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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

Case-mix heterogeneity across studies complicates meta-analyses. As a result of this, treatments that are equally effective on patient subgroups may appear to have different effectiveness on patient populations with different case mix. It…

Applications · Statistics 2021-08-25 Tat-Thang Vo , Raphael Porcher , Anna Chaimani , Stijn Vansteelandt

Recent work has focused on nonparametric estimation of conditional treatment effects, but inference has remained relatively unexplored. We propose a class of nonparametric tests for both quantitative and qualitative treatment effect…

Methodology · Statistics 2026-04-07 Oliver Dukes , Mats J. Stensrud , Riccardo Brioschi , Aaron Hudson

Efficacy testing is a cornerstone of clinical trials, ensuring that medical interventions achieve their intended therapeutic effects. Over the decades, a wide range of statistical methodologies have been developed to address the…

Other Statistics · Statistics 2026-04-13 Dhrubajyoti Ghosh , Samhita Pal

It is generally appreciated that a frequentist analysis of a group sequential trial must in order to avoid inflating type I error account for the fact that one or more interim analyses were performed. It is also to a lesser extent realised…

Methodology · Statistics 2025-12-12 Simon Bang Kristensen , Erik Thorlund Parner

App-based N-of-1 trials offer a scalable experimental design for assessing the effects of health interventions at an individual level. Their practical success depends on the strong motivation of participants, which, in turn, translates into…

Methodology · Statistics 2023-09-15 Ivana Malenica , Yongyi Guo , Kyra Gan , Stefan Konigorski

A number of biomedical problems require performing many hypothesis tests, with an attendant need to apply stringent thresholds. Often the data take the form of a series of predictor vectors, each of which must be compared with a single…

Methodology · Statistics 2014-05-13 Yi-Hui Zhou , Fred Wright

In causal inference, interference occurs when the treatment of one unit may affect the outcomes of other units. The goal of this work is to serve as a guide to the use of linear outcome modeling for estimating causal effects in settings…

Methodology · Statistics 2026-04-01 Eric Tong , Salvador V. Balkus

This paper proposes a framework that incorporates the two-way fixed effects model as a special case to conduct causal inference with a continuous treatment. Treatments are allowed to change over time and potential outcomes are dependent on…

Methodology · Statistics 2025-07-01 Zhiguo Xiao , Peikai Wu

Comparative binary outcome data are of fundamental interest in statistics and are often pooled in meta-analyses. Here we examine the simplest case where for each study there are two patient groups and a binary event of interest, giving rise…

Methodology · Statistics 2018-06-12 Rose Baker , Dan Jackson

Causal inference using observational text data is becoming increasingly popular in many research areas. This paper presents the Bayesian Topic Regression (BTR) model that uses both text and numerical information to model an outcome…

Machine Learning · Statistics 2021-09-14 Maximilian Ahrens , Julian Ashwin , Jan-Peter Calliess , Vu Nguyen

Causal inference on a population of units connected through a network often presents technical challenges, including how to account for interference. In the presence of local interference, for instance, potential outcomes of a unit depend…

Methodology · Statistics 2018-04-02 Laura Forastiere , Edoardo M. Airoldi , Fabrizia Mealli

A fundamental limitation of causal inference in observational studies is that perceived evidence for an effect might instead be explained by factors not accounted for in the primary analysis. Methods for assessing the sensitivity of a…

Methodology · Statistics 2018-09-14 Colin B. Fogarty

Recent advances in interrupted time series analysis permit characterization of a typical non-linear interruption effect through use of generalized additive models. Concurrently, advances in latent time series modeling allow efficient…

Applications · Statistics 2025-11-11 RJ Waken , Fengxian Wang , Sarah A. Eisenstein , Tim McBride , Kim Johnson , Karen Joynt-Maddox

This paper studies inference in randomized controlled trials with covariate-adaptive randomization when there are multiple treatments. More specifically, we study inference about the average effect of one or more treatments relative to…

Econometrics · Economics 2019-01-21 Federico A. Bugni , Ivan A. Canay , Azeem M. Shaikh

The delayed access to specialized psychiatric assessments and care for patients at risk of suicidal tendencies in emergency departments creates a notable gap in timely intervention, hindering the provision of adequate mental health support…

Individualized treatment rules aim to identify if, when, which, and to whom treatment should be applied. A globally aging population, rising healthcare costs, and increased access to patient-level data have created an urgent need for…

Methodology · Statistics 2019-01-04 Ying-Qi Zhao , Eric B. Laber , Yang Ning , Sumona Saha , Bruce Sands

Randomized clinical trials are the gold standard when estimating the average treatment effect. However, they are usually not a random sample from the real-world population because of the inclusion/exclusion rules. Meanwhile, observational…

Methodology · Statistics 2024-12-11 Kuan Jiang , Wenjie Hu , Shu Yang , Xinxing Lai , Xiaohua Zhou

We consider sequential treatment regimes where each unit is exposed to combinations of interventions over time. When interventions are described by qualitative labels, such as "close schools for a month due to a pandemic" or "promote this…

Machine Learning · Statistics 2024-10-31 Jialin Yu , Andreas Koukorinis , Nicolò Colombo , Yuchen Zhu , Ricardo Silva

Utilizing non-concurrent control data (NCC) in the analysis of late-entering arms in platform trials has recently received considerable attention. While incorporating NCC can lead to increased power and lower sample sizes, it might…

Methodology · Statistics 2025-09-12 Pavla Krotka , Martin Posch , Mohamed Gewily , Günter Höglinger , Marta Bofill Roig