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Compliance subsampling designs for comparative research: estimation and optimal planning. (English) Zbl 1209.62172

Summary: For studies with treatment noncompliance, analyses have been developed recently to better estimate treatment efficacy. However, the advantage and cost of measuring compliance data have implications on the study design that have not been as systematically explored. In order to estimate better treatment efficacy with lower cost, we propose a new class of compliance subsampling (CSS) designs where, after subjects are assigned treatment, compliance behavior is measured for only subgroups of subjects. The sizes of the subsamples are allowed to relate to the treatment assignment, the assignment probability, the total sample size, the anticipated distributions of outcome and compliance, and the cost parameters of the study. The CSS design methods relate to prior work (i) on two-phase designs in which a covariate is subsampled and (ii) on causal inference because the subsampled postrandomization compliance behavior is not the true covariate of interest. For each CSS design, we develop efficient estimation of treatment efficacy under binary outcome and all-or-none observed compliance. Then we derive a minimal cost CSS design that achieves a required precision for estimating treatment efficacy. We compare the properties of the CSS design to those of conventional protocols in a study of patient choices for medical care at the end of life.

MSC:

62K05 Optimal statistical designs
62N02 Estimation in survival analysis and censored data
62P10 Applications of statistics to biology and medical sciences; meta analysis
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