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Factor models are widely applied to the analysis of multivariate data across disparate fields of research. However, modern scientific data are often incomplete, and estimating a factor model from partially observed data can be very…

Methodology · Statistics 2026-02-24 Giuseppe Vinci

Given the cost and duration of phase III and phase IV clinical trials, the development of statistical methods for go/no-go decisions is vital. In this paper, we introduce a Bayesian methodology to compute the probability of success based on…

Methodology · Statistics 2020-10-27 Ethan M. Alt , Matthew A. Psioda , Joseph G. Ibrahim

Ecological Momentary Assessments (EMA) capture real-time thoughts and behaviors in natural settings, producing rich longitudinal data for statistical and physiological analyses. However, the robustness of these analyses can be compromised…

Methodology · Statistics 2023-11-21 Yiheng Wei , Donald Hedeker

Existing causal methods for time-varying exposure and time-varying confounding focus on estimating the average causal effect of a time-varying binary treatment on an end-of-study outcome, offering limited tools for characterizing marginal…

Methodology · Statistics 2026-01-21 Yu Luo , Kuan Liu , Ramandeep Singh , Daniel J. Graham

Phase I dose-escalation trials must be guided by a safety model in order to avoid exposing patients to unacceptably high risk of toxicities. Traditionally, these trials are based on one type of schedule. In more recent practice, however,…

Methodology · Statistics 2020-08-18 Burak Kürsad Günhan , Sebastian Weber , Tim Friede

This paper introduces a novel approach for estimating heterogeneous treatment effects of binary treatment in panel data, particularly focusing on short panel data with large cross-sectional data and observed confoundings. In contrast to…

Methodology · Statistics 2024-06-05 Meijia Wang , Ignacio Martinez , P. Richard Hahn

Many applied decision-making problems have a dynamic component: The policymaker needs not only to choose whom to treat, but also when to start which treatment. For example, a medical doctor may choose between postponing treatment (watchful…

Methodology · Statistics 2020-05-01 Xinkun Nie , Emma Brunskill , Stefan Wager

Mediation analysis plays a crucial role in causal inference as it can investigate the pathways through which treatment influences outcome. Most existing mediation analysis assumes that mediation effects are static and homogeneous within…

Methodology · Statistics 2026-03-02 Yijiao Zhang , Yubai Yuan , Yuexia Zhang , Zhongyi Zhu , Annie Qu

Gene therapies aim to address the root causes of diseases, particularly those stemming from rare genetic defects that can be life-threatening or severely debilitating. Although an increasing number of gene therapies have received regulatory…

Methodology · Statistics 2026-01-19 Tianyu Pan , Yiyao Shi , Xiang Zhang , Weining Shen , Ting Ye

Meta-analysis is a systematic approach for understanding a phenomenon by analyzing the results of many previously published experimental studies. It is central to deriving conclusions about the summary effect of treatments and interventions…

Computers and Society · Computer Science 2021-04-13 Lu Cheng , Dmitriy A. Katz-Rogozhnikov , Kush R. Varshney , Ioana Baldini

Clinical research and drug development in orphan diseases is challenging, since large-scale randomized studies are difficult to conduct. Formally synthesizing the evidence is therefore of great value, yet this is rarely done in the drug…

Methodology · Statistics 2018-09-11 Simon Wandel , Beat Neuenschwander , Tim Friede , Christian Röver

In clinical trials, ensuring the quality and validity of data for downstream analysis and results is paramount, thus necessitating thorough data monitoring. This typically involves employing edit checks and manual queries during data…

Other Statistics · Statistics 2026-01-15 Yuxi Zhao , Margaret Gamalo

The doubly robust estimator, which models both the propensity score and outcomes, is a popular approach to estimate the average treatment effect in the potential outcome setting. The primary appeal of this estimator is its theoretical…

Methodology · Statistics 2024-09-11 Kaoru Babasaki , Shonosuke Sugasawa , Kosaku Takanashi , Kenichiro McAlinn

Bayesian Optimization aims at optimizing an unknown non-convex/concave function that is costly to evaluate. We are interested in application scenarios where concurrent function evaluations are possible. Under such a setting, BO could choose…

Artificial Intelligence · Computer Science 2012-05-02 Javad Azimi , Ali Jalali , Xiaoli Fern

Bayesian approaches to clinical analyses for the purposes of patient phenotyping have been limited by the computational challenges associated with applying the Markov-Chain Monte-Carlo (MCMC) approach to large real-world data. Approximate…

Applications · Statistics 2023-03-27 Brian Buckley , Adrian O'Hagan , Marie Galligan

Diabetes, a pervasive and enduring health challenge, imposes significant global implications on health, financial healthcare systems, and societal well-being. This study undertakes a comprehensive exploration of various structural learning…

Machine Learning · Computer Science 2024-03-22 Sheresh Zahoor , Anthony C. Constantinou , Tim M Curtis , Mohammed Hasanuzzaman

Genetic interactions play an important role in the progression of complex diseases, providing explanation of variations in disease phenotype missed by main genetic effects. Comparatively, there are fewer investigations on prognostic…

Methodology · Statistics 2021-09-23 Xing Qin , Shuangge Ma , Mengyun Wu

It is generally appreciated that a frequentist analysis of a group sequential trial must in order to avoid inflating type I error account for the fact that one or more interim analyses were performed. It is also to a lesser extent realised…

Methodology · Statistics 2025-12-12 Simon Bang Kristensen , Erik Thorlund Parner

We consider joint selection of fixed and random effects in general mixed-effects models. The interpretation of estimated mixed-effects models is challenging since changing the structure of one set of effects can lead to different choices of…

Methodology · Statistics 2020-02-26 Maud Delattre , Marie-Anne Poursat

It is becoming increasingly common for researchers to consider incorporating external information from large studies to improve the accuracy of statistical inference instead of relying on a modestly sized dataset collected internally. With…

Methodology · Statistics 2021-07-20 Tian Gu , Jeremy M. G. Taylor , Bhramar Mukherjee