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A treatment regime is a function that maps individual patient information to a recommended treatment, hence explicitly incorporating the heterogeneity in need for treatment across individuals. Patient responses are dichotomous and can be…

Machine Learning · Statistics 2016-07-07 Yingfei Wang , Warren Powell

Triple difference designs have become increasingly popular in empirical economics. The advantage of a triple difference design is that, within a treatment group, it allows for another subgroup of the population -- potentially less impacted…

Econometrics · Economics 2025-06-04 Laura Caron

In many practical tasks it is needed to estimate an effect of treatment on individual level. For example, in medicine it is essential to determine the patients that would benefit from a certain medicament. In marketing, knowing the persons…

Methodology · Statistics 2019-02-28 Aleksey Buzmakov

Individualized treatment rules, cornerstones of precision medicine, inform patient treatment decisions with the goal of optimizing patient outcomes. These rules are generally unknown functions of patients' pre-treatment covariates, meaning…

Methodology · Statistics 2025-04-23 Philippe Boileau , Ning Leng , Sandrine Dudoit

Cell heterogeneity plays an important role in patient responses to drug treatments. In many cancers, it is associated with poor treatment outcomes. Many modern drug combination therapies aim to exploit cell heterogeneity, but determining…

Quantitative Methods · Quantitative Biology 2025-02-18 Simon F. Martina-Perez , Samuel W. S. Johnson , Rebecca M. Crossley , Jennifer C. Kasemeier , Paul M. Kulesa , Ruth E. Baker

Hierarchical random effect models are used for different purposes in clinical research and other areas. In general, the main focus is on population parameters related to the expected treatment effects or group differences among all units of…

Applications · Statistics 2021-04-07 Maryna Prus , Norbert Benda , Rainer Schwabe

Quantifying treatment effect heterogeneity is a crucial task in many areas of causal inference, e.g. optimal treatment allocation and estimation of subgroup effects. We study the problem of estimating the level sets of the conditional…

Methodology · Statistics 2023-07-03 Matteo Bonvini , Edward H. Kennedy , Luke J. Keele

Multistate process data are common in studies of chronic diseases such as cancer. These data are ideal for precision medicine purposes as they can be leveraged to improve more refined health outcomes, compared to standard survival outcomes,…

Methodology · Statistics 2022-11-28 Giorgos Bakoyannis

We apply classical statistical decision theory to a large class of treatment choice problems with partial identification. We show that, in a general class of problems with Gaussian likelihood, all decision rules are admissible; it is…

Econometrics · Economics 2025-06-24 José Luis Montiel Olea , Chen Qiu , Jörg Stoye

Dynamic treatment regimes are of growing interest across the clinical sciences as these regimes provide one way to operationalize and thus inform sequential personalized clinical decision making. A dynamic treatment regime is a sequence of…

Methodology · Statistics 2013-11-27 Eric B. Laber , Min Qian , Dan J. Lizotte , William E. Pelham , Susan A. Murphy

Dynamic treatment regimes formalize precision medicine as a sequence of decision rules, one for each stage of clinical intervention, that map current patient information to a recommended intervention. Optimal regimes are typically defined…

Methodology · Statistics 2024-07-09 Kyle Duke , Eric B. Laber , Marie Davidian , Michael Newcomb , Brian Mustanksi

Platform trials are randomized clinical trials that allow simultaneous comparison of multiple interventions, usually against a common control. Arms to test experimental interventions may enter and leave the platform over time. This implies…

Methodology · Statistics 2024-04-12 Marta Bofill Roig , Ekkehard Glimm , Tobias Mielke , Martin Posch

We consider a multi-armed bandit problem with covariates. Given a realization of the covariate vector, instead of targeting the treatment with highest conditional expectation, the decision maker targets the treatment which maximizes a…

Machine Learning · Statistics 2024-12-25 Anders Bredahl Kock , David Preinerstorfer , Bezirgen Veliyev

We consider planning longitudinal covariate measurements in follow-up studies where covariates are time-varying. We assume that the entire cohort cannot be selected for longitudinal measurements due to financial limitations and study how a…

Methodology · Statistics 2024-07-03 Jaakko Reinikainen , Juha Karvanen

In personalised decision making, evidence is required to determine whether an action (treatment) is suitable for an individual. Such evidence can be obtained by modelling treatment effect heterogeneity in subgroups. The existing…

Methodology · Statistics 2022-06-24 Jiuyong Li , Lin Liu , Shisheng Zhang , Saisai Ma , Thuc Duy Le , Jixue Liu

Clinical trials are an instrument for making informed decisions based on evidence from well-designed experiments. Here we consider adaptive designs mainly from the perspective of multi-arm Phase II clinical trials, in which one or more…

Methodology · Statistics 2021-08-31 Elja Arjas , Dario Gasbarra

Discovering subgroups with the maximum average treatment effect is crucial for targeted decision making in domains such as precision medicine, public policy, and education. While most prior work is formulated in the potential outcome…

Machine Learning · Computer Science 2025-11-26 Lincen Yang , Zhong Li , Matthijs van Leeuwen , Saber Salehkaleybar

The increasing prevalence of rich sources of data and the availability of electronic medical record databases and electronic registries opens tremendous opportunities for enhancing medical research. For example, controlled trials are…

Methodology · Statistics 2015-09-23 Liwen Ouyang , Daniel W. Apley , Sanjay Mehrotra

A dynamic treatment regime is a sequence of treatment decision rules tailored to an individual's evolving status over time. In precision medicine, much focus has been placed on finding an optimal dynamic treatment regime which, if followed…

Other Statistics · Statistics 2025-10-13 Chunyu Wang , Brian DM Tom

Identifying optimal medical treatments to improve survival has long been a critical goal of pharmacoepidemiology. Traditionally, we use an average treatment effect measure to compare outcomes between treatment plans. However, new methods…

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