Two-Step Targeted Minimum-Loss Based Estimation for Non-Negative Two-Part Outcomes

Fuente: arXiv
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Autores principales: Williams, Nicholas T., Liu, Richard, Hoffman, Katherine L., Forrest, Sarah, Rudolph, Kara E., Díaz, Iván
Formato: Preprint
Publicado: 2024
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author Williams, Nicholas T.
Liu, Richard
Hoffman, Katherine L.
Forrest, Sarah
Rudolph, Kara E.
Díaz, Iván
author_facet Williams, Nicholas T.
Liu, Richard
Hoffman, Katherine L.
Forrest, Sarah
Rudolph, Kara E.
Díaz, Iván
contents Non-negative two-part outcomes are defined as outcomes with a density function that have a zero point mass but are otherwise positive. Examples, such as healthcare expenditure and hospital length of stay, are common in healthcare utilization research. Despite the practical relevance of non-negative two-part outcomes, very few methods exist to leverage knowledge of their semicontinuity to achieve improved performance in estimating causal effects. In this paper, we develop a nonparametric two-step targeted minimum-loss based estimator (denoted as hTMLE) for non-negative two-part outcomes. We present methods for a general class of interventions referred to as modified treatment policies, which can accommodate continuous, categorical, and binary exposures. The two-step TMLE uses a targeted estimate of the intensity component of the outcome to produce a targeted estimate of the binary component of the outcome that may improve finite sample efficiency. We demonstrate the efficiency gains achieved by the two-step TMLE with simulated examples and then apply it to a cohort of Medicaid beneficiaries to estimate the effect of chronic pain and physical disability on days' supply of opioids.
format Preprint
id arxiv_https___arxiv_org_abs_2401_04263
institution arXiv
publishDate 2024
record_format arxiv
spellingShingle Two-Step Targeted Minimum-Loss Based Estimation for Non-Negative Two-Part Outcomes
Williams, Nicholas T.
Liu, Richard
Hoffman, Katherine L.
Forrest, Sarah
Rudolph, Kara E.
Díaz, Iván
Methodology
Non-negative two-part outcomes are defined as outcomes with a density function that have a zero point mass but are otherwise positive. Examples, such as healthcare expenditure and hospital length of stay, are common in healthcare utilization research. Despite the practical relevance of non-negative two-part outcomes, very few methods exist to leverage knowledge of their semicontinuity to achieve improved performance in estimating causal effects. In this paper, we develop a nonparametric two-step targeted minimum-loss based estimator (denoted as hTMLE) for non-negative two-part outcomes. We present methods for a general class of interventions referred to as modified treatment policies, which can accommodate continuous, categorical, and binary exposures. The two-step TMLE uses a targeted estimate of the intensity component of the outcome to produce a targeted estimate of the binary component of the outcome that may improve finite sample efficiency. We demonstrate the efficiency gains achieved by the two-step TMLE with simulated examples and then apply it to a cohort of Medicaid beneficiaries to estimate the effect of chronic pain and physical disability on days' supply of opioids.
title Two-Step Targeted Minimum-Loss Based Estimation for Non-Negative Two-Part Outcomes
topic Methodology
url https://arxiv.org/abs/2401.04263