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An individualized treatment rule (ITR) is a decision rule that aims to improve individual patients health outcomes by recommending optimal treatments according to patients specific information. In observational studies, collected data may…

Methodology · Statistics 2023-10-03 Zeyu Bian , Erica EM Moodie , Susan M Shortreed , Sylvie D Lambert , Sahir Bhatnagar

Randomized controlled trials (RCTs) are the accepted standard for treatment effect estimation but they can be infeasible due to ethical reasons and prohibitive costs. Single-arm trials, where all patients belong to the treatment group, can…

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

We propose a difference-in-differences (DiD) framework with mediation for possibly multivalued discrete or continuous treatments and mediators, aimed at identifying the direct effect of the treatment on the outcome (net of effects operating…

Econometrics · Economics 2026-03-02 Martin Huber , Sarina Joy Oberhänsli

We propose a dynamic allocation procedure that increases power and efficiency when measuring an average treatment effect in sequential randomized trials exploiting some subjects' previous assessed responses. Subjects arrive sequentially and…

Methodology · Statistics 2021-06-03 Adam Kapelner , Abba Krieger

Dynamic treatment regimes (DTRs) formalize medical decision-making as a sequence of rules for different stages, mapping patient-level information to recommended treatments. In practice, estimating an optimal DTR using observational data…

Methodology · Statistics 2024-12-11 Jian Sun , Bo Fu , Li Su

Recent advances in dynamic treatment regimes (DTRs) facilitate the search for optimal treatments, which are tailored to individuals' specific needs and able to maximize their expected clinical benefits. However, existing algorithms relying…

Machine Learning · Statistics 2024-10-18 Hanwen Ye , Wenzhuo Zhou , Ruoqing Zhu , Annie Qu

Heterogeneous treatment effect estimation in high-stakes applications demands models that simultaneously optimize precision, interpretability, and calibration. Many existing tree-based causal inference techniques, however, exhibit high…

Machine Learning · Computer Science 2025-04-21 Yichen Liu

Dynamic treatment regimes have been proposed to personalize treatment decisions by utilizing historical patient data, but they may not always improve on the current standard of care. It is thus meaningful to integrate the standard of care…

Applications · Statistics 2025-12-11 Johannes Hruza , Arvid Sjölander , Erin Gabriel , Samir Bhatt , Michael Sachs

Randomized experiments ensure robust causal inference that are critical to effective learning analytics research and practice. However, traditional randomized experiments, like A/B tests, are limiting in large scale digital learning…

Applications · Statistics 2019-02-04 Timothy NeCamp , Josh Gardner , Christopher Brooks

Randomized experiments are the gold standard for causal inference but face significant challenges in business applications, including limited traffic allocation, the need for heterogeneous treatment effect estimation, and the complexity of…

Methodology · Statistics 2025-08-18 Zhenkang Peng , Chengzhang Li , Ying Rong , Renyu Zhang

A fundamental principle of clinical medicine is that a treatment should only be administered to those patients who would benefit from it. Treatment strategies that assign treatment to patients as a function of their individual…

Applications · Statistics 2025-06-13 Nicholas Williams , Kara Rudolph , Iván Díaz

Observational cohort studies are increasingly being used for comparative effectiveness research to assess the safety of therapeutics. Recently, various doubly robust methods have been proposed for average treatment effect estimation by…

Methodology · Statistics 2025-03-11 Xiaoqing Tan , Shu Yang , Wenyu Ye , Douglas E. Faries , Ilya Lipkovich , Zbigniew Kadziola

Single-arm trials are an important study design for evaluating drug efficacy and safety without enrolling patients into a control arm. Although they do not provide the gold-standard evidence of randomized controlled trials, they are…

Applications · Statistics 2026-05-14 Daniele Bertolini , Franklin Fuller , Aaron M. Smith , Jonathan R. Walsh , Run Zhuang

We introduce mixed model trace regression (MMTR), a mixed model linear regression extension for scalar responses and high-dimensional matrix-valued covariates. MMTR's fixed effects component is equivalent to trace regression, with an…

Methodology · Statistics 2025-03-19 Ian Hultman , Sanvesh Srivastava

To promote precision medicine, individualized treatment regimes (ITRs) are crucial for optimizing the expected clinical outcome based on patient-specific characteristics. However, existing ITR research has primarily focused on scenarios…

Methodology · Statistics 2024-02-20 Chang Wang , Lu Wang

Recently a new experimental approach, the hybrid experimental design (HED), was introduced to enable investigators to answer scientific questions about building behavioral interventions in which human-delivered and digital components are…

Methodology · Statistics 2026-02-26 Mengbing Li , Inbal Nahum-Shani , Walter Dempsey

Large language models (LLMs) achieve strong performance in long-horizon decision-making tasks through multi-step interaction and reasoning at test time. While practitioners commonly believe a higher task success rate necessitates the use of…

Artificial Intelligence · Computer Science 2026-05-15 Yuanzhe Li , Jianing Deng , Jingtong Hu , Tianlong Chen , Song Wang , Huanrui Yang

Individualized treatment decisions can improve health outcomes, but using data to make these decisions in a reliable, precise, and generalizable way is challenging with a single dataset. Leveraging multiple randomized controlled trials…

Many rare diseases offer limited established treatment options, leading patients to switch therapies when new medications emerge. To analyze the impact of such treatment switches within the low sample size limitations of rare disease…

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