Yang Lan-Yan, Chi Yunchan, Chow Shein-Chung
Department of Statistics, National Cheng Kung University, Tainan, Taiwan.
J Biopharm Stat. 2011 May;21(3):437-52. doi: 10.1080/10543406.2010.481803.
In clinical research, it is not uncommon to modify a trial procedure and/or statistical methods of ongoing clinical trials through protocol amendments. A major modification to the study protocol could result in a shift in target patient population. In addition, frequent and significant modifications could lead to a totally different study that is unable to address the medical questions that the original study intended to answer. In this article, we propose a logistic regression model for statistical inference based on a binary study endpoint for trials with protocol amendments. Under the proposed method, sample size adjustment is also derived.
在临床研究中，通过方案修正案修改正在进行的临床试验的试验程序和/或统计方法并不罕见。对研究方案的重大修改可能导致目标患者群体的转变。此外，频繁且重大的修改可能会导致一项完全不同的研究，无法解决原始研究旨在回答的医学问题。在本文中，我们针对有方案修正案的试验，基于二元研究终点提出了一种用于统计推断的逻辑回归模型。在所提出的方法下，还推导了样本量调整。
