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Related papers: An Estimator-Robust Design for Augmenting Randomiz…

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Randomized controlled trials often suffer from interference, a violation of the Stable Unit Treatment Values Assumption (SUTVA) in which a unit's treatment assignment affects the outcomes of its neighbors. This interference causes bias in…

Methodology · Statistics 2025-02-06 Vydhourie Thiyageswaran , Tyler McCormick , Jennifer Brennan

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 control trials (RCTs) have been the gold standard to evaluate the effectiveness of a program, policy, or treatment on an outcome of interest. However, many RCTs assume that study participants are willing to share their…

Applications · Statistics 2021-12-07 Manjusha Kancharla , Hyunseung Kang

We develop a method for hybrid analyses that uses external controls to augment internal control arms in randomized controlled trials (RCT) where the degree of borrowing is determined based on similarity between RCT and external control…

Methodology · Statistics 2023-05-11 Evan Kwiatkowski , Jiawen Zhu , Xiao Li , Herbert Pang , Grazyna Lieberman , Matthew A. Psioda

Several recently developed methods have the potential to harness machine learning in the pursuit of target quantities inspired by causal inference, including inverse weighting, doubly robust estimating equations and substitution estimators…

Given an (optimal) dynamic treatment rule, it may be of interest to evaluate that rule -- that is, to ask the causal question: what is the expected outcome had every subject received treatment according to that rule? In this paper, we study…

Methodology · Statistics 2021-10-27 Lina Montoya , Jennifer Skeem , Mark van der Laan , Maya Petersen

Background: Target trial emulation (TTE) that applies trial design principles to improve the analysis of non-randomized studies is increasingly being used. Applications of TTE to emulate cluster randomized trials (RCTs) have been limited.…

Applications · Statistics 2025-12-17 Shomoita Alam , Nathaniel Dyrkton , Susan Shepherd , Ibrahim Sana , Kevin Phelan , Jay JH Park

We study randomized experiments in bipartite systems where only a subset of treatment-side units are eligible for assignment while all units continue to interact, generating interference. We formalize eligibility-constrained bipartite…

Methodology · Statistics 2025-11-17 Albert Tan , Mohsen Bayati , James Nordlund , Roman Istomin

Common causal estimands include the average treatment effect (ATE), the average treatment effect of the treated (ATT), and the average treatment effect on the controls (ATC). Using augmented inverse probability weighting methods, parametric…

Methodology · Statistics 2022-09-23 Roland A. Matsouaka , Yi Liu , Yunji Zhou

The Average Treatment Effect (ATE) is a foundational metric in causal inference, widely used to assess intervention efficacy in randomized controlled trials (RCTs). However, in many applications -- particularly in healthcare -- this static…

Machine Learning · Computer Science 2025-07-23 Julianna Piskorz , Krzysztof Kacprzyk , Harry Amad , Mihaela van der Schaar

Clinical randomized controlled trials (RCTs) collect hundreds of measurements spanning various metric types (e.g., laboratory tests, cognitive/motor assessments, etc.) across 100s-1000s of subjects to evaluate the effect of a treatment, but…

Machine Learning · Computer Science 2024-06-25 Sayeri Lala , Niraj K. Jha

Existing statistical methods for the analysis of micro-randomized trials (MRTs) are designed to estimate causal excursion effects using data from a single MRT. In practice, however, researchers can often find previous MRTs that employ…

Methodology · Statistics 2025-05-13 Easton Huch , Inbal Nahum-Shani , Lindsey Potter , Cho Lam , David W. Wetter , Walter Dempsey

Randomized trials are typically designed to detect average treatment effects but often lack the statistical power to uncover individual-level treatment effect heterogeneity, limiting their value for personalized decision-making. To address…

Machine Learning · Statistics 2026-03-19 Rickard Karlsson , Piersilvio De Bartolomeis , Issa J. Dahabreh , Jesse H. Krijthe

In modern statistics, interests shift from pursuing the uniformly minimum variance unbiased estimator to reducing mean squared error (MSE) or residual squared error. Shrinkage based estimation and regression methods offer better prediction…

Methodology · Statistics 2025-02-25 Tianyu Zhan , Haoda Fu , Jian Kang

PURPOSE Providing rapid answers and early acces to patients to innovative treatments without randomized clinical trial (RCT) is growing, with benefit estimated from single-arm trials. This has become common in oncology, impacting the…

Stepped-wedge cluster-randomized trials (SW-CRTs) are widely used in healthcare and implementation science, providing an ethical advantage by ensuring all clusters eventually receive the intervention. The staggered rollout of treatment…

Methodology · Statistics 2026-04-03 Xi Fang , Xueqi Wang , Patrick J. Heagerty , Bingkai Wang , Fan Li

Cluster-randomized trials (CRTs) are widely used to evaluate group-level interventions and increasingly collect multiple outcomes capturing complementary dimensions of benefit and risk. Investigators often seek a single global summary of…

Methodology · Statistics 2026-01-22 Xinyuan Chen , Fan Li

Robust decision making involves making decisions in the presence of uncertainty and is often used in critical domains such as healthcare, supply chains, and finance. Causality plays a crucial role in decision-making as it predicts the…

Methodology · Statistics 2025-07-23 Saideep Nannapaneni , Joseph Sakaya , Kyle Caron , Pedro HM Albuquerque , Zaid Tashman

Study populations are typically sampled from limited points in space and time, and marginalized groups are underrepresented. To assess the external validity of randomized and observational studies, we propose and evaluate the worst-case…

Machine Learning · Statistics 2022-02-04 Sookyo Jeong , Hongseok Namkoong

This project considers how one might augment a limited amount of data from randomized controlled trial (RCT) with more plentiful data from an observational database (ODB), in order to estimate a causal effect. In our motivating setting, the…

Methodology · Statistics 2018-10-23 Evan Rosenman , Art B. Owen , Michael Baiocchi , Hailey Banack