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A common goal in comparative effectiveness research is to estimate treatment effects on pre-specified subpopulations of patients. Though widely used in medical research, causal inference methods for such subgroup analysis remain…

The propensity score is widely used for causal inference in observational studies, but common parametric estimators can produce biased and inefficient effect estimates when model assumptions are violated. Nonparametric approaches reduce…

Methodology · Statistics 2026-04-09 Maosen Peng , Yan Li , Chong Wu , Liang Li

Inverse Probability Weighting (IPW) is widely used in empirical work in economics and other disciplines. As Gaussian approximations perform poorly in the presence of "small denominators," trimming is routinely employed as a regularization…

Econometrics · Economics 2019-05-28 Xinwei Ma , Jingshen Wang

Propensity score methods are widely used for estimating treatment effects from observational studies. A popular approach is to estimate propensity scores by maximum likelihood based on logistic regression, and then apply inverse probability…

Methodology · Statistics 2017-10-24 Zhiqiang Tan

Marginal structural models (MSMs) estimate the causal effect of a time-varying treatment in the presence of time-dependent confounding via weighted regression. The standard approach of using inverse probability of treatment weighting (IPTW)…

Methodology · Statistics 2019-08-13 Nathan Kallus , Michele Santacatterina

In observational studies with survival or time-to-event outcomes, a propensity score weighted marginal Cox proportional hazard model with the treatment variable as the only predictor is commonly used to estimate the causal marginal hazard…

Methodology · Statistics 2026-02-02 Zixian Zhao , Chengxin Yang , Fan Li

When making causal inferences, post-treatment confounders complicate analyses of time-varying treatment effects. Conditioning on these variables naively to estimate marginal effects may inappropriately block causal pathways and may induce…

Applications · Statistics 2019-04-02 Xiang Zhou , Geoffrey T. Wodtke

The inverse probability of treatment weighting (IPTW) approach is commonly used in propensity score analysis to infer causal effects in regression models. Due to oversized IPTW weights and errors associated with propensity score estimation,…

Methodology · Statistics 2021-09-02 Tenglong Li , Jordan Lawson

With the increasing availability of data from historical studies and real-world data sources, hybrid control designs that incorporate external data into the evaluation of current studies are being increasingly adopted. In these designs, it…

Methodology · Statistics 2025-06-23 Masahiro Kojima , Shunichiro Orihara , Keisuke Hanada , Tomohiro Ohigashi

There are now many options for doubly robust estimation; however, there is a concerning trend in the applied literature to believe that the combination of a propensity score and an adjusted outcome model automatically results in a doubly…

In many observational studies in social science and medicine, subjects or units are connected, and one unit's treatment and attributes may affect another's treatment and outcome, violating the stable unit treatment value assumption (SUTVA)…

Methodology · Statistics 2024-06-25 Zhaonan Qu , Ruoxuan Xiong , Jizhou Liu , Guido Imbens

Nested case-control (NCC) is a sampling method widely used for developing and evaluating risk models with expensive biomarkers on large prospective cohort studies. The biomarker values are typically obtained on a sub-cohort, consisting of…

Methodology · Statistics 2021-04-07 Qian M. Zhou , Xuan Wang , Yingye Zheng , Tianxi Cai

This paper develops methods for estimating the natural direct and indirect effects in causal mediation analysis. The efficient influence function-based estimator (EIF-based estimator) and the inverse probability weighting estimator (IPW…

Methodology · Statistics 2025-12-11 Kentaro Kawato

Personalized medicine has received increasing attention among statisticians, computer scientists, and clinical practitioners. A major component of personalized medicine is the estimation of individualized treatment rules (ITRs). Recently,…

Methodology · Statistics 2015-08-14 Xin Zhou , Nicole Mayer-Hamblett , Umer Khan , Michael R. Kosorok

We propose a new likelihood-based approach for estimation, inference and variable selection for parametric cure regression models in time-to-event analysis under random right-censoring. In this context, it often happens that some subjects…

Methodology · Statistics 2020-07-17 Kevin Burke , Valentin Patilea

In a clustered observational study, a treatment is assigned to groups and all units within the group are exposed to the treatment. We develop a new method for statistical adjustment in clustered observational studies using approximate…

Methodology · Statistics 2023-03-06 Luke Keele , Eli Ben-Michael , Lindsay Page

Inverse probability of treatment weighting (IPTW) is widely used to estimate causal effects, but guidance is limited for count exposures. It is also unclear how IPTW performs when combined with multiple imputation in this context. In this…

Methodology · Statistics 2026-03-26 Martin N. Danka , Jessica K. Bone , George B. Ploubidis , Richard J. Silverwood

Causal inference is only valid when its underlying assumptions are satisfied, one of the most central being the ignorability or unconfoundedness assumption. However, this hypothesis is often unrealistic in observational studies, as some…

In clinical trials, multiple outcomes of different priorities commonly occur as the patient's response may not be adequately characterized by a single outcome. Win statistics are appealing summary measures for between-group difference at…

Methodology · Statistics 2025-06-04 Ying Cui , Bo Huang , Gaohong Dong , Ryuji Uozumi , Lu Tian

This paper proposes a versatile covariate adjustment method that directly incorporates covariate balance in regression discontinuity (RD) designs. The new empirical entropy balancing method reweights the standard local polynomial RD…

Econometrics · Economics 2024-05-29 Jun Ma , Zhengfei Yu