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The use of machine learning systems to support decision making in healthcare raises questions as to what extent these systems may introduce or exacerbate disparities in care for historically underrepresented and mistreated groups, due to…

Machine Learning · Computer Science 2019-07-16 Stephen Pfohl , Tony Duan , Daisy Yi Ding , Nigam H. Shah

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

Target trial emulation has improved comparative effectiveness research by making the causal question, assumptions, and analysis plan explicit. However, target trial protocols are usually developed iteratively. After examining the data,…

Methodology · Statistics 2026-04-30 Mats Julius Stensrud

This article is a response to recent proposals by Pearl and others for a new approach to personalised treatment decisions, in contrast to the traditional one based on statistical decision theory. We argue that this approach is dangerously…

Methodology · Statistics 2025-03-31 A. Philip Dawid , Stephen Senn

The treatment allocation mechanism in a randomized clinical trial can be optimized by maximizing the nonparametric efficiency bound for a specific measure of treatment effect. Optimal treatment allocations which may or may not depend on…

Methodology · Statistics 2025-05-23 Wei Zhang , Zhiwei Zhang , Aiyi Liu

Matching is a widely used causal inference design that aims to approximate a randomized experiment using observational data by forming matched sets of treated and control units based on similarities in their covariates. Ideally, treated…

Methodology · Statistics 2026-04-06 Jianan Zhu , Jeffrey Zhang , Zijian Guo , Siyu Heng

Historical data from previous clinical trials, observational studies and health records may be utilized in analysis of clinical trials data to strengthen inference. Under the Bayesian framework incorporation of information obtained from any…

Applications · Statistics 2021-03-23 Shirin Golchi

Oversubscribed treatments are often allocated using randomized waiting lists. Applicants are ranked randomly, and treatment offers are made following that ranking until all seats are filled. To estimate causal effects, researchers often…

Methodology · Statistics 2018-10-24 Clement de Chaisemartin , Luc Behaghel

The impact of machine learning models on healthcare will depend on the degree of trust that healthcare professionals place in the predictions made by these models. In this paper, we present a method to provide people with clinical expertise…

Machine Learning · Computer Science 2021-03-05 Aniruddh Raghu , John Guttag , Katherine Young , Eugene Pomerantsev , Adrian V. Dalca , Collin M. Stultz

Over the past few decades, statistical methods for causal inference have made impressive strides, enabling progress across a range of scientific fields. However, much of this methodological development has been confined to individual…

Methodology · Statistics 2025-09-30 Wenqi Shi , José R. Zubizarreta

Causal inference from observational data provides strong evidence for the best action in decision-making without performing expensive randomized trials. The effect of an action is usually not identifiable under unobserved confounding, even…

Machine Learning · Computer Science 2026-02-02 Md Musfiqur Rahman , Ziwei Jiang , Hilaf Hasson , Murat Kocaoglu

Multivariate methods that relate outcomes to risk factors have been adopted clinically to individualize treatment. This has promoted the belief that individuals have a true or unique risk. The logic of assigning an individual a single risk…

Quantitative Methods · Quantitative Biology 2010-01-15 Ralph H. Stern

Measuring treatment efficacy in mixture of subgroups from a randomized clinical trial is a fundamental problem in personalized medicine development, in deciding whether to treat the entire patient population or to target a subgroup. We show…

Methodology · Statistics 2014-09-03 Ying Ding , Hui-Min Lin , Jason C. Hsu

In a randomised clinical trial, when the result of the primary endpoint shows a significant benefit, the secondary endpoints are scrutinised to identify additional effects of the treatment. However, this approach entails a risk of…

Statistical pragmatism embraces all efficient methods in statistical inference. Augmentation of the collected data is used herein to obtain representative population information from a large class of non-representative population's units.…

Statistics Theory · Mathematics 2015-12-04 Yannis G. Yatracos

Accurate predictions, as with machine learning, may not suffice to provide optimal healthcare for every patient. Indeed, prediction can be driven by shortcuts in the data, such as racial biases. Causal thinking is needed for data-driven…

Targeted Learning is a subfield of statistics that unifies advances in causal inference, machine learning and statistical theory to help answer scientifically impactful questions with statistical confidence. Targeted Learning is driven by…

Personalized healthcare decisions require reasoning about how physiological and behavioral variables influence an individual patient over time. Existing temporal causal discovery methods are poorly matched to this setting: cohort-level…

Machine Learning · Computer Science 2026-05-11 Elahe Khatibi , Ziyu Wang , Saba A. Farahani , Di Huang , Hung Cao , Ramesh Jain , Amir M. Rahmani

Clinical decisions to treat and diagnose patients are affected by implicit biases formed by racism, ableism, sexism, and other stereotypes. These biases reflect broader systemic discrimination in healthcare and risk marginalizing already…

Machine Learning · Computer Science 2025-01-29 Kara Liu , Russ Altman , Vasilis Syrgkanis

The idea of Artificial Intelligence (AI) has a long history. It turned out, however, that reaching intelligence at human levels is more complicated than originally anticipated. Currently we are experiencing a renewed interest in AI, fueled…

Computers and Society · Computer Science 2019-06-27 Michael van Hartskamp , Sergio Consoli , Wim Verhaegh , Milan Petković , Anja van de Stolpe
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