Assessing non-inferiority of a new treatment in a three-arm clinical trial including a placebo
Assessing non-inferiority of a new treatment in a three-arm clinical trial including a placebo
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DOI：
10.1002/sim.1450
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发表时间：
2003-03-30
影响因子：
2
通讯作者：
Hauschke, D
中科院分区：
文献类型：
--
作者：
Pigeot, I;Schäfer, J;Hauschke, D
在非劣效性试验中，必须证明新实验药物与活性对照相比的非劣效性，如果伦理上合理，建议使用额外的安慰剂组进行内部验证。本文的重点是这样的设计。假设正态性和方差齐性，我们将推导出一个统计检验程序，该程序相当于基于Fieller置信区间的评估。根据本试验的功效函数，进行样本量计算以达到给定功效。此外，总样本量的最优分配。作为一种替代方法，这个参数的过程中，自助百分位数区间进行了讨论，并最终与Fieller的置信区间在轻度哮喘患者的研究。版权所有（C）2003约翰威利父子有限公司。
In non-inferiority trials, where non-inferiority of a new experimental drug compared to an active control has to be shown, it may be advisable to use an additional placebo group for internal validation if ethically justifiable. The focus of this paper is on such designs. Assuming normality and homogeneity of variances we will derive a statistical test procedure which turns out to be equivalent to the assessment based on Fieller's confidence interval. Based on the power function of this test, sample size calculations are carried out to achieve a given power. Additionally, the optimal allocation of the total sample size is derived. As an alternative to this parametric procedure, the bootstrap percentile interval is discussed and finally compared with Fieller's confidence interval in a study on mildly asthmatic patients. Copyright (C) 2003 John Wiley Sons, Ltd.