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Interference occurs when a unit's treatment (or exposure) affects another unit's outcome. In some settings, units may be grouped into clusters such that it is reasonable to assume that interference, if present, only occurs between…

Methodology · Statistics 2023-08-24 Chanhwa Lee , Donglin Zeng , Michael G. Hudgens

In stepped wedge cluster randomized trials (SW-CRTs), interventions are sequentially rolled out to clusters over multiple periods. It is common practice to analyze SW-CRTs using discrete-time linear mixed models, in which measurements are…

Methodology · Statistics 2025-11-25 Hao Wang , Guangyu Tong , Heather Allore , Monica Taljaard , Fan Li

While palliative care is increasingly commonly delivered to hospitalized patients with serious illnesses, few studies have estimated its causal effects. Courtright et al. (2016) adopted a cluster-randomized stepped-wedge design to assess…

We consider the identification of average treatment effects on the treated (ATT) in difference-in-differences (DiD) settings in the presence of endogenous sample selection. We first establish that the conventional DiD estimand generally…

Econometrics · Economics 2026-02-17 Gayani Rathnayake , Akanksha Negi , Otavio Bartalotti , Xueyan Zhao

In adaptive clinical trials, the conventional end-of-trial point estimate of a treatment effect is prone to bias, that is, a systematic tendency to deviate from its true value. As stated in recent FDA guidance on adaptive designs, it is…

We present a sequential testing method to identify a practically significant effect. We build on the existing mixture sequential probability ratio test (mSPRT) that can sequentially test for a non-zero treatment effect by using a truncated…

Methodology · Statistics 2025-09-10 Kyu Min Shim

We describe group sequential tests which efficiently incorporate information from multiple endpoints allowing for early stopping at pre-planned interim analyses. We formulate a testing procedure where several outcomes are examined, and…

Methodology · Statistics 2024-05-09 Abigail J. Burdon , Thomas Jaki

Simulating Standardized Patients with cognitive impairment offers a scalable and ethical solution for clinical training. However, existing methods rely on discrete prompt engineering and fail to capture the heterogeneity of deficits across…

Artificial Intelligence · Computer Science 2026-04-17 Weikang Zhang , Zimo Zhu , Zhichuan Yang , Chen Huang , Wenqiang Lei , See-Kiong Ng

Modeling neural population dynamics underlying noisy single-trial spiking activities is essential for relating neural observation and behavior. A recent non-recurrent method - Neural Data Transformers (NDT) - has shown great success in…

Neurons and Cognition · Quantitative Biology 2022-06-13 Trung Le , Eli Shlizerman

The bulk of causal inference studies rule out the presence of interference between units. However, in many real-world scenarios, units are interconnected by social, physical, or virtual ties, and the effect of the treatment can spill from…

Methodology · Statistics 2023-11-03 Falco J. Bargagli-Stoffi , Costanza Tortù , Laura Forastiere

In causal inference, the correct formulation of the scientific question of interest is a crucial step. Here we apply the estimand framework to a comparison of the outcomes of patient-level clinical trials and observational data to help…

Group sequential designs in clinical trials allow for interim efficacy and futility monitoring. Adjustment for baseline covariates can increase power and precision of estimated effects. However, inconsistently applying covariate adjustment…

Methodology · Statistics 2023-08-11 Marlena S. Bannick , Sonya L. Heltshe , Noah Simon

Treatment noncompliance is pervasive in infectious disease cluster-randomized trials. Although all individuals within a cluster are assigned the same treatment condition, the treatment uptake status may vary across individuals due to…

Methodology · Statistics 2025-12-19 Chao Cheng , Georgia Papadogeorgou , Fan Li

Covariate-specific treatment effects (CSTEs) represent heterogeneous treatment effects across subpopulations defined by certain selected covariates. In this article, we consider marginal structural models where CSTEs are linearly…

Methodology · Statistics 2021-05-25 Peng Wu , Zhiqiang Tan , Wenjie Hu , Xiao-Hua Zhou

Background: In settings where proof-of-principle trials have succeeded but the effectiveness of different forms of implementation remains uncertain, trials that not only generate information about intervention effects but also provide…

Quantitative Methods · Quantitative Biology 2017-05-16 Guy Harling , Rui Wang , Jukka-Pekka Onnela , Victor De Gruttola

At the present time, sequential item recommendation models are compared by calculating metrics on a small item subset (target set) to speed up computation. The target set contains the relevant item and a set of negative items that are…

Information Retrieval · Computer Science 2021-07-29 Alexander Dallmann , Daniel Zoller , Andreas Hotho

While randomized controlled trials (RCTs) are critical for establishing the efficacy of new therapies, there are limitations regarding what comparisons can be made directly from trial data. RCTs are limited to a small number of comparator…

We study identifying and estimating the causal effect of a treatment variable on a long-term outcome using data from an observational and an experimental domain. The observational data are subject to unobserved confounding. Furthermore,…

Introduction: Modelling of relative treatment effects is an important aspect to consider when extrapolating the long-term survival outcomes of treatments. Flexible parametric models offer the ability to accurately model the observed data,…

Methodology · Statistics 2024-01-02 Philip Cooney , Arthur White

In this paper, we introduce a unified estimator to analyze various treatment effects in causal inference, including but not limited to the average treatment effect (ATE) and the quantile treatment effect (QTE). The proposed estimator is…

Methodology · Statistics 2025-03-31 Kuan-Hsun Wu , Li-Pang Chen
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