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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

Estimating individualised treatment effect (ITE) -- that is the causal effect of a set of variables (also called exposures, treatments, actions, policies, or interventions), referred to as \textit{composite treatments}, on a set of outcome…

Machine Learning · Computer Science 2025-12-19 Vinod Kumar Chauhan , Lei Clifton , Gaurav Nigam , David A. Clifton

Dropout is common in clinical studies, with up to half of patients leaving early due to side effects or other reasons. When dropout is informative (i.e., dependent on survival time), it introduces censoring bias, because of which treatment…

Machine Learning · Computer Science 2026-05-12 Yuxin Wang , Dennis Frauen , Jonas Schweisthal , Maresa Schröder , Stefan Feuerriegel

Accurately estimating the intra-class correlation coefficient (ICC) is crucial for adequately powering clustered randomized trials (CRTs). Challenges arise due to limited prior data on the specific outcome within the target population,…

Methodology · Statistics 2025-04-23 Chen Yang , Márcio A. Diniz , Deukwoo Kwon , Madhu Mazumdar

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

While average treatment effects (ATE) and conditional average treatment effects (CATE) provide valuable population- and subgroup-level summaries, they fail to capture uncertainty at the individual level. For high-stakes decision-making,…

Methodology · Statistics 2026-03-31 Juraj Bodik , Yaxuan Huang , Bin Yu

Controlled experiments are widely used in many applications to investigate the causal relationship between input factors and experimental outcomes. A completely randomized design is usually used to randomly assign treatment levels to…

Methodology · Statistics 2026-05-12 Yiou Li , Lulu Kang , Xiao Huang

Background: Stepped wedge cluster randomized trials (SW-CRTs) involve sequential measurements within clusters over time. Initially, all clusters start in the control condition before crossing over to the intervention on a staggered…

Methodology · Statistics 2026-01-21 Jale Basten , Katja Ickstadt , Nina Timmesfeld

We provide sufficient conditions for the identification of the heterogeneous treatment effects, defined as the conditional expectation for the differences of potential outcomes given the untreated outcome, under the nonignorable treatment…

Methodology · Statistics 2019-01-15 Keisuke Takahata , Takahiro Hoshino

When scholars suspect units are dependent on each other within clusters but independent of each other across clusters, they employ cluster-robust standard errors (CRSEs). Nevertheless, what to cluster over is sometimes unknown. For…

Methodology · Statistics 2025-11-12 Kentaro Fukumoto

We consider sequential hypothesis testing based on observations which are received in groups of random size. The observations are assumed to be independent both within and between the groups. We assume that the group sizes are independent…

Methodology · Statistics 2021-10-11 Andrey Novikov , Xóchitl Itxel Popoca-Jiménez

Comparing clusterings is central to evaluating unsupervised models, yet the many existing similarity measures can produce widely divergent, sometimes contradictory, evaluations. Clustering similarity measures are typically organized into…

Machine Learning · Statistics 2025-11-06 Alexander J. Gates

Cluster expansion (CE) is effective in modeling the stability of metallic alloys, but sometimes cluster expansions fail. Failures are often attributed to atomic relaxation in the DFT-calculated data, but there is no metric for quantifying…

Materials Science · Physics 2017-07-19 Andrew H. Nguyen , Conrad W. Rosenbrock , C. Shane Reese , Gus L. W. Hart

Pragmatic trials evaluating health care interventions often adopt cluster randomization due to scientific or logistical considerations. Previous reviews have shown that co-primary endpoints are common in pragmatic trials but infrequently…

Methodology · Statistics 2022-05-03 Siyun Yang , Mirjam Moerbeek , Monica Taljaard , Fan Li

Cluster randomized trials (CRTs) are popular in public health and in the social sciences to evaluate a new treatment or policy where the new policy is randomly allocated to clusters of units rather than individual units. CRTs often feature…

Methodology · Statistics 2019-08-16 Hyunseung Kang , Luke Keele

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.…

Cluster sampling is common in survey practice, and the corresponding inference has been predominantly design-based. We develop a Bayesian framework for cluster sampling and account for the design effect in the outcome modeling. We consider…

Methodology · Statistics 2020-06-24 Susanna Makela , Yajuan Si , Andrew Gelman

Cluster randomized trials (CRTs) are a popular design to study the effect of interventions in infectious disease settings. However, standard analysis of CRTs primarily relies on strong parametric methods, usually mixed-effect models to…

Methodology · Statistics 2021-09-23 Chan Park , Hyunseung Kang

I introduce a generic method for inference about a scalar parameter in research designs with a finite number of heterogeneous clusters where only a single cluster received treatment. This situation is commonplace in…

Econometrics · Economics 2020-10-09 Andreas Hagemann

Heterogeneous treatment effects (HTEs) are increasingly estimated using machine learning models that produce highly personalized predictions of treatment effects. In practice, however, predicted treatment effects are rarely interpreted,…

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