Stratified analysis in randomized trials with noncompliance.
This article develops methods for stratified analyses of additive or multiplicative causal effect on binary outcomes in randomized trials with noncompliance. The methods are based on a weighted estimating function for an unbiased estimating function under randomization in each stratum. When known weights are used, the derived estimator is a natural extension of the instrumental variable estimator for stratified analyses, and test-based confidence limits are solutions of a quadratic equation in the causal parameter. Optimal weights that maximize asymptotic efficiency incorporate variability in compliance aspects across strata. An assessment based on asymptotic relative efficiency shows that a substantial enhancement in efficiency can be gained by using optimal weights instead of conventional ones, which do not incorporate the variability in compliance aspects across strata. Application to a field trial for coronary heart disease is provided.
Matsui
Shigeyuki
S
Department of Pharmacoepidemiology, School of Public Health, Kyoto University, Kyoto 606-8501, Japan. matsui@pbh.med.kyoto-u.ac.jp
D002784:Cholesterol / Q000097:blood; D003327:Coronary Disease / Q000517:prevention & control; D003627:Data Interpretation, Statistical; D006801:Humans; D006973:Hypertension / Q000188:drug therapy; D008297:Male; D008875:Middle Aged; D016032:Randomized Controlled Trials as Topic; D016540:Smoking Cessation; D016312:Treatment Refusal
D002784:Cholesterol