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Heterogeneous treatment effect estimation is critical in oncology, particularly in multi-arm trials with overlapping therapeutic components and long-term survivors. These shared mechanisms pose a central challenge to identifying causal…

Methodology · Statistics 2025-10-29 Peter Chang , John Kairalla , Arkaprava Roy

Causal effect estimation for dynamic treatment regimes (DTRs) contributes to sequential decision making. However, censoring and time-dependent confounding under DTRs are challenging as the amount of observational data declines over time due…

Machine Learning · Statistics 2021-09-27 Adi Lin , Jie Lu , Junyu Xuan , Fujin Zhu , Guangquan Zhang

Causal mediation analysis is a powerful tool in environmental health research, allowing researchers to uncover the pathways through which exposures influence health outcomes. While traditional mediation methods have been widely applied to…

Discrete biomarkers derived as cell densities or counts from tissue microarrays and immunostaining are widely used to study immune signatures in relation to survival outcomes in cancer. Although routinely collected, these signatures are not…

In many clinical and epidemiological studies, collecting longitudinal measurements together with time-to-event outcomes is essential. Accurately estimating the association between longitudinal markers and event risks, as well as identifying…

\textbf{Background:} Mediation analysis is widely used to investigate how treatments and programs exert their effects, but standard ordinary least squares (OLS) inference can be unreliable when regression errors are non-Gaussian. In medical…

Methodology · Statistics 2026-04-10 Mijeong Kim

During drug development, evidence can emerge to suggest a treatment is more effective in a specific patient subgroup. Whilst early trials may be conducted in biomarker-mixed populations, later trials are more likely to enrol…

Methodology · Statistics 2023-06-07 Lorna Wheaton , Dan Jackson , Sylwia Bujkiewicz

We propose a new Bayesian non-parametric (BNP) method for estimating the causal effects of mediation in the presence of a post-treatment confounder. We specify an enriched Dirichlet process mixture (EDPM) to model the joint distribution of…

Methodology · Statistics 2023-05-10 Woojung Bae , Michael J. Daniels , Michael G. Perri

We consider the Bayesian estimation of the parameters of a finite mixture model from independent order statistics arising from imperfect ranked set sampling designs. As a cost-effective method, ranked set sampling enables us to incorporate…

Multi-energy CT takes advantage of the non-linearly varying attenuation properties of elemental media with respect to energy, enabling more precise material identification than single-energy CT. The increased precision comes with the cost…

Medical Physics · Physics 2020-02-14 Jussi Toivanen , Alexander Meaney , Samuli Siltanen , Ville Kolehmainen

Phase 1-2 designs provide a methodological advance over phase 1 designs for dose finding by using both clinical response and toxicity. A phase 1-2 trial still may fail to select a truly optimal dose. because early response is not a perfect…

Applications · Statistics 2024-04-03 Cheng-Han Yang , Peter F. Thall , Ruitao Lin

Causal mediation analysis in cluster-randomized trials (CRTs) is essential for explaining how cluster-level interventions affect individual outcomes, yet it is complicated by interference, post-treatment confounding, and hierarchical…

Methodology · Statistics 2026-03-25 Yuki Ohnishi , Michael J. Daniels , Lei Yang , Fan Li

With reference to a stratified case-control procedure based on a binary variable of primary interest, we derive the expression of the distortion induced by the sampling design on the parameters of the logistic model of a secondary variable.…

Methodology · Statistics 2024-01-18 Marco Doretti , Minna Genbäck , Elena Stanghellini

An essential goal of program evaluation and scientific research is the investigation of causal mechanisms. Over the past several decades, causal mediation analysis has been used in medical and social sciences to decompose the treatment…

Methodology · Statistics 2016-01-15 K. C. G. Chan , K. Imai , S. C. P. Yam , Z. Zhang

Often linear regression is used to perform mediation analysis. However, in many instances, the underlying relationships may not be linear, as in the case of placental-fetal hormones and fetal development. Although, the exact functional form…

Methodology · Statistics 2023-10-16 Qing Yin , Jong-Hyeon Jeong , Xu Qin , Shyamal D Peddada , Jennifer Adibi

Cost-effectiveness analyses (CEAs) compare the costs and health outcomes of treatment regimes to inform medical decisions. With observational claims data, CEAs must address nonrandom treatment assignment, administrative censoring, and…

Methodology · Statistics 2026-01-22 Esteban Fernández-Morales , Emily M. Ko , Nandita Mitra , Youjin Lee , Arman Oganisian

Mediation analysis is a useful tool to evaluate surrogate endpoints in clinical trials. We propose a novel method, the M-survival learner, for estimating heterogeneous indirect treatment effects in the presence of censored outcomes. The…

Methodology · Statistics 2026-04-16 Xingyu Li , Qing Liu , Xun Jiang , Hong Amy Xia , Brian P. Hobbs , Peng Wei

Background: Radiotherapy treatment planning involves solving large-scale optimization problems that are often approximated and solved sub-optimally due to time constraints. Central to these problems is the dose influence matrix which…

Medical Physics · Physics 2024-10-02 Mojtaba Tefagh , Gourav Jhanwar , Masoud Zarepisheh

Dysregulation in signal transduction pathways can lead to a variety of complex disorders, including cancer. Computational approaches such as network analysis are important tools to understand system dynamics as well as to identify critical…

Molecular Networks · Quantitative Biology 2015-11-12 Bhanwar Lal Puniya , Laura Allen , Colleen Hochfelder , Mahbubul Majumder , Tomáš Helikar

Dose-finding trials for oncology studies are traditionally designed to assess safety in the early stages of drug development. With the rise of molecularly targeted therapies and immuno-oncology compounds, biomarker-driven approaches have…

Methodology · Statistics 2025-09-15 Xijin Chen , Pavel Mozgunov , Richard D. Baird , Thomas Jaki