Efficient Targeted Maximum Likelihood Estimators for Two-Phase Design Problems

Fuente: arXiv
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Autori principali: Qiu, Sky, Gruber, Susan, Shaw, Pamela A., Williamson, Brian D., van der Laan, Mark J.
Natura: Preprint
Pubblicazione: 2026
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author Qiu, Sky
Gruber, Susan
Shaw, Pamela A.
Williamson, Brian D.
van der Laan, Mark J.
author_facet Qiu, Sky
Gruber, Susan
Shaw, Pamela A.
Williamson, Brian D.
van der Laan, Mark J.
contents In a typical two-phase design, a random sample is drawn from the target population in phase 1, during which only a subset of variables is collected. In phase 2, a subsample of the phase-1 cohort is selected, and additional variables are measured. This setting induces a coarsened data structure on the data from the second phase. We assume coarsening at random, that is, the phase-2 sampling mechanism depends only on variables fully observed. We review existing estimators, including the generalized raking estimator and the inverse probability of censoring weighted targeted maximum likelihood estimation (IPCW-TMLE) along with its extensions that also target the phase-2 sampling mechanism to improve efficiency. We further introduce a new class of estimators constructed within the TMLE framework that are asymptotically equivalent.
format Preprint
id arxiv_https___arxiv_org_abs_2602_24131
institution arXiv
publishDate 2026
record_format arxiv
spellingShingle Efficient Targeted Maximum Likelihood Estimators for Two-Phase Design Problems
Qiu, Sky
Gruber, Susan
Shaw, Pamela A.
Williamson, Brian D.
van der Laan, Mark J.
Methodology
Machine Learning
In a typical two-phase design, a random sample is drawn from the target population in phase 1, during which only a subset of variables is collected. In phase 2, a subsample of the phase-1 cohort is selected, and additional variables are measured. This setting induces a coarsened data structure on the data from the second phase. We assume coarsening at random, that is, the phase-2 sampling mechanism depends only on variables fully observed. We review existing estimators, including the generalized raking estimator and the inverse probability of censoring weighted targeted maximum likelihood estimation (IPCW-TMLE) along with its extensions that also target the phase-2 sampling mechanism to improve efficiency. We further introduce a new class of estimators constructed within the TMLE framework that are asymptotically equivalent.
title Efficient Targeted Maximum Likelihood Estimators for Two-Phase Design Problems
topic Methodology
Machine Learning
url https://arxiv.org/abs/2602.24131