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In the genomic era, the identification of gene signatures associated with disease is of significant interest. Such signatures are often used to predict clinical outcomes in new patients and aid clinical decision-making. However, recent…

Methodology · Statistics 2019-03-27 Naim U. Rashid , Quefeng Li , Jen Jen Yeh , Joseph G. Ibrahim

Accurate identification of EGFR and KRAS mutations is essential for precision therapy in non-small cell lung cancer (NSCLC), but tissue genotyping is invasive and may not capture tumor heterogeneity. CT-based radiogenomics offers a…

Subgroup-specific meta-analysis synthesizes treatment effects for patient subgroups across randomized trials. Methods include joint or separate modeling of subgroup effects and treatment-by-subgroup interactions, but inconsistencies arise…

Methodology · Statistics 2025-08-22 Renato Panaro , Christian Röver , Tim Friede

In observational studies, covariate imbalance generates confounding, resulting in biased comparisons. Although propensity score-based weighting approaches facilitate unconfounded group comparisons for implicit target populations, existing…

Methodology · Statistics 2023-07-03 Subharup Guha , David C. Christiani , Yi Li

Precision medicine promises to transform health care by offering individualised treatments that dramatically improve clinical outcomes. A necessary prerequisite is to identify subgroups of patients who respond differently to different…

Machine Learning · Computer Science 2026-03-03 Adam Marcus , Paul Agapow

A common goal in comparative effectiveness research is to estimate treatment effects on pre-specified subpopulations of patients. Though widely used in medical research, causal inference methods for such subgroup analysis remain…

Identifying subgroups, which respond differently to a treatment, both in terms of efficacy and safety, is an important part of drug development. A well-known challenge in exploratory subgroup analyses is the small sample size in the…

Computation · Statistics 2016-06-28 Marius Thomas , Björn Bornkamp

Propensity Score Matching (PSM) is an useful method to reduce the impact ofTreatment - Selection Bias in the estimation of causal effects in observational studies. After matching, the PSM significantly reduces the sample under…

Methodology · Statistics 2019-02-01 Daniel García Iglesias

Investigators often use multi-source data (e.g., multi-center trials, meta-analyses of randomized trials, pooled analyses of observational cohorts) to learn about the effects of interventions in subgroups of some well-defined target…

Methodology · Statistics 2024-02-06 Guanbo Wang , Alexander Levis , Jon Steingrimsson , Issa Dahabreh

When treating depression, clinicians are interested in determining the optimal treatment for a given patient, which is challenging given the amount of treatments available. To advance individualized treatment allocation, integrating data…

This paper proposes a new non-parametric bootstrap method to quantify the uncertainty of average treatment effect estimate for the treated from matching estimators. More specifically, it seeks to quantify the uncertainty associated with the…

Methodology · Statistics 2024-08-21 Jing Li

To investigate whether treating cancer patients with erythropoiesis-stimulating agents (ESAs) would increase the mortality risk, Bennett et al. [Journal of the American Medical Association 299 (2008) 914--924] conducted a meta-analysis with…

Applications · Statistics 2010-10-11 Rui Wang , Lu Tian , Tianxi Cai , L. J. Wei

Heterogeneous treatment effect (HTE) estimation from observational data poses significant challenges due to treatment selection bias. Existing methods address this bias by minimizing distribution discrepancies between treatment groups in…

Machine Learning · Computer Science 2026-03-26 Hao Wang , Zhichao Chen , Zhaoran Liu , Xu Chen , Haoxuan Li , Zhouchen Lin

Estimating heterogeneous treatment effect (HTE) for survival outcomes has gained increasing attention, as it captures the variation in treatment efficacy across patients or subgroups in delaying disease progression. However, most existing…

Methodology · Statistics 2025-11-27 Na Bo , Ying Ding

Subgroup analyses within randomized controlled trials are often underpowered due to limited sample sizes. We address this challenge by leveraging trial participants outside the subgroup of interest to augment estimation within the subgroup.…

Methodology · Statistics 2026-05-18 Sky Qiu , Nerissa Nance , Rachael Phillips , Jens Tarp , Maya Petersen , Mark van der Laan

The restricted mean survival time (RMST) difference offers an interpretable causal contrast to estimate the treatment effect for time-to-event outcomes, yet a wide range of available estimators leaves limited guidance for practice. We…

Methodology · Statistics 2026-03-02 Charlotte Voinot , Clément Berenfeld , Imke Mayer , Bernard Sebastien , Julie Josse

Representative risk estimation is fundamental to clinical decision-making. However, risks are often estimated from non-representative epidemiologic studies, which usually underrepresent minorities. "Model-based" methods use population…

Methodology · Statistics 2023-04-12 Lingxiao Wang , Yan Li , Barry I. Graubard , Hormuzd A. Katki

Population-adjusted indirect comparisons estimate treatment effects when access to individual patient data is limited and there are cross-trial differences in effect modifiers. Popular methods include matching-adjusted indirect comparison…

Applications · Statistics 2021-11-05 Antonio Remiro-Azócar , Anna Heath , Gianluca Baio

Randomized control trials (RCTs) are the gold standard for estimating causal effects, but often use samples that are non-representative of the actual population of interest. We propose a reweighting method for estimating population average…

Methodology · Statistics 2022-02-09 Kellie Ottoboni , Jason Poulos

Commonly, clinical trials report effects not only for the full study population but also for patient subgroups. Meta-analyses of subgroup-specific effects and treatment-by-subgroup interactions may be inconsistent, especially when trials…

Methodology · Statistics 2025-12-23 Renato Panaro , Christian Röver , Tim Friede