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In large observational studies, the case-cohort design is commonly used to reduce the cost associated with covariate measurement. For survival outcomes, literature has suggested that the restricted mean survival time (RMST) be a more…

Methodology · Statistics 2026-05-08 Andy Ni , Wei-En Lu , Bo Lu

An empirical investigation of the interaction of sample size and discretization - in this case the entropy-based method CAIM (Class-Attribute Interdependence Maximization) - was undertaken to evaluate the impact and potential bias…

Machine Learning · Statistics 2012-01-09 Casey Bennett

The problem of estimating the ratio of the means of a two-component Poisson mixture model is considered, when each component is subject to zero-inflation, i.e., excess zero counts. The. resulting {\it zero-inflated Poisson mixture (ZIPM)…

Statistics Theory · Mathematics 2022-03-31 Michael D. Perlman

Compressed sensing (sparse signal recovery) has been a popular and important research topic in recent years. By observing that natural signals are often nonnegative, we propose a new framework for nonnegative signal recovery using…

Methodology · Statistics 2013-10-04 Ping Li , Cun-Hui Zhang , Tong Zhang

We propose a new approach for sparse regression and marginal testing, for data with correlated features. Our procedure first clusters the features, and then chooses as the cluster prototype the most informative feature in that cluster. Then…

Methodology · Statistics 2015-03-16 Stephen Reid , Robert Tibshirani

This paper develops power and sample size formulas for causal inference with time-to-event outcomes. The target estimand is the marginal hazard ratio: the coefficient of a marginal structural Cox proportional hazard model with treatment as…

Methodology · Statistics 2026-05-19 Chengxin Yang , Bo Liu , Fan Li

Risk-adjusted quality measures are used to evaluate healthcare providers while controlling for factors beyond their control. Existing healthcare provider profiling approaches typically assume that the risk adjustment is perfect and the…

Applications · Statistics 2022-07-26 Nicholas Hartman , Joseph M. Messana , Jian Kang , Abhijit S. Naik , Tempie H. Shearon , Kevin He

Treatment effect estimates are often available from randomized controlled trials as a single average treatment effect for a certain patient population. Estimates of the conditional average treatment effect (CATE) are more useful for…

Methodology · Statistics 2023-09-12 Wouter A. C. van Amsterdam , Rajesh Ranganath

Sequential Multiple-Assignment Randomized Trials (SMARTs) play an increasingly important role in psychological and behavioral health research. This experimental approach enables researchers to answer scientific questions about how to…

Methodology · Statistics 2023-06-21 John J. Dziak , Daniel Almirall , Walter Dempsey , Catherine Stanger , Inbal Nahum-Shani

The micro-randomized trial (MRT) is a new experimental design which allows for the investigation of the proximal effects of a "just-in-time" treatment, often provided via a mobile device as part of a mobile health intervention. As with a…

Methodology · Statistics 2020-08-07 Nicholas J. Seewald , Ji Sun , Peng Liao

Simulation offers a simple and flexible way to estimate the power of a clinical trial when analytic formulae are not available. The computational burden of using simulation has, however, restricted its application to only the simplest of…

Binary endpoints are common in clinical trials and conditional odds ratios have traditionally been used to assess treatment effects. However, the interpretation of odds ratios is difficult, they are non-collapsible and rely on strong…

Methodology · Statistics 2026-05-20 Martin Schnuerch , Alex Ocampo , Klaus Kähler Holst , Christian Stock

Stepped wedge cluster-randomized trial (CRTs) designs randomize clusters of individuals to intervention sequences, ensuring that every cluster eventually transitions from a control period to receive the intervention under study by the end…

Meta-analyses frequently include trials that report multiple effect sizes based on a common set of study participants. These effect sizes will generally be correlated. Cluster-robust variance-covariance estimators are a fruitful approach…

Methodology · Statistics 2022-03-07 Thilo Welz , Wolfgang Viechtbauer , Markus Pauly

Meta-analysis is a well-established method for integrating results from several independent studies to estimate a common quantity of interest. However, meta-analysis is prone to selection bias, notably when particular studies are…

Applications · Statistics 2024-11-20 Layna Charlie Dennett , Antony Overstall , Dankmar Boehning

Uplift modeling and Heterogeneous Treatment Effect (HTE) estimation aim at predicting the causal effect of an action, such as a medical treatment or a marketing campaign on a specific individual. In this paper, we focus on data from…

Machine Learning · Computer Science 2024-12-16 Krzysztof Rudaś , Szymon Jaroszewicz

Hybrid controlled trials (HCTs), which augment randomized controlled trials (RCTs) with external controls (ECs), are increasingly receiving attention as a way to address limited power, slow accrual, and ethical concerns in clinical…

Methodology · Statistics 2025-05-02 Jiajun Liu , Ke Zhu , Shu Yang , Xiaofei Wang

Adaptive sample size re-estimation, early stopping, and trial re-design at interim analyses can reduce expected sample sizes in randomised trials. Cluster randomised trials, in which groups of participants are randomly allocated to…

Methodology · Statistics 2026-03-09 Samuel I. Watson , James Martin

The most widely used internal measure for clustering evaluation is the silhouette coefficient, whose naive computation requires a quadratic number of distance calculations, which is clearly unfeasible for massive datasets. Surprisingly,…

Data Structures and Algorithms · Computer Science 2021-01-21 Federico Altieri , Andrea Pietracaprina , Geppino Pucci , Fabio Vandin

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