具有有序三级分类终点的非劣效性试验设计与分析
Noninferiority trial design and analysis with an ordered three-level categorical endpoint.
作者信息
Brittain Erica, Hu Zonghui
机构信息
Biostatistics Research Branch, National Institute of Allergy and Infectious Diseases, Bethesda, Maryland 20892, USA.
出版信息
J Biopharm Stat. 2009 Jul;19(4):685-99. doi: 10.1080/10543400902964142.
This paper extends standard methodology for noninferiority trial design from a binary endpoint to an ordered three-level endpoint, such as "success," "intermediate," and "failure." A metric that summarizes outcome on this endpoint is proposed, and the corresponding sample size requirements are presented. This ordered endpoint can be collapsed into two different binary endpoints, respectively lumping "intermediate" outcomes with "success" or with "failure." We describe how the ordered three-level endpoint compares with these two binary endpoints with respect to the noninferiority margin and sample size requirements.
本文将非劣效性试验设计的标准方法从二元终点扩展到有序三级终点，如“成功”“中等”和“失败”。提出了一种总结该终点结果的指标，并给出了相应的样本量要求。这个有序终点可以分别合并为两个不同的二元终点，即将“中等”结果与“成功”或“失败”归为一类。我们描述了有序三级终点在非劣效性界值和样本量要求方面与这两个二元终点的比较情况。
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