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We consider a randomized controlled trial between two groups. The objective is to identify a population with characteristics such that the test therapy is more effective than the control therapy. Such a population is called a subgroup. This…

Methodology · Statistics 2021-12-06 Shintaro Yuki , Kensuke Tanioka , Hiroshi Yadohisa

Developing targeted therapies based on patients' baseline characteristics and genomic profiles such as biomarkers has gained growing interests in recent years. Depending on patients' clinical characteristics, the expression of specific…

Applications · Statistics 2019-02-26 Yanxun Xu , Florica Constantine , Yuan Yuan , Yili L. Pritchett

Effect modification means the size of a treatment effect varies with an observed covariate. Generally speaking, a larger treatment effect with more stable error terms is less sensitive to bias. Thus, we might be able to conclude that a…

Methodology · Statistics 2026-05-19 Yijun Fan , Dylan S. Small

Estimating treatment effects conditional on observed covariates can improve the ability to tailor treatments to particular individuals. Doing so effectively requires dealing with potential confounding, and also enough data to adequately…

Safety analyses in terms of adverse events (AEs) are an important aspect of benefit-risk assessments of therapies. Compared to efficacy analyses AE analyses are often rather simplistic. The probability of an AE of a specific type is…

Applications · Statistics 2021-05-20 Regina Stegherr , Claudia Schmoor , Michael Lübbert , Tim Friede , Jan Beyersmann

We consider the problem of selecting the optimal subgroup to treat when data on covariates is available from a randomized trial or observational study. We distinguish between four different settings including (i) treatment selection when…

Methodology · Statistics 2018-02-28 Tyler J. VanderWeele , Alex R. Luedtke , Mark J. van der Laan , Ronald C. Kessler

Nested error regression models are useful tools for analysis of grouped data, especially in the case of small area estimation. This paper suggests a nested error regression model using uncertain random effects in which the random effect in…

Methodology · Statistics 2017-02-28 Shonosuke Sugasawa , Tatsuya Kubokawa

In modern drug development, the broader availability of high-dimensional observational data provides opportunities for scientist to explore subgroup heterogeneity, especially when randomized clinical trials are unavailable due to cost and…

Methodology · Statistics 2021-02-24 Xinzhou Guo , Linqing Wei , Chong Wu , Jingshen Wang

Accurately predicting conditional average treatment effects (CATEs) is crucial in personalized medicine and digital platform analytics. Since the treatments of interest often cannot be directly randomized, observational data is leveraged to…

Methodology · Statistics 2024-11-05 Miruna Oprescu , Nathan Kallus

Causal mediation analysis of observational data is an important tool for investigating the potential causal effects of medications on disease-related risk factors, and on time-to-death (or disease progression) through these risk factors.…

In various biomedical studies, analysis often focuses on data magnitudes, particularly when algebraic signs are irrelevant or lost. For repeated measures studies involving magnitude outcomes, incorporating random effects is essential as…

Methodology · Statistics 2025-07-16 Wen Teng , Niall D. Ferguson , Ewan C. Goligher , Anna Heath

This study examines the application of Bayesian approach in the context of clinical trials, emphasizing their increasing importance in contemporary biomedical research. While conventional frequentist approach provides a foundational basis…

Methodology · Statistics 2026-01-16 Paramahansa Pramanik , Arnab Kumar Maity , Anjan Mandal , Haley Kate Robinson

Datasets containing large samples of time-to-event data arising from several small heterogeneous groups are commonly encountered in statistics. This presents problems as they cannot be pooled directly due to their heterogeneity or analyzed…

Machine Learning · Statistics 2016-12-05 Alexandre Piché , Russell Steele , Ian Shrier , Stephanie Long

Gun violence is a major source of injury and death in the United States. However, relatively little is known about the effects of firearm injuries on survivors and their family members and how these effects vary across subpopulations. To…

Applications · Statistics 2024-06-04 Eric R. Cohn , Zirui Song , Jose R. Zubizarreta

We propose a novel approach to Bayesian analysis that is provably robust to outliers in the data and often has computational advantages over standard methods. Our technique is based on splitting the data into non-overlapping subgroups,…

Statistics Theory · Mathematics 2016-06-03 Stanislav Minsker , Sanvesh Srivastava , Lizhen Lin , David B. Dunson

When evaluating the efficacy of social programs and medical treatments using randomized experiments, the estimated overall average causal effect alone is often of limited value and the researchers must investigate when the treatments do and…

Applications · Statistics 2013-05-27 Kosuke Imai , Marc Ratkovic

We propose a multi-metric flexible Bayesian framework to support efficient interim decision-making in multi-arm multi-stage phase II clinical trials. Multi-arm multi-stage phase II studies increase the efficiency of drug development, but…

Applications · Statistics 2023-12-15 Suzanne M. Dufault , Angela M. Crook , Katie Rolfe , Patrick P. J. Phillips

Heterogeneous treatment effects, which vary according to individual covariates, are crucial in fields such as personalized medicine and tailored treatment strategies. In many applications, rather than considering the heterogeneity induced…

Methodology · Statistics 2025-08-26 Peng Wu , Pengtao Zeng , Zhaoqing Tian , Shaojie Wei

Suppose it is of interest to characterize effect heterogeneity of an intervention across levels of a baseline covariate using only pre- and post- intervention outcome measurements from those who received the intervention, i.e. with no…

Methodology · Statistics 2023-06-21 Zach Shahn

While observational data are routinely used to estimate causal effects of biomedical treatments, doing so requires special methods to adjust for observed confounding. These methods invariably rely on untestable statistical and causal…

Methodology · Statistics 2026-03-02 Arman Oganisian