Abstract

Abstract Covariate adjustment is often used in statistical analysis of randomized experiments to increase efficiency of estimators of treatment effects. In this paper, we study covariate adjustment based on the empirical and Euclidean likelihoods, and propose weighted versions that arise as natural alternatives. The weighted methods incorporate the auxiliary information that the covariates have equal means among the treatment groups due to randomization. We show that the empirical and the Euclidean likelihoods and their weighted versions are first order equivalent to Koch’s nonparametric covariance adjustment. Allowing the weights to be negative, the resulting pseudo Euclidean likelihood is equivalent to Koch’s method, and its weighted version can be viewed as a weighted version of Koch’s method. In a simulation study, we assess the finite sample properties of the proposed methods. The analysis of a clinical trial data set illustrates an application of these methods to a practical situation.

Highlights

  • Analysis of covariance is used in statistical analysis of randomized experiments to increase efficiency of estimators of treatment effects and to induce equivalence of the treatment groups generated by randomization (Snedecor and Cochran, 1980)

  • We propose covariate adjustment methods for randomized clinical trials that incorporate the auxiliary information of equal means of the covariates among the treatment groups due to randomization to Koch’s approach

  • These methods include the EL, the Euclidean likelihood (UL), and our proposed weighted versions that arise as natural alternatives: the weighted empirical likelihood (WEL) and the weighted Euclidean likelihood (WUL)

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Summary

Introduction

Analysis of covariance is used in statistical analysis of randomized experiments to increase efficiency of estimators of treatment effects and to induce equivalence (with respect to the covariates) of the treatment groups generated by randomization (Snedecor and Cochran, 1980). We propose covariate adjustment methods for randomized clinical trials that incorporate the auxiliary information of equal means of the covariates among the treatment groups due to randomization to Koch’s approach. These methods include the EL, the Euclidean likelihood (UL), and our proposed weighted versions that arise as natural alternatives: the weighted empirical likelihood (WEL) and the weighted Euclidean likelihood (WUL). Alternative approaches to covariate adjustment have been proposed to estimate treatment effects in clinical trials such as: matching, stratification, inverse probability weighting, and using propensity score as a covariate. We summarize the results developed in this paper in a conclusion and the proofs of theoretical results are provided in the appendix

Multivariate mean with auxiliary information
Application of WEL in sample surveys
Common multivariate mean
Computational aspects
Treatment effect adjusting for covariates
Unadjusted treatment effect estimation
Koch’s nonparametric covariance adjustment
Multivariate analysis of covariance
Simulation models
Simulation results
Data analysis
Findings
Conclusion
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