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A score test for binary data with patient non‐compliance
Author(s) -
Branson Michael,
Whitehead John
Publication year - 2003
Publication title -
statistics in medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.996
H-Index - 183
eISSN - 1097-0258
pISSN - 0277-6715
DOI - 10.1002/sim.1542
Subject(s) - test (biology) , statistics , randomization , test statistic , statistic , sample size determination , binary data , score test , exact test , binary number , compliance (psychology) , computer science , medicine , mathematics , clinical trial , statistical hypothesis testing , psychology , arithmetic , paleontology , social psychology , biology
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 © 2003 John Wiley & Sons, Ltd.