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While randomized controlled trials (RCTs) are the gold standard for estimating treatment effects in medical research, there is increasing use of and interest in using real-world data for drug development. One such use case is the…

Methodology · Statistics 2021-10-11 Devin Incerti , Michael T Bretscher , Ray Lin , Chris Harbron

When considering the effect a treatment will cause in a population of interest, we often look to evidence from randomized controlled trials. In settings where multiple trials on a treatment are available, we may wish to synthesize the…

Methodology · Statistics 2023-09-08 Nicole Schnitzler , Eloise Kaizar

Random-effects meta-analyses are very commonly used in medical statistics. Recent methodological developments include multivariate (multiple outcomes) and network (multiple treatments) meta-analysis. Here we provide a new model and…

Methodology · Statistics 2017-08-16 Dan Jackson , Sylwia Bujkiewicz , Martin Law , Richard D Riley , Ian White

The conditional average treatment effect (CATE) is frequently estimated to refute the homogeneous treatment effect assumption. Under this assumption, all units making up the population under study experience identical benefit from a given…

We combine two recently proposed nonparametric difference-in-differences methods, extending them to enable the examination of treatment effect heterogeneity in the staggered adoption setting using machine learning. The proposed method,…

Econometrics · Economics 2023-10-19 Julia Hatamyar , Noemi Kreif , Rudi Rocha , Martin Huber

Counterfactual estimation over time is important in various applications, such as personalized medicine. However, time-dependent confounding bias in observational data still poses a significant challenge in achieving accurate and efficient…

Machine Learning · Computer Science 2026-03-13 Nghia D. Nguyen , Pablo Robles-Granda , Lav R. Varshney

Inferring the causal effects of time-varying treatments is often hindered by highly variable inverse propensity weights, particularly in settings with limited covariate overlap. Building on the key framework of Imai and Ratkovic (2015), we…

Methodology · Statistics 2025-11-06 Yige Li , María de los Angeles Resa , José R. Zubizarreta

We consider the task of evaluating policies of algorithmic resource allocation through randomized controlled trials (RCTs). Such policies are tasked with optimizing the utilization of limited intervention resources, with the goal of…

Artificial Intelligence · Computer Science 2023-02-07 Aditya Mate , Bryan Wilder , Aparna Taneja , Milind Tambe

There are a number of measures of direct and indirect effects in the literature. They are suitable in some cases and unsuitable in others. We describe a case where the existing measures are unsuitable and propose new suitable ones. We also…

Methodology · Statistics 2023-10-12 Jose M. Peña

Uplift modeling is an emerging machine learning approach for estimating the treatment effect at an individual or subgroup level. It can be used for optimizing the performance of interventions such as marketing campaigns and product designs.…

Machine Learning · Statistics 2020-03-27 Zhenyu Zhao , Totte Harinen

Causal mediation analysis aims at disentangling a treatment effect into an indirect mechanism operating through an intermediate outcome or mediator, as well as the direct effect of the treatment on the outcome of interest. However, the…

Econometrics · Economics 2020-05-05 Martin Huber , Lukáš Lafférs

Differences-in-differences (DiD) is a causal inference method for observational longitudinal data that assumes parallel expected potential outcome trajectories between treatment groups under the counterfactual scenario where all units…

Methodology · Statistics 2026-05-12 Michael Jetsupphasuk , Didong Li , Michael G. Hudgens

Machine learning (ML) holds great promise for improving healthcare, but it is critical to ensure that its use will not propagate or amplify health disparities. An important step is to characterize the (un)fairness of ML models - their…

Machine Learning · Computer Science 2023-08-09 Alexander Brown , Nenad Tomasev , Jan Freyberg , Yuan Liu , Alan Karthikesalingam , Jessica Schrouff

Treatment effects of stochastic policy shifts quantify differences in outcomes across counterfactual scenarios with varying treatment distributions. Stochastic policy shifts may be of interest in settings where it is unrealistic or…

Methodology · Statistics 2026-03-31 Michael Jetsupphasuk , Chenwei Fang , Didong Li , Michael G. Hudgens

Controlling bias in training datasets is vital for ensuring equal treatment, or parity, between different groups in downstream applications. A naive solution is to transform the data so that it is statistically independent of group…

Machine Learning · Computer Science 2021-06-04 Umang Gupta , Aaron M Ferber , Bistra Dilkina , Greg Ver Steeg

Matching in causal inference from observational data aims to construct treatment and control groups with similar distributions of covariates, thereby reducing confounding and ensuring an unbiased estimation of treatment effects. This…

Artificial Intelligence · Computer Science 2025-04-15 Sahil Shikalgar , Md. Noor-E-Alam

A key challenge in multi-agent reinforcement learning (MARL) lies in designing learning signals that effectively promote coordination among agents. Designing such signals requires estimating how one agent's current action affects its…

Multiagent Systems · Computer Science 2026-05-12 Haohan Yu , Jinmiao Cong , Shengzhi Wang , Lu Wang , Chanjuan Liu

Contextual sensing and delivery of digital interventions to improve health outcomes have gained significant traction in behavioral and psychiatric studies. Micro-randomized trials (MRTs) are a common experimental design for obtaining…

Methodology · Statistics 2025-04-01 Jieru Shi , Zhenke Wu , Walter Dempsey

Randomized controlled trials (RCTs) face inherent limitations, such as ethical or resource constraints, which lead to a limited number of study participants. To address these limitations, recent research endeavors have sought to incorporate…

Methodology · Statistics 2025-02-20 Peijin Wang , Hwanhee Hong , Kyungeun Jeon , Laine Elliott Thomas

In many clinical contexts, estimating effects of treatment in time-to-event data is complicated not only by confounding, censoring, and heterogeneity, but also by the presence of a cured subpopulation in which the event of interest never…

Methodology · Statistics 2026-02-06 Yuqi Li , Quinn Lanners , Matthew M. Engelhard