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Three critical issues for causal inference that often occur in modern, complicated experiments are interference, treatment nonadherence, and missing outcomes. A great deal of research efforts has been dedicated to developing causal…

Methodology · Statistics 2023-04-06 Yuki Ohnishi , Arman Sabbaghi

We describe the Bedside Patient Rescue (BPR) project, the goal of which is risk prediction of adverse events for non-ICU patients using ~200 variables (vitals, lab results, assessments, ...). There are several missing predictor values for…

The parallel cluster randomized trial with baseline (PB-CRT) is a common variant of the standard parallel cluster randomized trial (P-CRT). We define two natural estimands in the context of PB-CRTs with informative cluster sizes, the…

Methodology · Statistics 2025-05-01 Kenneth Menglin Lee , Fan Li

We extend Fisher's randomization test (FRT) to test conditional independence between observed outcomes and treatments given covariates in both randomized experiments and observational studies, with no restriction on the variable type of…

Methodology · Statistics 2025-06-12 Zhen Zhong

Randomised controlled trials in reproductive medicine are often subject to outcome truncation, where study outcomes are only defined in a subset of participants. Examples include birthweight (measurable only in the subgroup of participants…

Not only does mobile health technology enable researchers to track changes in multiple longitudinal outcomes of interest and to record the occurrence of health-related events over time, but it also allows for the delivery of repeated…

Construction of just-in-time adaptive interventions, such as prompts delivered by mobile apps to promote and maintain behavioral change, requires knowledge about time-varying moderated effects to inform when and how we deliver intervention…

Methodology · Statistics 2022-12-06 Jieru Shi , Zhenke Wu , Walter Dempsey

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…

Methodology · Statistics 2024-09-19 Sarah E. Robertson , Jon A. Steingrimsson , Issa J. Dahabreh

We propose a novel nonparametric Bayesian IRT model in this paper by introducing the clustering effect at question level and further assume heterogeneity at examinee level under each question cluster, characterized by the mixture of…

Methodology · Statistics 2022-11-23 Tianyu Pan , Weining Shen , Clintin P. Davis-Stober , Guanyu Hu

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

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…

Methodology · Statistics 2024-11-19 Jiaxin Yu , Tianchen Qian

A key question in causal inference analyses is how to find subgroups with elevated treatment effects. This paper takes a machine learning approach and introduces a generative model, Causal Rule Sets (CRS), for interpretable subgroup…

Artificial Intelligence · Computer Science 2021-05-21 Tong Wang , Cynthia Rudin

Traditional statistical inference in cluster randomized trials typically invokes the asymptotic theory that requires the number of clusters to approach infinity. In this article, we propose an alternative conformal causal inference…

Methodology · Statistics 2024-10-03 Bingkai Wang , Fan Li , Mengxin Yu

The paper considers two different designs; a two-arm superiority cluster randomised controlled trial (RCT) with a continuous outcome, and a twoarm superiority cluster RCT with a binary outcome. From a Bayesian perspective, for the analysis…

Methodology · Statistics 2025-11-11 Abdullah Aloufi , Kevin Wilson , Nina Wilson , Lisa Shaw , Christopher Price

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

Intercurrent events, such as treatment switching, rescue medication, dropout, or truncation by death, frequently complicate intention-to-treat analyses in randomized clinical trials. Existing causal inference frameworks typically target…

Methodology · Statistics 2026-03-12 Georgi Baklicharov , Kelly Van Lancker , Stijn Vansteelandt

Multiple long-term conditions (MLTC) are increasingly observed in clinical practice globally. Clustering methods to group diseases into commonly co-occurring clusters have been of interest for further understanding of how MLTC group…

Applications · Statistics 2026-03-02 James Rafferty , Keith R Abrams , Munir Pirmohamed , Mark Davies , Rhiannon K Owen

Clinical trials often collect data on multiple outcomes, such as overall survival (OS), progression-free survival (PFS), and response to treatment (RT). In most cases, however, study designs only use primary outcome data for interim and…

Applications · Statistics 2026-04-28 Massimiliano Russo , Steffen Ventz , Lorenzo Trippa

In this paper, we propose the use of causal inference techniques for survival function estimation and prediction for subgroups of the data, upto individual units. Tree ensemble methods, specifically random forests were modified for this…

Econometrics · Economics 2018-03-23 Vikas Ramachandra

Randomized clinical trials (RCTs) are ideal for estimating causal effects, because the distributions of background covariates are similar in expectation across treatment groups. When estimating causal effects using observational data,…

Methodology · Statistics 2019-02-27 Anthony D. Scotina , Roee Gutman