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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

Background: In settings where proof-of-principle trials have succeeded but the effectiveness of different forms of implementation remains uncertain, trials that not only generate information about intervention effects but also provide…

Quantitative Methods · Quantitative Biology 2017-05-16 Guy Harling , Rui Wang , Jukka-Pekka Onnela , Victor De Gruttola

Stepped wedge cluster randomized trials (SW-CRTs) with binary outcomes are increasingly used in prevention and implementation studies. Marginal models represent a flexible tool for analyzing SW-CRTs with population-averaged interpretations,…

Methodology · Statistics 2021-01-05 Fan Li , Hengshi Yu , Paul J. Rathouz , Elizabeth L. Turner , John S. Preisser

A practical limitation of cluster randomized controlled trials (cRCTs) is that the number of available clusters may be small, resulting in an increased risk of baseline imbalance under simple randomization. Constrained randomization…

Methodology · Statistics 2022-01-19 Yunji Zhou , Elizabeth L. Turner , Ryan A. Simmons , Fan Li

We consider learning personalized assignments to one of many treatment arms from a randomized controlled trial. Standard methods that estimate heterogeneous treatment effects separately for each arm may perform poorly in this case due to…

Machine Learning · Statistics 2023-11-02 Rahul Ladhania , Jann Spiess , Lyle Ungar , Wenbo Wu

While the gold standard for clinical trials is to blind all parties -- participants, researchers, and evaluators -- to treatment assignment, this is not always a possibility. When some or all of the above individuals know the treatment…

Methodology · Statistics 2020-12-22 E. J. Greene , P. Peduzzi , J. Dziura , C. Meng , M. E. Miller , T. G. Travison , D. Esserman

With increasing data availability, causal effects can be evaluated across different data sets, both randomized controlled trials (RCTs) and observational studies. RCTs isolate the effect of the treatment from that of unwanted (confounding)…

When observations are independent, formulae and software are readily available to plan and design studies of appropriate size and power to detect important associations. When observations are correlated or clustered, results obtained from…

Applications · Statistics 2021-03-15 David M. Thompson

In studies of discrimination, researchers often seek to estimate a causal effect of race or gender on outcomes. For example, in the criminal justice context, one might ask whether arrested individuals would have been subsequently charged or…

Methodology · Statistics 2022-04-06 Johann Gaebler , William Cai , Guillaume Basse , Ravi Shroff , Sharad Goel , Jennifer Hill

Prediction algorithms that quantify the expected benefit of a given treatment conditional on patient characteristics can critically inform medical decisions. Quantifying the performance of treatment benefit prediction algorithms is an…

Methodology · Statistics 2023-05-17 Yuan Xia , Paul Gustafson , Mohsen Sadatsafavi

As alternatives to the time-to-first-event analysis of composite endpoints, the {\it net benefit} (NB) and the {\it win ratio} (WR) -- which assess treatment effects using prioritized component outcomes based on clinical importance -- have…

Methodology · Statistics 2020-11-24 Roland A. Matsouaka , Adrian Coles

The purpose of many health studies is to estimate the effect of an exposure on an outcome. It is not always ethical to assign an exposure to individuals in randomised controlled trials, instead observational data and appropriate study…

In a clinical trial, the random allocation aims to balance prognostic factors between arms, preventing true confounders. However, residual differences due to chance may introduce near-confounders. Adjusting on prognostic factors is…

Methodology · Statistics 2024-11-18 Joe de Keizer , Rémi Lenain , Raphaël Porcher , Sarah Zoha , Arthur Chatton , Yohann Foucher

Estimating the Individual Treatment Effect from observational data, defined as the difference between outcomes with and without treatment or intervention, while observing just one of both, is a challenging problems in causal learning. In…

Machine Learning · Computer Science 2020-05-07 Céline Beji , Michaël Bon , Florian Yger , Jamal Atif

Given a set of pairwise comparisons, the classical ranking problem computes a single ranking that best represents the preferences of all users. In this paper, we study the problem of inferring individual preferences, arising in the context…

Machine Learning · Statistics 2015-12-18 Rui Wu , Jiaming Xu , R. Srikant , Laurent Massoulié , Marc Lelarge , Bruce Hajek

A key aspect of patient-focused drug development is identifying and measuring outcomes that are important to patients in clinical trials. Many medical conditions affect multiple symptom domains, and a consensus approach to determine the…

Methodology · Statistics 2025-10-14 David S. Robertson , Thomas Jaki

In this paper, we discuss causal inference on the efficacy of a treatment or medication on a time-to-event outcome with competing risks. Although the treatment group can be randomized, there can be confoundings between the compliance and…

Methodology · Statistics 2016-12-06 Cheng Zheng , Ran Dai , Parameswaran Hari , Mei-Jie Zhang

The target trial framework enables causal inference from longitudinal observational data by emulating randomized trials initiated at multiple time points. Precision is often improved by pooling information across trials, with standard…

Methodology · Statistics 2026-01-08 Edoardo Efrem Gervasoni , Liesbet De Bus , Stijn Vansteelandt , Oliver Dukes

We systematically investigate issues due to mis-specification that arise in estimating causal effects when (treatment) interference is informed by a network available pre-intervention, i.e., in situations where the outcome of a unit may…

Methodology · Statistics 2018-10-22 Vishesh Karwa , Edoardo M. Airoldi

In multicenter randomized trials, when effect modifiers have a different distribution across centers, comparisons between treatment groups that average over centers may not apply to any of the populations underlying the individual centers.…