Nonparametric assessment of regimen response curve estimators

In the framework of dynamic marginal structural models, regimen-response curve is a function that describes the relation between the mean outcome and the parameters in the class of decision rules. The modeling choice of the regimen-response curve is crucial in constructing an optimal regime, as a misspecified model can lead to a biased estimate with questionable causal interpretability. However, the existing literature lacks methods to evaluate and compare different working models. To address this problem, we will leverage risk to assess the "goodness-of-fit" of an imposed working model. We consider the counterfactual risk as our target parameter and derive inverse probability weighting and canonical gradients to map it to the observed data. We provide asymptotic properties of the resulting risk estimators, considering both fixed and data-dependent target parameters. We will show that the inverse probability weighting estimator can be efficient and asymptotic linear when the weight functions are estimated using a sieve-based estimator. The proposed method is implemented on the LS1 study to estimate a regimen-response curve for patients with Parkinson's disease.

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Publication Details

Journal
Biometrics
Published
2025-04-02
DOI
https://doi.org/10.1093/biomtc/ujaf066
Primary Topic
Advanced Causal Inference Techniques
Type
article
Field-Weighted Citation Impact
0.00

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article

Nonparametric assessment of regimen response curve estimators

Benjamin R. Baer, Ashkan Ertefaie, Cuong Pham
Biometrics
Advanced Causal Inference Techniques
article

Nonparametric assessment of regimen response curve estimators

Benjamin R. Baer, Ashkan Ertefaie, Cuong Pham
article en

Abstract

In the framework of dynamic marginal structural models, regimen-response curve is a function that describes the relation between the mean outcome and the parameters in the class of decision rules. The modeling choice of the regimen-response curve is crucial in constructing an optimal regime, as a misspecified model can lead to a biased estimate with questionable causal interpretability. However, the existing literature lacks methods to evaluate and compare different working models. To address this problem, we will leverage risk to assess the "goodness-of-fit" of an imposed working model. We consider the counterfactual risk as our target parameter and derive inverse probability weighting and canonical gradients to map it to the observed data. We provide asymptotic properties of the resulting risk estimators, considering both fixed and data-dependent target parameters. We will show that the inverse probability weighting estimator can be efficient and asymptotic linear when the weight functions are estimated using a sieve-based estimator. The proposed method is implemented on the LS1 study to estimate a regimen-response curve for patients with Parkinson's disease.

BiometricsVol. 81(2)
University of St Andrews (GB), University of Rochester Medical Center (US), University of Pennsylvania (US)
National Institutes of Health
Peace, Justice and strong institutions
Openalex Percentile: Top 88%
Advanced Causal Inference Techniques
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Nonparametric assessment of regimen response curve estimators — Benjamin R. Baer, Ashkan Ertefaie, et al. · Biometrics (2025) | TGRS Research Map | TGRS