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Patient subtyping based on temporal observations can lead to significantly nuanced subtyping that acknowledges the dynamic characteristics of diseases. Existing methods for subtyping trajectories treat the evolution of clinical observations…

Machine Learning · Computer Science 2018-11-30 Nikhil Galagali , Minnan Xu-Wilson

The way that people make choices or exhibit preferences can be strongly affected by the set of available alternatives, often called the choice set. Furthermore, there are usually heterogeneous preferences, either at an individual level…

Computer Science and Game Theory · Computer Science 2020-08-04 Kiran Tomlinson , Austin R. Benson

Understanding effect modification -- how treatment effects vary across subpopulations -- is practically important in observational studies, as it helps identify which subgroups are likely to benefit from a given treatment. In this paper, we…

Methodology · Statistics 2026-05-12 Yu Gui , Dylan S Small , Zhimei Ren

Data-driven techniques are used in cyber-physical systems (CPS) for controlling autonomous vehicles, handling demand responses for energy management, and modeling human physiology for medical devices. These data-driven techniques extract…

Machine Learning · Computer Science 2017-08-16 Sangdon Park , James Weimer , Insup Lee

An important task in clinical medicine is the construction of risk prediction models for specific subgroups of patients based on high-dimensional molecular measurements such as gene expression data. Major objectives in modeling…

Methodology · Statistics 2020-03-23 Katrin Madjar , Jörg Rahnenführer

Classical tests for a difference in means control the type I error rate when the groups are defined a priori. However, when the groups are instead defined via clustering, then applying a classical test yields an extremely inflated type I…

Methodology · Statistics 2022-11-01 Lucy L. Gao , Jacob Bien , Daniela Witten

It is a common practice in randomized clinical trials with the standard survival outcome to follow patients until a prespecified number of events have been observed, a type of trial known as the event-driven trial. The event-driven design…

Methodology · Statistics 2026-02-10 Jingwen Zhang , Satoshi Hattori

Heterogeneity is a hallmark of complex diseases. Regression-based heterogeneity analysis, which is directly concerned with outcome-feature relationships, has led to a deeper understanding of disease biology. Such an analysis identifies the…

Methodology · Statistics 2022-11-29 Ziye Luo , Xinyue Yao , Yifan Sun , Xinyan Fan

Cluster-randomized experiments are widely used due to their logistical convenience and policy relevance. To analyze them properly, we must address the fact that the treatment is assigned at the cluster level instead of the individual level.…

Methodology · Statistics 2021-08-06 Fangzhou Su , Peng Ding

In a regression context, when the relevant subset of explanatory variables is uncertain, it is common to use a data-driven model selection procedure. Classical linear model theory, applied naively to the selected sub-model, may not be valid…

Statistics Theory · Mathematics 2017-12-08 Liang Hong , Todd A. Kuffner , Ryan Martin

An important objective in the development of targeted therapies is to identify the populations where the treatment under consideration has positive benefit risk balance. We consider pivotal clinical trials, where the efficacy of a treatment…

External controls from historical trials or observational data can augment randomized controlled trials when large-scale randomization is impractical or unethical, such as in drug evaluation for rare diseases. However, non-randomized…

Methodology · Statistics 2025-05-08 Ke Zhu , Shu Yang , Xiaofei Wang

Breakthroughs in cancer biology have defined new research programs emphasizing the development of therapies that target specific pathways in tumor cells. Innovations in clinical trial design have followed with master protocols defined by…

Methodology · Statistics 2020-07-09 Alexander M. Kaizer , Joseph S. Koopmeiners , Nan Chen , Brian P. Hobbs

Treatment effect estimation is a fundamental problem in causal inference. We focus on designing efficient randomized controlled trials, to accurately estimate the effect of some treatment on a population of $n$ individuals. In particular,…

Machine Learning · Computer Science 2022-10-14 Raghavendra Addanki , David Arbour , Tung Mai , Cameron Musco , Anup Rao

In electronic health records (EHR) analysis, clustering patients according to patterns in their data is crucial for uncovering new subtypes of diseases. Existing medical literature often relies on classical hypothesis testing methods to…

Methodology · Statistics 2024-05-07 Zihan Zhu , Xin Gai , Anru R. Zhang

I examine the problem of treatment choice when a planner observes (i) covariates that describe each member of a population of interest and (ii) the outcomes of an experiment in which subjects randomly drawn from this population are randomly…

Applications · Statistics 2013-02-01 Charles F. Manski

In this paper, we propose a new approach to causal inference with panel data. Instead of using panel data to adjust for differences in the distribution of unobserved heterogeneity between the treated and comparison groups, we instead use…

Econometrics · Economics 2025-12-01 Brantly Callaway , Derek Dyal , Pedro H. C. Sant'Anna , Emmanuel S. Tsyawo

Clinical trial adaptation refers to any adjustment of the trial protocol after the onset of the trial. The main goal is to make the process of introducing new medical interventions to patients more efficient by reducing the cost and the…

Machine Learning · Computer Science 2014-11-17 Ognjen Arandjelovic

The use of unsupervised learning to identify patient subgroups has emerged as a potentially promising direction to improve the efficiency of Intensive Care Units (ICUs). By identifying subgroups of patients with similar levels of medical…

Machine Learning · Computer Science 2024-03-06 Harry Mayne , Guy Parsons , Adam Mahdi

Selective inference methods are developed for group lasso estimators for use with a wide class of distributions and loss functions. The method includes the use of exponential family distributions, as well as quasi-likelihood modeling for…

Methodology · Statistics 2024-03-28 Yiling Huang , Sarah Pirenne , Snigdha Panigrahi , Gerda Claeskens
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