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Treatment effect estimands based on win statistics, including the win ratio, win odds, and win difference are increasingly popular targets for summarizing endpoints in clinical trials. Such win estimands offer an intuitive approach for…

Methodology · Statistics 2026-02-13 Kenneth M. Lee , Xi Fang , Fan Li , Michael O. Harhay

Applied researchers are increasingly interested in whether and how treatment effects vary in randomized evaluations, especially variation not explained by observed covariates. We propose a model-free approach for testing for the presence of…

Methodology · Statistics 2014-12-17 Peng Ding , Avi Feller , Luke Miratrix

Platform trials allow treatment arms to enter and exit over time while maintaining a shared control arm, yielding concurrent and non-concurrent controls (NCC). Pooling NCC is often motivated as a strategy to improve statistical efficiency,…

Methodology · Statistics 2026-03-12 Antonio D'Alessandro , Samrachana Adhikari , Michele Santacatterina

This paper presents a randomization-based framework for estimating causal effects under interference between units, motivated by challenges that arise in analyzing experiments on social networks. The framework integrates three components:…

Statistics Theory · Mathematics 2018-06-21 Peter M. Aronow , Cyrus Samii

Causal inference on populations embedded in social networks poses technical challenges, since the typical no interference assumption frequently does not hold. Existing methods developed in the context of network interference rely upon the…

Methodology · Statistics 2024-04-12 Vanessa McNealis , Erica E. M. Moodie , Nema Dean

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

Estimating conditional average treatment effects (CATE) is challenging, especially when treatment information is missing. Although this is a widespread problem in practice, CATE estimation with missing treatments has received little…

Machine Learning · Statistics 2023-04-19 Milan Kuzmanovic , Tobias Hatt , Stefan Feuerriegel

We often seek to estimate the impact of an exposure naturally occurring or randomly assigned at the cluster-level. For example, the literature on neighborhood determinants of health continues to grow. Likewise, community randomized trials…

Methodology · Statistics 2021-07-08 Laura B. Balzer , Wenjing Zheng , Mark J. van der Laan , Maya L. Petersen

Randomized Controlled Trials are one of the pillars of science; nevertheless, they rely on hand-crafted hypotheses and expensive analysis. Such constraints prevent causal effect estimation at scale, potentially anchoring on popular yet…

Machine Learning · Computer Science 2026-01-07 Tommaso Mencattini , Riccardo Cadei , Francesco Locatello

Randomized controlled trials (RCTs) are the standard for evaluating the effectiveness of clinical interventions. To address the limitations of RCTs on real-world populations, we developed a methodology that uses a large observational…

Machine Learning · Computer Science 2024-10-21 Panayiotis Petousis , David Gordon , Susanne B. Nicholas , Alex A. T. Bui

Leveraging external controls -- relevant individual patient data under control from external trials or real-world data -- has the potential to reduce the cost of randomized controlled trials (RCTs) while increasing the proportion of trial…

Methodology · Statistics 2022-07-13 Yanyao Yi , Ying Zhang , Yu Du , Ting Ye

Multi-regional clinical trials (MRCTs) enable efficient global drug development by assessing treatment effects across regions within a single protocol. While powered for overall efficacy, MRCTs are typically not designed to provide…

This review provides a systematic overview of methods that combine covariate-based clustering of observational units (patients) with outcome models for clinical studies. We distinguish between informed-cluster models, where the outcome…

Some interventions may include important spillover or dissemination effects between study participants. For example, vaccines, cash transfers, and education programs may exert a causal effect on participants beyond those to whom individual…

Applications · Statistics 2019-02-05 Forrest W. Crawford , Olga Morozova , Ashley L. Buchanan , Donna Spiegelman

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

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

Online experiments such as Randomised Controlled Trials (RCTs) or A/B-tests are the bread and butter of modern platforms on the web. They are conducted continuously to allow platforms to estimate the causal effect of replacing system…

Machine Learning · Computer Science 2023-04-24 Olivier Jeunen

Recent research in causal inference has made important progress in addressing challenges to the external validity of trial findings. Such methods weight trial participant data to more closely resemble the distribution of effect-modifying…

Methodology · Statistics 2024-07-18 Justin M. Clark , Kollin W. Rott , James S. Hodges , Jared D. Huling

Variance reduction for causal inference in the presence of network interference is often achieved through either outcome modeling, typically analyzed under unit-randomized Bernoulli designs, or clustered experimental designs, typically…

Methodology · Statistics 2026-01-19 Matthew Eichhorn , Samir Khan , Johan Ugander , Christina Lee Yu

The study of causal effects in the presence of unmeasured spatially varying confounders has garnered increasing attention. However, a general framework for identifiability, which is critical for reliable causal inference from observational…

Methodology · Statistics 2026-02-27 Tommy Tang , Xinran Li , Bo Li
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