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In this paper, a Bayesian approach is developed for simultaneously comparing multiple experimental treatments with a common control treatment in an exploratory clinical trial. The sample size is set to ensure that, at the end of the study,…

Statistics Theory · Mathematics 2019-11-14 John Whitehead , Faye Cleary , Amanda Turner

The use of case-crossover designs has become widespread in epidemiological and medical investigations of transient associations. However, the most popular reference-select strategy, the time-stratified schema, is not a suitable solution for…

Methodology · Statistics 2020-01-22 Xiaoming Wang , Sukun Wang

The recently published ICH E9 addendum on estimands in clinical trials provides a framework for precisely defining the treatment effect that is to be estimated, but says little about estimation methods. Here we report analyses of a clinical…

Applications · Statistics 2023-09-25 Camila Olarte Parra , Rhian M. Daniel , David Wright , Jonathan W. Bartlett

We define causal estimands for experiments on single time series, extending the potential outcome framework to dealing with temporal data. Our approach allows the estimation of some of these estimands and exact randomization based p-values…

Methodology · Statistics 2020-02-17 Iavor Bojinov , Neil Shephard

External controls (ECs) from historical trials or real-world data have gained increasing attention as a way to augment hybrid and single-arm trials, especially when balanced randomization is infeasible. While most existing work has focused…

Methodology · Statistics 2025-12-15 Yujing Gao , Xiang Zhang , Shu Yang

Cluster-randomized experiments are widely used due to their logistical convenience and policy relevance. To analyze them properly, we must address the fact that the treatment is assigned at the cluster level instead of the individual level.…

Methodology · Statistics 2021-08-06 Fangzhou Su , Peng Ding

It is often of interest in observational studies to measure the causal effect of a treatment on time-to-event outcomes. In a medical setting, observational studies commonly involve patients who initiate medication therapy and others who do…

Methodology · Statistics 2019-10-07 Reagan Mozer , Mark E. Glickman

Area-specific causal inference is important in many policy and survey applications, where the goal is to evaluate treatment effects for small geographic or demographic domains. Existing causal small area estimation methods, however,…

Statistics Theory · Mathematics 2026-05-06 Tsubasa Ito , Shonosuke Sugasawa

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

Randomized experiments on social networks pose statistical challenges, due to the possibility of interference between units. We propose new methods for estimating attributable treatment effects in such settings. The methods do not require…

Methodology · Statistics 2015-10-13 David S. Choi

When designing and evaluating an experiment or observational study, it is useful to have a realistic hypothesis regarding the average treatment effect. We present an approach to conceptualizing this average by first considering a…

Methodology · Statistics 2026-04-10 Andrew Gelman , Amy Krefman , Lauren Kennedy , Jessica Hullman

We study targeted maximum likelihood estimation (TMLE) of the average treatment effect in a semiparametric regression model whose mean function is indexed by a finite-dimensional parameter, while the additive error distribution is left…

Methodology · Statistics 2026-04-20 Mijeong Kim

In this paper, we introduce a unified estimator to analyze various treatment effects in causal inference, including but not limited to the average treatment effect (ATE) and the quantile treatment effect (QTE). The proposed estimator is…

Methodology · Statistics 2025-03-31 Kuan-Hsun Wu , Li-Pang Chen

The treatment allocation mechanism in a randomized clinical trial can be optimized by maximizing the nonparametric efficiency bound for a specific measure of treatment effect. Optimal treatment allocations which may or may not depend on…

Methodology · Statistics 2025-05-23 Wei Zhang , Zhiwei Zhang , Aiyi Liu

Sustained treatment strategies are common in many domains, particularly in medicine, where many treatment are delivered repeatedly over time. The effects of adherence to a treatment strategy throughout follow-up are often more relevant to…

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

Test-time adaptation (TTA) aims to improve model generalizability when test data diverges from training distribution, offering the distinct advantage of not requiring access to training data and processes, especially valuable in the context…

Machine Learning · Computer Science 2024-02-28 Yige Yuan , Bingbing Xu , Liang Hou , Fei Sun , Huawei Shen , Xueqi Cheng

To draw real-world evidence about the comparative effectiveness of multiple time-varying treatments on patient survival, we develop a joint marginal structural survival model and a novel weighting strategy to account for time-varying…

Methodology · Statistics 2023-08-08 Liangyuan Hu , Jiayi Ji , Himanshu Joshi , Erick Scott , Fan Li

We investigate the task of estimating the conditional average causal effect of treatment-dosage pairs from a combination of observational data and assumptions on the causal relationships in the underlying system. This has been a…

Machine Learning · Computer Science 2022-05-31 Alexis Bellot , Anish Dhir , Giulia Prando

Many popular methods for building confidence intervals on causal effects under high-dimensional confounding require strong "ultra-sparsity" assumptions that may be difficult to validate in practice. To alleviate this difficulty, we here…

Statistics Theory · Mathematics 2019-05-06 Jelena Bradic , Stefan Wager , Yinchu Zhu