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We evaluate the performance of targeted maximum likelihood estimation (TMLE) for estimating the average treatment effect in missing data scenarios under varying levels of positivity violations. We employ model- and design-based simulations,…

统计方法学 · 统计学 2026-05-12 Christoph Wiederkehr , Christian Heumann , Michael Schomaker

Public policy-makers use cost-effectiveness analyses (CEA) to decide which health and social care interventions to provide. Appropriate methods have not been developed for handling missing data in complex settings, such as for CEA that use…

统计方法学 · 统计学 2012-06-27 Karla Diaz-Ordaz , Michael G. Kenward , Richard Grieve

The time series cluster kernel (TCK) provides a powerful tool for analysing multivariate time series subject to missing data. TCK is designed using an ensemble learning approach in which Bayesian mixture models form the base models. Because…

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…

统计方法学 · 统计学 2022-11-29 Angela Y. Zhu , Nandita Mitra , Karla Hemming , Michael O. Harhay , Fan Li

Cluster-level dynamic treatment regimens can be used to guide sequential, intervention or treatment decision-making at the cluster level in order to improve outcomes at the individual or patient-level. In a cluster-level DTR, the…

统计方法学 · 统计学 2016-07-15 Timothy NeCamp , Amy Kilbourne , Daniel Almirall

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…

统计方法学 · 统计学 2022-01-19 Yunji Zhou , Elizabeth L. Turner , Ryan A. Simmons , Fan Li

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…

统计方法学 · 统计学 2025-11-12 Kentaro Fukumoto

Background: When planning a cluster randomized trial, evaluators often have access to an enumerated cohort representing the target population of clusters. Practicalities of conducting the trial, such as the need to oversample clusters with…

统计方法学 · 统计学 2024-09-19 Sarah E. Robertson , Jon A. Steingrimsson , Issa J. Dahabreh

Cluster-randomized trials (CRTs) are widely used to evaluate interventions delivered at the clinic, practice, or community level. Although standard analyses typically target average treatment effects, such summaries mask potentially…

统计方法学 · 统计学 2026-04-16 Changjun Li , Xi Fang , Michael O. Harhay , Andrew B. Forbes , F. Perry Wilson , Guangyu Tong , Fan Li

Clustering is widely used in unsupervised learning to find homogeneous groups of observations within a dataset. However, clustering mixed-type data remains a challenge, as few existing approaches are suited for this task. This study…

机器学习 · 统计学 2025-11-26 Badih Ghattas , Alvaro Sanchez San-Benito

Randomized trials are viewed as the benchmark for assessing causal effects of treatments on outcomes of interest. Nonetheless, challenges such as measurement error can undermine the standard causal assumptions for randomized trials. In…

统计方法学 · 统计学 2025-08-27 Dane Isenberg , Nandita Mitra , Steven C. Marcus , Rinad S. Beidas , Kristin A. Linn

Unlike the commonly used parametric regression models such as mixed models, that can easily violate the required statistical assumptions and result in invalid statistical inference, target maximum likelihood estimation allows more realistic…

应用统计 · 统计学 2020-06-17 Chi Zhang , Jennifer Ahern , Mark J. van der Laan

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…

统计方法学 · 统计学 2026-03-09 Samuel I. Watson , James Martin

In empirical work it is common to estimate parameters of models and report associated standard errors that account for "clustering" of units, where clusters are defined by factors such as geography. Clustering adjustments are typically…

统计理论 · 数学 2022-09-21 Alberto Abadie , Susan Athey , Guido Imbens , Jeffrey Wooldridge

Micro-randomized trials (MRTs) are increasingly utilized for optimizing mobile health interventions, with the causal excursion effect (CEE) as a central quantity for evaluating interventions under policies that deviate from the experimental…

统计方法学 · 统计学 2024-11-19 Jiaxin Yu , Tianchen Qian

Inferring treatment effects on a survival time outcome based on data from an observational study is challenging due to the presence of censoring and possible confounding. An additional challenge occurs when a unit's treatment affects the…

统计方法学 · 统计学 2025-08-13 Chanhwa Lee , Donglin Zeng , Michael Emch , John D. Clemens , Michael G. Hudgens

In longitudinal panels and other regression models with unobserved effects, fixed effects estimation is often paired with cluster-robust variance estimation (CRVE) in order to account for heteroskedasticity and un-modeled dependence among…

统计方法学 · 统计学 2022-11-08 James E. Pustejovsky , Elizabeth Tipton

In cluster-randomized trials (CRTs), entire clusters of individuals are randomized to treatment, and outcomes within a cluster are typically correlated. While frequentist approaches are standard practice for CRT analysis, Bayesian methods…

统计方法学 · 统计学 2025-11-27 Ruyi Liu , Joshua L. Warren , Yuki Ohnishi , Donna Spiegelman , Liangyuan Hu , Fan Li

Misclassification in binary outcomes is not uncommon and statistical methods to investigate its impact on policy-driving study results are lacking. While misclassifying binary outcomes is a statistically ubiquitous phenomena, we focus on…

The conclusions of randomized controlled trials may be biased when the outcome of one unit depends on the treatment status of other units, a problem known as interference. In this work, we study interference in the setting of one-sided…

统计方法学 · 统计学 2022-11-01 Jennifer Brennan , Vahab Mirrokni , Jean Pouget-Abadie