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Doctors use statistics to advance medical knowledge; we use a medical analogy to introduce statistical inference "from scratch" and to highlight an improvement. Your doctor, perhaps implicitly, predicts the effectiveness of a treatment for…

Methodology · Statistics 2015-11-29 Keli Liu , Xiao-Li Meng

Instrumental variable (IV) methods are used to estimate causal effects in settings with unobserved confounding, where we cannot directly experiment on the treatment variable. Instruments are variables which only affect the outcome…

Methodology · Statistics 2023-05-26 Elisabeth Ailer , Jason Hartford , Niki Kilbertus

Instrumental variable (IV) regression is a standard strategy for learning causal relationships between confounded treatment and outcome variables from observational data by utilizing an instrumental variable, which affects the outcome only…

Machine Learning · Computer Science 2023-06-28 Liyuan Xu , Yutian Chen , Siddarth Srinivasan , Nando de Freitas , Arnaud Doucet , Arthur Gretton

In observational studies, treatments are typically not randomized and therefore estimated treatment effects may be subject to confounding bias. The instrumental variable (IV) design plays the role of a quasi-experimental handle since the IV…

Methodology · Statistics 2016-08-30 Lan Liu , Wang Miao , Baoluo Sun , James Robins , Eric Tchetgen Tchetgen

In a randomized controlled trial, treatment switching (also called contamination or crossover) occurs when a patient initially assigned to one treatment arm changes to another arm during the course of follow-up. Overlooking treatment…

Methodology · Statistics 2022-09-27 Andrew Ying

The goal of personalized decision making is to map a unit's characteristics to an action tailored to maximize the expected outcome for that unit. Obtaining high-quality mappings of this type is the goal of the dynamic regime literature. In…

Machine Learning · Computer Science 2018-10-01 Razieh Nabi , Phyllis Kanki , Ilya Shpitser

Accurately quantifying uncertainty of individual treatment effects (ITEs) across multiple decision points is crucial for personalized decision-making in fields such as healthcare, finance, education, and online marketplaces. Previous work…

Methodology · Statistics 2025-12-10 Swaraj Bose , Walter Dempsey

Precision medicine is a rapidly expanding area of health research wherein patient level information is used to inform treatment decisions. A statistical framework helps to formalize the individualization of treatment decisions that…

Methodology · Statistics 2021-12-23 Coraline Danieli , Erica Moodie

The effect of vigorous physical activity on mortality in the elderly is difficult to estimate using conventional approaches to causal inference that define this effect by comparing the mortality risks corresponding to hypothetical scenarios…

Applications · Statistics 2007-12-18 Oliver Bembom , Mark J. van der Laan

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

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…

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

Estimating individualized treatment rules - particularly in the context of right-censored outcomes - is challenging because the treatment effect heterogeneity of interest is often small, thus difficult to detect. While this motivates the…

Applications · Statistics 2022-02-16 Erica EM Moodie , Janie Coulombe , Coraline Danieli , Christel Renoux , Susan M Shortreed

One of the key challenges when developing a predictive model is the capability to describe the domain knowledge and the cause-effect relationships in a simple way. Decision rules are a useful and important methodology in this context,…

Machine Learning · Computer Science 2021-10-19 Francisco Valente , Jorge Henriques , Simão Paredes , Teresa Rocha , Paulo de Carvalho , João Morais

Comparative effectiveness research frequently employs the instrumental variable design since randomized trials can be infeasible for many reasons. In this study, we investigate and compare treatments for emergency cholecystitis --…

Applications · Statistics 2023-07-13 Kenta Takatsu , Alexander W. Levis , Edward Kennedy , Rachel Kelz , Luke Keele

Temporally dense single-person "small data" have become widely available thanks to mobile apps and wearable sensors. Many caregivers and self-trackers want to use these data to help a specific person change their behavior to achieve desired…

Methodology · Statistics 2025-09-30 Eric J. Daza , Igor Matias , Logan Schneider

Individual participant data (IPD) meta-analyses of randomised trials are considered a reliable way to assess participant-level treatment effect modifiers but may not make the best use of the available data. Traditionally, effect modifiers…

For many complex diseases, there is a wide variety of ways in which an individual can manifest the disease. The challenge of personalized medicine is to develop tools that can accurately predict the trajectory of an individual's disease,…

Machine Learning · Statistics 2016-01-22 Peter Schulam , Suchi Saria

Quantile optimal treatment regimes (OTRs) aim to assign treatments that maximize a specified quantile of patients' outcomes. Compared to treatment regimes that target the mean outcomes, quantile OTRs offer fairer regimes when a lower…

Methodology · Statistics 2026-01-07 Junwen Xia , Jingxiao Zhang , Dehan Kong

Pricing based on individual customer characteristics is widely used to maximize sellers' revenues. This work studies offline personalized pricing under endogeneity using an instrumental variable approach. Standard instrumental variable…

Methodology · Statistics 2023-02-27 Rui Miao , Zhengling Qi , Cong Shi , Lin Lin