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Related papers: The R.O.A.D. to precision medicine

200 papers

Observational studies provide the only evidence on the effectiveness of interventions when randomized controlled trials (RCTs) are impractical due to cost, ethical concerns, or time constraints. While many methodologies aim to draw causal…

Randomized Controlled Trials (RCTs) are the gold standard for comparing the effectiveness of a new treatment to the current one (the control). Most RCTs allocate the patients to the treatment group and the control group by uniform…

Machine Learning · Statistics 2018-10-22 Onur Atan , William R. Zame , Mihaela van der Schaar

Causal inference is vital for informed decision-making across fields such as biomedical research and social sciences. Randomized controlled trials (RCTs) are considered the gold standard for internal validity of inferences, whereas…

Methodology · Statistics 2025-12-02 Ruoqi Yu , Bikram Karmakar , Jessica Vandeleest , Eleanor Bimla Schwarz

Identifying patient subgroups with different treatment responses is an important task to inform medical recommendations, guidelines, and the design of future clinical trials. Existing approaches for treatment effect estimation primarily…

Methodology · Statistics 2025-12-10 Vincent Jeanselme , Chang Ho Yoon , Fabian Falck , Brian Tom , Jessica Barrett

Precision medicine provides customized treatments to patients based on their characteristics and is a promising approach to improving treatment efficiency. Large scale omics data are useful for patient characterization, but often their…

Machine Learning · Statistics 2023-01-24 Jianchang Hu , Silke Szymczak

The authors propose robust adaptive strategies based on stochastic minimax optimization for a series of simulated treatments on a one-dimensional patient phantom. The plan applied during the first fractions should be able to handle…

Optimization and Control · Mathematics 2018-10-01 Michelle Böck , Anders Forsgren , Kjell Eriksson , Björn Hårdemark

Geometric uncertainty can degrade treatment quality in radiation therapy. While margins and robust optimization mitigate these effects, they provide only implicit control over clinical goal fulfillment probability. We therefore develop a…

Medical Physics · Physics 2026-01-14 Albin Fredriksson , Erik Engwall , Jenneke de Jong , Johan Sundström

We consider the optimization of an uncertain objective over continuous and multi-dimensional decision spaces in problems in which we are only provided with observational data. We propose a novel algorithmic framework that is tractable,…

Machine Learning · Statistics 2018-10-30 Dimitris Bertsimas , Christopher McCord

In many applied optimization settings, parameters that define the constraints may not guarantee the best possible solution, and superior solutions might exist that are infeasible for the given parameter values. Removing such constraints,…

Optimization and Control · Mathematics 2024-07-22 Farzin Ahmadi , Todd R. McNutt , Kimia Ghobadi

Personalized medicine aims at identifying best treatments for a patient with given characteristics. It has been shown in the literature that these methods can lead to great improvements in medicine compared to traditional methods…

Machine Learning · Statistics 2018-11-26 Oleg Sysoev , Krzysztof Bartoszek , Eva-Charlotte Ekstrom , Katarina Ekholm Selling

This project considers how one might augment a limited amount of data from randomized controlled trial (RCT) with more plentiful data from an observational database (ODB), in order to estimate a causal effect. In our motivating setting, the…

Methodology · Statistics 2018-10-23 Evan Rosenman , Art B. Owen , Michael Baiocchi , Hailey Banack

Randomized controlled trials (RCTs) serve as the cornerstone for understanding causal effects, yet extending inferences to target populations presents challenges due to effect heterogeneity and underrepresentation. Our paper addresses the…

Methodology · Statistics 2025-05-19 Harsh Parikh , Rachael Ross , Elizabeth Stuart , Kara Rudolph

Precision oncology aims to prescribe the optimal cancer treatment to the right patients, maximizing therapeutic benefits. However, identifying patient subgroups that may benefit more from experimental cancer treatments based on randomized…

Methodology · Statistics 2026-01-06 Xingyu Li , Qing Liu , Tony Jiang , Hong Amy Xia , Peng Wei , Brian P. Hobbs

Randomized controlled trials (RCTs) are increasingly prevalent in education research, and are often regarded as a gold standard of causal inference. Two main virtues of randomized experiments are that they (1) do not suffer from…

Randomized Controlled Trials (RCTs) are the gold standard for evaluating the effect of new medical treatments. Treatments must pass stringent regulatory conditions in order to be approved for widespread use, yet even after the regulatory…

Machine Learning · Statistics 2025-03-13 Omer Noy Klein , Alihan Hüyük , Ron Shamir , Uri Shalit , Mihaela van der Schaar

To promote precision medicine, individualized treatment regimes (ITRs) are crucial for optimizing the expected clinical outcome based on patient-specific characteristics. However, existing ITR research has primarily focused on scenarios…

Methodology · Statistics 2024-02-20 Chang Wang , Lu Wang

In the realm of precision medicine, effective patient stratification and disease subtyping demand innovative methodologies tailored for multi-omics data. Clustering techniques applied to multi-omics data have become instrumental in…

Machine Learning · Computer Science 2024-01-30 Bastian Pfeifer , Christel Sirocchi , Marcus D. Bloice , Markus Kreuzthaler , Martin Urschler

A Randomized Control Trial (RCT) is considered as the gold standard for evaluating the effect of any intervention or treatment. However, its feasibility is often hindered by ethical, economical, and legal considerations, making…

Machine Learning · Computer Science 2024-03-13 Md Saiful Islam , Sahil Shikalgar , Md. Noor-E-Alam

Sequential multiple assignment randomized trials (SMARTs) are used to construct data-driven optimal intervention strategies for subjects based on their intervention and covariate histories in different branches of health and behavioral…

Methodology · Statistics 2022-04-28 Palash Ghosh , Xiaoxi Yan , Bibhas Chakraborty

Augmenting randomized controlled trials (RCTs) with external real-world data (RWD) has the potential to improve the finite sample efficiency of treatment effect estimators. We describe using adaptive targeted maximum likelihood estimation…

Methodology · Statistics 2025-01-30 Sky Qiu , Jens Tarp , Andrew Mertens , Mark van der Laan
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