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Compliance Subsampling Designs for Comparative Research: Estimation and Optimal Planning
Constantine E. Frangakis and Stuart G. Baker
Vol. 57, No. 3 (Sep., 2001), pp. 899-908
Published by: International Biometric Society
Stable URL: http://www.jstor.org/stable/3068431
Page Count: 10
You can always find the topics here!Topics: Cost estimates, Compliance costs, Cost efficiency, Physicians, Treatment compliance, Tax noncompliance, Health outcomes, Research design, Statistical estimation, Biometrics
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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.
Biometrics © 2001 International Biometric Society