In behavioral medicine trials, such as smoking cessation trials, two or more active treatments are often compared. Noncompliance by some subjects with their assigned treatment poses a challenge to the data analyst. Causal parameters of interest might include those defined by subpopulations based on their potential compliance status under each assignment, using the principal stratification framework (e.g., causal effect of new therapy compared to standard therapy among subjects that would comply with either intervention). Even if subjects in one arm do not have access to the other treatment(s), the causal effect of each treatment typically can only be identified from the outcome, randomization and compliance data within certain bounds. We propose to use additional information -- compliance-predictive covariates -- to help identify the causal effects. Our approach is to specify marginal compliance models conditional on covariates within each arm of the study. Parameters from these models can be identified from the data. We then link the two compliance models through an association model that depends on a parameter that is not identifiable, but has a meaningful interpretation; this parameter forms the basis for a sensitivity analysis. We demonstrate the benefit of utilizing covariate information in both a simulation study and in an analysis of data from a smoking cessation trial.


Biostatistics | Categorical Data Analysis | Clinical Trials | Epidemiology | Statistical Models