"Within-trial" prognostic score adjustment is targeted maximum likelihood estimation
Abstract
Adjustment for ``super'' or ``prognostic'' composite covariates has become more popular in randomized trials recently. These prognostic covariates are often constructed from historical data by fitting a predictive model of the outcome on the raw covariates. A natural question that we have been asked by applied researchers is whether this can be done without the historical data: can the prognostic covariate be constructed or derived from the trial data itself, possibly using different folds of the data, before adjusting for it? Here we clarify that such ``within-trial'' prognostic adjustment is nothing more than a form of targeted maximum likelihood estimation (TMLE), a well-studied procedure for optimal inference. We demonstrate the equivalence with a simulation study and discuss the pros and cons of within-trial prognostic adjustment (standard efficient estimation) relative to standard TMLE and standard prognostic adjustment with historical data.
Cite
@article{arxiv.2507.23446,
title = {"Within-trial" prognostic score adjustment is targeted maximum likelihood estimation},
author = {Emilie Højbjerre-Frandsen and Alejandro Schuler},
journal= {arXiv preprint arXiv:2507.23446},
year = {2025}
}
Comments
14 pages, 2 figures