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Covariate adjustment for randomized controlled trials revisited
1Celgene International, Boudry, Switzerland.
Abstract:
Covariate adjustment for the estimation of treatment effect for randomized controlled trials (RCT) is a simple approach with a long history, hence, its pros and cons have been well-investigated and published in the literature. It is worthwhile to revisit this topic since recently there has been significant investigation and development on model assumptions, robustness to model mis-specification, in particular, regarding the Neyman-Rubin model and the average treatment effect estimand. This paper discusses key results of the investigation and development and their practical implication on pharmaceutical statistics. Accordingly, we recommend that appropriate covariate adjustment should be more widely used for RCTs for both hypothesis testing and estimation.
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