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Dynamic treatment regimes have been proposed to personalize treatment decisions by utilizing historical patient data, but they may not always improve on the current standard of care. It is thus meaningful to integrate the standard of care…

Applications · Statistics 2025-12-11 Johannes Hruza , Arvid Sjölander , Erin Gabriel , Samir Bhatt , Michael Sachs

Randomized controlled trials are the gold standard for evaluating the efficacy of an intervention. However, there is often a trade-off between selecting the most scientifically relevant primary endpoint versus a less relevant, but more…

Methodology · Statistics 2026-01-12 Jack M. Wolf , Joseph S. Koopmeiners , David M. Vock

Mid-study design modifications are becoming increasingly accepted in confirmatory clinical trials, so long as appropriate methods are applied such that error rates are controlled. It is therefore unfortunate that the important case of…

Applications · Statistics 2014-05-08 Dominic Magirr , Thomas Jaki , Franz Koenig , Martin Posch

Many applied decision-making problems have a dynamic component: The policymaker needs not only to choose whom to treat, but also when to start which treatment. For example, a medical doctor may choose between postponing treatment (watchful…

Methodology · Statistics 2020-05-01 Xinkun Nie , Emma Brunskill , Stefan Wager

The rise and spread of antibiotic resistance causes worsening medical cost and mortality especially for life-threatening bacteria infections, thereby posing a major threat to global health. Prescribing behavior of physicians is one of the…

Populations and Evolution · Quantitative Biology 2018-10-23 Xingru Chen , Feng Fu

The tolerance of bacterial populations to biocidal or antibiotic treatment has been well documented in both biofilm and planktonic settings. However, there is still very little known about the mechanisms that produce this tolerance.…

Populations and Evolution · Quantitative Biology 2008-07-15 Patrick De Leenheer , Nick Cogan

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

Many clinical studies require the follow-up of patients over time. This is challenging: apart from frequently observed drop-out, there are often also organizational and financial challenges, which can lead to reduced data collection and, in…

Machine Learning · Computer Science 2022-10-26 Fateme Nateghi Haredasht , Celine Vens

In many randomized experiments, the treatment effect of the long-term metric (i.e. the primary outcome of interest) is often difficult or infeasible to measure. Such long-term metrics are often slow to react to changes and sufficiently…

Imitation learning has enabled robots to perform complex, long-horizon tasks in challenging dexterous manipulation settings. As new methods are developed, they must be rigorously evaluated and compared against corresponding baselines…

Recent work has focused on nonparametric estimation of conditional treatment effects, but inference has remained relatively unexplored. We propose a class of nonparametric tests for both quantitative and qualitative treatment effect…

Methodology · Statistics 2026-04-07 Oliver Dukes , Mats J. Stensrud , Riccardo Brioschi , Aaron Hudson

We seek to understand the probability an individual benefits from treatment (PIBT), an inestimable quantity that must be bounded in practice. Given the innate uncertainty in the population-level bounds on PIBT, we seek to better understand…

Methodology · Statistics 2024-04-04 Gabriel Ruiz , Oscar Hernan Madrid Padilla

L.J. Savage once hoped to show that "the superficially incompatible systems of ideas associated on the one hand with [subjective Bayesianism] and on the other hand with [classical statistics]...lend each other mutual support and…

Statistics Theory · Mathematics 2017-07-04 Greg Gandenberger

Patient care may be improved by recommending treatments based on patient characteristics when there is treatment effect heterogeneity. Recently, there has been a great deal of attention focused on the estimation of optimal treatment rules…

Methodology · Statistics 2024-01-29 Michael Jetsupphasuk , Michael G. Hudgens , Jessie K. Edwards , Stephen R. Cole

The term natural experiment is used inconsistently. In one interpretation, it refers to an experiment where a treatment is randomly assigned by someone other than the researcher. In another interpretation, it refers to a study in which…

Methodology · Statistics 2020-02-04 Rocio Titiunik

While randomized controlled trials (RCTs) are critical for establishing the efficacy of new therapies, there are limitations regarding what comparisons can be made directly from trial data. RCTs are limited to a small number of comparator…

Phase III randomized clinical trials play a monumentally critical role in the evaluation of new medical products. Because of the intrinsic nature of uncertainty embedded in our capability in assessing the efficacy of a medical product,…

Methodology · Statistics 2019-02-25 Changyu Shen , Xiaochun Li

We study a game theoretic model of standardized testing for college admissions. Students are of two types; High and Low. There is a college that would like to admit the High type students. Students take a potentially costly standardized…

Computer Science and Game Theory · Computer Science 2021-02-17 Sampath Kannan , Mingzi Niu , Aaron Roth , Rakesh Vohra

Externally controlled trials (ECTs) are increasingly used when randomized controls are infeasible, unethical, or insufficient, including applications in rare diseases, oncology, pediatrics, and post-approval effectiveness research. Although…

The ICH E9(R1) addendum (2019) proposed principal stratification (PS) as one of five strategies for dealing with intercurrent events. Therefore, understanding the strengths, limitations, and assumptions of PS is important for the broad…