Machine learning in policy evaluation: new tools for causal inference

N Kreif, K DiazOrdaz - arXiv preprint arXiv:1903.00402, 2019 - arxiv.org
While machine learning (ML) methods have received a lot of attention in recent years, these
methods are primarily for prediction. Empirical researchers conducting policy evaluations …

[HTML][HTML] Application of targeted maximum likelihood estimation in public health and epidemiological studies: a systematic review

MJ Smith, RV Phillips, MA Luque-Fernandez… - Annals of …, 2023 - Elsevier
Purpose The targeted maximum likelihood estimation (TMLE) statistical data analysis
framework integrates machine learning, statistical theory, and statistical inference to provide …

Targeted learning: toward a future informed by real-world evidence

S Gruber, RV Phillips, H Lee, M Ho… - Statistics in …, 2024 - Taylor & Francis
Abstract The 21st Century Cures Act of 2016 includes a provision for the US Food and Drug
Administration10. 13039/100000038 (FDA) to evaluate the potential use of Real-World …

When can nonrandomized studies support valid inference regarding effectiveness or safety of new medical treatments?

JM Franklin, R Platt, NA Dreyer… - Clinical …, 2022 - Wiley Online Library
The randomized controlled trial (RCT) is the gold standard for evaluating the causal effects
of medications. Limitations of RCTs have led to increasing interest in using real‐world …

Statistical modeling: the three cultures

A Daoud, D Dubhashi - arXiv preprint arXiv:2012.04570, 2020 - arxiv.org
Two decades ago, Leo Breiman identified two cultures for statistical modeling. The data
modeling culture (DMC) refers to practices aiming to conduct statistical inference on one or …

Selective machine learning of doubly robust functionals

Y Cui, EJ Tchetgen Tchetgen - Biometrika, 2024 - academic.oup.com
While model selection is a well-studied topic in parametric and nonparametric regression or
density estimation, selection of possibly high-dimensional nuisance parameters in …

Machine learning for improving high‐dimensional proxy confounder adjustment in healthcare database studies: An overview of the current literature

R Wyss, C Yanover, T El‐Hay, D Bennett… - … and drug safety, 2022 - Wiley Online Library
Purpose Supplementing investigator‐specified variables with large numbers of empirically
identified features that collectively serve as 'proxies' for unspecified or unmeasured factors …

Real‐world evidence of bariatric surgery and cardiovascular benefits using electronic health records data: A lesson in bias

JA Rassen, W Murk… - Diabetes, Obesity and …, 2021 - Wiley Online Library
Aim To reproduce and correct studies on bariatric surgery and the reduction in major
adverse cardiovascular events (MACE) among patients with obesity and type 2 diabetes …

Performance of modeling and balancing approach methods when using weights to estimate treatment effects in observational time-to-event settings

GWF Barros, M Eriksson, J Häggström - Plos one, 2023 - journals.plos.org
In observational studies weighting techniques are often used to overcome bias due to
confounding. Modeling approaches, such as inverse propensity score weighting, are …

Adaptive debiased machine learning using data-driven model selection techniques

L van der Laan, M Carone, A Luedtke… - arXiv preprint arXiv …, 2023 - arxiv.org
Debiased machine learning estimators for nonparametric inference of smooth functionals of
the data-generating distribution can suffer from excessive variability and instability. For this …