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Evaluation of clinical prediction models across multiple clusters, whether centers or datasets, is becoming increasingly common. A comprehensive evaluation includes an assessment of the agreement between the estimated risks and the observed…

Methodology · Statistics 2026-04-22 Lasai Barreñada , Bavo D. C. Campo , Laure Wynants , Ben Van Calster

In cancer research, clustering techniques are widely used for exploratory analyses and dimensionality reduction, playing a critical role in the identification of novel cancer subtypes, often with direct implications for patient management.…

The traditional model specification of stepped-wedge cluster-randomized trials assumes a homogeneous treatment effect across time while adjusting for fixed-time effects. However, when treatment effects vary over time, the constant effect…

We consider a potential outcomes model in which interference may be present between any two units but the extent of interference diminishes with spatial distance. The causal estimand is the global average treatment effect, which compares…

Methodology · Statistics 2022-09-16 Michael P. Leung

Randomized experimentation (also known as A/B testing or bucket testing) is widely used in the internet industry to measure the metric impact obtained by different treatment variants. A/B tests identify the treatment variant showing the…

Sample size calculations can be challenging with skewed continuous outcomes in randomized controlled trials (RCTs). Standard t-test-based calculations may require data transformation, which may be difficult before data collection.…

We consider the design and analysis of multi-factor experiments using fractional factorial and incomplete designs within the potential outcome framework. These designs are particularly useful when limited resources make running a full…

Methodology · Statistics 2022-01-31 Nicole E. Pashley , Marie-Abele C. Bind

This paper studies experimental designs for estimation and inference on policies with spillover effects. Units are organized into a finite number of large clusters and interact in unknown ways within each cluster. First, we introduce a…

Econometrics · Economics 2024-05-06 Davide Viviano , Jess Rudder

Cluster-randomized trials (CRTs) involve randomizing entire groups of participants -- called clusters -- to treatment arms but are often comprised of a limited or fixed number of available clusters. While covariate adjustment can account…

Methodology · Statistics 2022-11-29 Angela Y. Zhu , Nandita Mitra , Karla Hemming , Michael O. Harhay , Fan Li

The presence of interference, where the outcome of an individual may depend on the treatment assignment and behavior of neighboring nodes, can lead to biased causal effect estimation. Current approaches to network experiment design focus on…

Machine Learning · Computer Science 2024-05-22 Zahra Fatemi , Jean Pouget-Abadie , Elena Zheleva

Ever since the seminal work of R. A. Fisher and F. Yates, factorial designs have been an important experimental tool to simultaneously estimate the effects of multiple treatment factors. In factorial designs, the number of treatment…

Methodology · Statistics 2024-03-21 Lei Shi , Jingshen Wang , Peng Ding

Micro-randomized trials (MRTs) are widely used to assess the marginal and moderated effect of mobile health (mHealth) treatments delivered via mobile devices. In many applications, the mHealth treatments are categorical with multiple levels…

Methodology · Statistics 2025-04-23 Jeremy Lin , Tianchen Qian

Randomized saturation designs are two-stage experiments: they first randomly assign treatment probabilities over the clusters and then randomly assign the treatment to the units within the clusters. The existing literature on randomized…

Methodology · Statistics 2026-05-29 Sizhu Lu , Lei Shi , Peng Ding

This paper studies the design of cluster experiments to estimate the global treatment effect in the presence of network spillovers. We provide a framework to choose the clustering that minimizes the worst-case mean-squared error of the…

Econometrics · Economics 2025-01-29 Davide Viviano , Lihua Lei , Guido Imbens , Brian Karrer , Okke Schrijvers , Liang Shi

Adaptive interventions, aka dynamic treatment regimens, are sequences of pre-specified decision rules that guide the provision of treatment for an individual given information about their baseline and evolving needs, including in response…

Methodology · Statistics 2024-05-02 Wenchu Pan , Daniel Almirall , Amy M. Kilbourne , Andrew Quanbeck , Lu Wang

Group sequential designs drive innovation in clinical, industrial, and corporate settings. Early stopping for failure in sequential designs conserves experimental resources, whereas early stopping for success accelerates access to improved…

Methodology · Statistics 2025-11-27 Luke Hagar , Shirin Golchi , Marina B. Klein

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

Across research disciplines, cluster randomized trials (CRTs) are commonly implemented to evaluate interventions delivered to groups of participants, such as communities and clinics. Despite advances in the design and analysis of CRTs,…

In the analysis of cluster randomized trials (CRTs), previous work has defined two meaningful estimands: the individual-average treatment effect (iATE) and cluster-average treatment effect (cATE) estimand, to address individual and…

Recently, methodology was presented to facilitate the incorporation of interim analyses in stepped-wedge (SW) cluster randomised trials (CRTs). Here, we extend this previous discussion. We detail how the stopping boundaries, allocation…

Methodology · Statistics 2018-03-28 Michael Grayling , David Robertson , James Wason , Adrian Mander