步恒定裕度方法的幂函数不连续,可能会导致样本大小确定方面的麻烦。此外,许多方法忽略了取决于估计的参考响应率的裕度变化,从而导致差的I型误差控制和幂函数逼近。在这项研究中,我们提出了一种可变余量方法来克服固定和逐步恒定余量方法中的困难。我们讨论了几种测试统计数据,并通过模拟研究评估了它们的性能。

更新日期:2019-09-05
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