4.5 Article

Power and sample size for observational studies of point exposure effects

期刊

BIOMETRICS
卷 78, 期 1, 页码 388-398

出版社

WILEY
DOI: 10.1111/biom.13405

关键词

causal inference; design effect; effective sample size; Há jek estimator; inverse probability weighting; marginal structural modeling

资金

  1. NIH [R01 AI085073]

向作者/读者索取更多资源

This paper investigates the impact of inverse probability of treatment weights (IPTWs) on sample size calculations when estimating causal effects. It presents a simplified design effect approximation method and discusses practical considerations.
Inverse probability of treatment weights (IPTWs) are commonly used to control for confounding when estimating causal effects of point exposures from observational data. When planning a study that will be analyzed with IPTWs, determining the required sample size for a given level of statistical power is challenging because of the effect of weighting on the variance of the estimated causal means. This paper considers the utility of the design effect to quantify the effect of weighting on the precision of causal estimates. The design effect is defined as the ratio of the variance of the causal mean estimator divided by the variance of a naive estimator if, counter to fact, no confounding had been present and weights were not needed. A simple, closed-form approximation of the design effect is derived that is outcome invariant and can be estimated during the study design phase. Once the design effect is approximated for each treatment group, sample size calculations are conducted as for a randomized trial, but with variances inflated by the design effects to account for weighting. Simulations demonstrate the accuracy of the design effect approximation, and practical considerations are discussed.

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