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The ability to predict individualized treatment effects (ITEs) based on a given patient's profile is essential for personalized medicine. We propose a hypothesis testing approach to choosing between two potential treatments for a given…

Methodology · Statistics 2020-08-11 Tianxi Cai , Tony Cai , Zijian Guo

As the COVID-19 pandemic progresses, researchers are reporting findings of randomized trials comparing standard care with care augmented by experimental drugs. The trials have small sample sizes, so estimates of treatment effects are…

Econometrics · Economics 2020-06-02 Charles F. Manski , Aleksey Tetenov

Individualized treatment rules (ITRs) tailor treatments according to individual patient characteristics. They can significantly improve patient care and are thus becoming increasingly popular. The data collected during randomized clinical…

Methodology · Statistics 2015-06-30 Stanislav Minsker , Ying-Qi Zhao , Guang Cheng

Identifying patients who benefit from a treatment is a key aspect of personalized medicine, which allows the development of individualized treatment rules (ITRs). Many machine learning methods have been proposed to create such rules.…

Familiar statistical tests and estimates are obtained by the direct observation of cases of interest: a clinical trial of a new drug, for instance, will compare the drug's effects on a relevant set of patients and controls. Sometimes,…

Methodology · Statistics 2010-12-09 Bradley Efron

Precision medicine seeks to discover an optimal personalized treatment plan and thereby provide informed and principled decision support, based on the characteristics of individual patients. With recent advancements in medical imaging, it…

Methodology · Statistics 2023-04-26 Xinyi Li , Michael R. Kosorok

There is intense interest in applying machine learning to problems of causal inference in fields such as healthcare, economics and education. In particular, individual-level causal inference has important applications such as precision…

Machine Learning · Statistics 2017-05-17 Uri Shalit , Fredrik D. Johansson , David Sontag

In modern dynamic constantly developing society, more and more people suffer from chronic and serious diseases and doctors and patients need special and sophisticated medical and health support. Accordingly, prominent health stakeholders…

Computers and Society · Computer Science 2022-08-10 Mirjana Ivanovic , Serge Autexier , Miltiadis Kokkonidis

Individualized treatment rules (ITRs) are deterministic decision rules that recommend treatments to individuals based on their characteristics. Though ubiquitous in medicine, ITRs are hardly ever evaluated in randomized controlled trials.…

Methodology · Statistics 2023-08-22 François Grolleau , Francois Petit , Raphaël Porcher

The call for patient-focused drug development is loud and clear, as expressed in the 21st Century Cures Act and in recent guidelines and initiatives of regulatory agencies. Among the factors contributing to modernized drug development and…

In recent years, there has been a growing interest in the prediction of individualized treatment effects. While there is a rapidly growing literature on the development of such models, there is little literature on the evaluation of their…

Methodology · Statistics 2023-12-22 J Hoogland , O Efthimiou , TL Nguyen , TPA Debray

Causal inference is capable of estimating the treatment effect (i.e., the causal effect of treatment on the outcome) to benefit the decision making in various domains. One fundamental challenge in this research is that the treatment…

Machine Learning · Computer Science 2021-12-28 Qian Li , Zhichao Wang , Shaowu Liu , Gang Li , Guandong Xu

Patients who are seriously ill may ask doctors to treat them with unapproved medication, about which not much is known, or else with known medication in a high dosage. Apart from strict legal constraints such cases may involve difficult…

Other Statistics · Statistics 2018-04-13 F. Thomas Bruss

The burden of diseases is rising worldwide, with unequal treatment efficacy for patient populations that are underrepresented in clinical trials. Healthcare, however, is driven by the average population effect of medical treatments and,…

Machine Learning · Computer Science 2024-02-08 Ghadeer O. Ghosheh , Moritz Gögl , Tingting Zhu

Causal inference plays an important role in explanatory analysis and decision making across various fields like statistics, marketing, health care, and education. Its main task is to estimate treatment effects and make intervention…

Methodology · Statistics 2024-07-22 Yingrong Wang , Haoxuan Li , Minqin Zhu , Anpeng Wu , Ruoxuan Xiong , Fei Wu , Kun Kuang

Randomized trials typically estimate average relative treatment effects, but decisions on the benefit of a treatment are possibly better informed by more individualized predictions of the absolute treatment effect. In case of a binary…

Methodology · Statistics 2021-08-20 J Hoogland , J IntHout , M Belias , MM Rovers , RD Riley , FE Harrell , KGM Moons , TPA Debray , JB Reitsma

Causal inference methods are widely applied in various decision-making domains such as precision medicine, optimal policy and economics. Central to causal inference is the treatment effect estimation of intervention strategies, such as…

Artificial Intelligence · Computer Science 2021-05-31 Tri Dung Duong , Qian Li , Guandong Xu

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

When resources are scarce, an allocation policy is needed to decide who receives a resource. This problem occurs, for instance, when allocating scarce medical resources and is often solved using modern ML methods. This paper introduces…

Machine Learning · Computer Science 2024-02-20 Niclas Boehmer , Yash Nair , Sanket Shah , Lucas Janson , Aparna Taneja , Milind Tambe

An individualized treatment rule (ITR) is a decision rule that aims to improve individual patients health outcomes by recommending optimal treatments according to patients specific information. In observational studies, collected data may…

Methodology · Statistics 2023-10-03 Zeyu Bian , Erica EM Moodie , Susan M Shortreed , Sylvie D Lambert , Sahir Bhatnagar