A score test for binary data with patient non-compliance

被引：8

作者：

Branson, M

Whitehead, J

机构：

[1] Novartis Pharma AG, CH-4056 Basel, Switzerland

[2] Univ Reading, Med & Pharmaceut Stat Res Unit, Reading RG6 6FN, Berks, England

来源：

STATISTICS IN MEDICINE | 2003年 / 22卷 / 20期

关键词：

non-compliance; binary data; score test; likelihood ratio test; Wald's test; sample size;

D O I：

10.1002/sim.1542

中图分类号：

Q [生物科学];

学科分类号：

07 ; 0710 ; 09 ;

摘要：

A score test is developed for binary clinical trial data, which incorporates patient non-compliance while respecting randomization. It is assumed in this paper that compliance is 'all-or-nothing', in the sense that a patient either accepts all of the treatment assigned as specified in the protocol, or none of it. Direct analytic comparisons of the adjusted test statistic for both the score test and the likelihood ratio test are made with the corresponding test statistics that adhere to the intention-to-treat principle. It is shown that no gain in power is possible over the intention-to-treat analysis, by adjusting for patient non-compliance. Sample size formulae are derived and simulation Studies are used to demonstrate that the sample size approximation holds. Copyright (C) 2003 John Wiley Sons, Ltd.

引用

页码：3115 / 3132

页数：18

共 41 条

[1]

Armitage P, 2001, STAT METHODS MED RES

[2]

Azzalini A., 1996, Statistical inference: Based on the likelihood

[3] Analysis of survival data from a randomized trial with all-or-none compliance: Estimating the cost-effectiveness of a cancer screening program [J].