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Across research disciplines, cluster randomized trials (CRTs) are commonly implemented to evaluate interventions delivered to groups of participants, such as communities and clinics. Despite advances in the design and analysis of CRTs,…

Randomized controlled trials (RCTs) in oncology often allow control group participants to crossover to experimental treatments, a practice that, while often ethically necessary, complicates the accurate estimation of long-term treatment…

Methodology · Statistics 2025-02-12 Harlan Campbell , Nicholas Latimer , Jeroen P Jansen , Shannon Cope

Randomized trials are typically designed to detect average treatment effects but often lack the statistical power to uncover individual-level treatment effect heterogeneity, limiting their value for personalized decision-making. To address…

Machine Learning · Statistics 2026-03-19 Rickard Karlsson , Piersilvio De Bartolomeis , Issa J. Dahabreh , Jesse H. Krijthe

Many practical decision-making problems in economics and healthcare seek to estimate the average treatment effect (ATE) from observational data. The Double/Debiased Machine Learning (DML) is one of the prevalent methods to estimate ATE in…

Econometrics · Economics 2022-12-07 Yiyan Huang , Cheuk Hang Leung , Xing Yan , Qi Wu , Shumin Ma , Zhiri Yuan , Dongdong Wang , Zhixiang Huang

Estimating heterogeneous treatment effects (HTEs) is crucial for precision medicine. While multiple studies can improve the generalizability of results, leveraging them for estimation is statistically challenging. Existing approaches often…

Methodology · Statistics 2025-12-22 Cathy Shyr , Boyu Ren , Prasad Patil , Giovanni Parmigiani

Objective: Randomized controlled trial (RCT) results often inform clinical decision-making, but the highly curated populations of trials and the care provided during the trial are often not reflective of real-world practice. The objective…

Applications · Statistics 2024-02-26 Guanbo Wang , Ting-Wei Ernie Liao , David Furfaro , Leo Anthony Celi , Kevin Sheng-Kai Ma

In recent years, precision treatment strategy have gained significant attention in medical research, particularly for patient care. We propose a novel framework for estimating conditional average treatment effects (CATE) in time-to-event…

Methodology · Statistics 2024-07-29 Runjia Li , Victor B. Talisa , Chung-Chou H. Chang

Structural equation modeling (SEM) and path analysis have long been central tools for studying complex causal relationships in the social and behavioral sciences, yet their reliance on parametric assumptions can lead to biased inference…

Other Statistics · Statistics 2026-03-10 Junjie Ma , Xiaoya Zhang , Guangye He , Yuting Han , Ting Ge , Feng Ji

The weighted average treatment effect (WATE) defines a versatile class of causal estimands for populations characterized by propensity score weights, including the average treatment effect (ATE), treatment effect on the treated (ATT), on…

Methodology · Statistics 2025-09-23 Yiming Wang , Yi Liu , Shu Yang

Observational studies can yield clinically actionable evidence at scale, but executing them on real-world databases is open-ended and requires coherent decisions across cohort construction, analysis, and reporting. Prior evaluations of LLM…

Artificial Intelligence · Computer Science 2026-03-25 Dubai Li , Yuxiang He , Yan Hu , Yu Tian , Jingsong Li

The primary analysis of clinical trials in diabetes therapeutic area often involves a mixed-model repeated measure (MMRM) approach to estimate the average treatment effect for longitudinal continuous outcome, and a generalized linear mixed…

Applications · Statistics 2022-08-03 Lingjing Jiang , Michael Rosenblum , Yu Du

Disruptions in clinical trials may be due to external events like pandemics, warfare, and natural disasters. Resulting complications may lead to unforeseen intercurrent events (events that occur after treatment initiation and affect the…

Applications · Statistics 2024-08-20 Rachael V. Phillips , Mark J. van der Laan

Electronic health record (EHR) data are increasingly used to support real-world evidence (RWE) studies. Yet its ability to generate reliable RWE is limited by the lack of readily available precise information on the timing of clinical…

The estimation of heterogeneous treatment effects (HTEs) has attracted considerable interest in many disciplines, most prominently in medicine and economics. Contemporary research has so far primarily focused on continuous and binary…

Methodology · Statistics 2022-10-07 Susanne Dandl , Andreas Bender , Torsten Hothorn

Understanding causal heterogeneous treatment effects based on pretreatment covariates is a crucial aspect of empirical work. Building on Calonico, Cattaneo, Farrell, Palomba, and Titiunik (2025), this article discusses the software package…

Execution-based feedback like unit testing is widely used in the development of coding agents through test-time scaling (TTS) and reinforcement learning (RL). This paradigm requires scalable and reliable collection of unit test cases to…

Computation and Language · Computer Science 2025-12-29 KaShun Shum , Binyuan Hui , Jiawei Chen , Lei Zhang , X. W. , Jiaxi Yang , Yuzhen Huang , Junyang Lin , Junxian He

Quantifying the heterogeneity of treatment effect is important for understanding how a commercial product or medical treatment affects different population subgroups. While much of treatment effect heterogeneity analysis focuses on the…

Reinforcement learning (RL) enables an agent interacting with an unknown MDP $M$ to optimise its behaviour by observing transitions sampled from $M$. A natural entity that emerges in the agent's reasoning is $\widehat{M}$, the maximum…

Machine Learning · Computer Science 2025-02-24 Shivaram Kalyanakrishnan , Sheel Shah , Santhosh Kumar Guguloth

In this study, we compared two groups, in which subjects were assigned to either the treatment or the control group. In such trials, if the efficacy of the treatment cannot be demonstrated in a population that meets the eligibility…

Methodology · Statistics 2024-07-26 Shintaro Yuki , Kensuke Tanioka , Hiroshi Yadohisa

We often seek to estimate the impact of an exposure naturally occurring or randomly assigned at the cluster-level. For example, the literature on neighborhood determinants of health continues to grow. Likewise, community randomized trials…

Methodology · Statistics 2021-07-08 Laura B. Balzer , Wenjing Zheng , Mark J. van der Laan , Maya L. Petersen
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