In randomized experiments, the intention-to-treat parameter is defined as the difference in expected outcomes between groups assigned to treatment and control arms. There is a large literature focusing on how (possibly misspecified) working models can sometimes exploit baseline covariate measurements to gain precision, although covariate adjustment is not strictly necessary. In Rubin and van der Laan (2008), we proposed the technique of empirical efficiency maximization for improving estimation by forming nonstandard fits of such working models. Considering a more realistic randomization scheme than in our original article, we suggest a new class of working models for utilizing covariate information, show our method can be implemented by adding weights to standard regression algorithms, and demonstrate benefits over existing estimators through numerical asymptotic efficiency calculations and simulations.
Clinical Trials | Statistical Methodology | Statistical Theory
Rubin, Daniel B. and van der Laan, Mark J., "Covariate Adjustment for the Intention-to-Treat Parameter with Empirical Efficiency Maximization" (February 2008). U.C. Berkeley Division of Biostatistics Working Paper Series. Working Paper 229.