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The progression-free survival ratio (PFSr) is a widely used measure in personalized oncology trials. It evaluates the effectiveness of treatment by comparing two consecutive event times - one under standard therapy and one under an…

统计方法学 · 统计学 2025-12-23 Merle Munko , Simon Mack , Marc Ditzhaus , Stefan Fröhling , Dennis Dobler , Dominic Edelmann

The Intensive Care Unit (ICU) is a hospital department where machine learning has the potential to provide valuable assistance in clinical decision making. Classical machine learning models usually only provide point-estimates and no…

机器学习 · 计算机科学 2024-07-26 David Ruhe , Giovanni Cinà , Michele Tonutti , Daan de Bruin , Paul Elbers

An important task in drug development is to identify patients, which respond better or worse to an experimental treatment. Identifying predictive covariates, which influence the treatment effect and can be used to define subgroups of…

统计方法学 · 统计学 2018-11-27 Marius Thomas , Björn Bornkamp , Katja Ickstadt

Tumors constitute a wide family of diseases kinetically characterized by the co-presence of multiple spatio-temporal scales. So, tumor cells ecologically interplay with other kind of cells, e.g. endothelial cells or immune system effectors,…

计算工程、金融与科学 · 计算机科学 2012-08-21 Giulio Caravagna , Alex Graudenzi , Marco Antoniotti , Giancarlo Mauri , Alberto d'Onofrio

Historical data about disease outcomes can be integrated into the analysis of clinical trials in many ways. We build on existing literature that uses prognostic scores from a predictive model to increase the efficiency of treatment effect…

统计方法学 · 统计学 2020-12-25 David Walsh , Alejandro Schuler , Diana Hall , Jon Walsh , Charles Fisher

Diagnostic clinics are among healthcare facilities that suffer from long waiting times which can cause medical issues and lead to increases in patient no-shows. Reducing waiting times without significant capital investments is a challenging…

最优化与控制 · 数学 2020-04-20 Mahsa Kiani , Burak Eksioglu , Tugce Isik , Alexandria Thomas , John Gilpin

Clinical trials are a systematic endeavor to assess the safety and efficacy of new drugs or treatments. Conducting such trials typically demands significant financial investment and meticulous planning, highlighting the need for accurate…

机器学习 · 计算机科学 2025-11-03 Tien Huu Do , Antoine Masquelier , Nae Eoun Lee , Jonathan Crowther

Bayesian approaches have become increasingly popular in causal inference problems due to their conceptual simplicity, excellent performance and in-built uncertainty quantification ('posterior credible sets'). We investigate Bayesian…

机器学习 · 统计学 2019-09-27 Kolyan Ray , Botond Szabo

The question of selecting the "best" amongst different choices is a common problem in statistics. In drug development, our motivating setting, the question becomes, for example: what is the dose that gives me a pre-specified risk of…

统计理论 · 数学 2018-03-15 Pavel Mozgunov , Thomas Jaki

This study proposes a new Bayesian approach to infer binary treatment effects. The approach treats counterfactual untreated outcomes as missing observations and infers them by completing a matrix composed of realized and potential untreated…

统计方法学 · 统计学 2021-04-20 Masahiro Tanaka

Phase 1-2 designs provide a methodological advance over phase 1 designs for dose finding by using both clinical response and toxicity. A phase 1-2 trial still may fail to select a truly optimal dose. because early response is not a perfect…

应用统计 · 统计学 2024-04-03 Cheng-Han Yang , Peter F. Thall , Ruitao Lin

Two useful strategies to speed up drug development are to increase the patient accrual rate and use novel adaptive designs. Unfortunately, these two strategies often conflict when the evaluation of the outcome cannot keep pace with the…

统计方法学 · 统计学 2018-07-24 Ruitao Lin , Ying Yuan

This study examines the application of Bayesian approach in the context of clinical trials, emphasizing their increasing importance in contemporary biomedical research. While conventional frequentist approach provides a foundational basis…

统计方法学 · 统计学 2026-01-16 Paramahansa Pramanik , Arnab Kumar Maity , Anjan Mandal , Haley Kate Robinson

I propose a finite sample inference procedure that uses a likelihood function derived from the randomization process within an experiment to conduct inference on various quantities that capture heterogeneous intervention effects. One such…

统计方法学 · 统计学 2020-09-03 Amanda Kowalski

The determination of the sample size required by a crossover trial typically depends on the specification of one or more variance components. Uncertainty about the value of these parameters at the design stage means that there is often a…

统计方法学 · 统计学 2018-03-28 Michael Grayling , Adrian Mander , James Wason

Optimization of complex functions, such as the output of computer simulators, is a difficult task that has received much attention in the literature. A less studied problem is that of optimization under unknown constraints, i.e., when the…

统计方法学 · 统计学 2010-07-06 Robert B. Gramacy , Herbert K. H. Lee

In this paper, we accomplish two objectives: First, we provide a new mathematical characterization of the value function for impulse control problems with implementation delay and present a direct solution method that differs from its…

最优化与控制 · 数学 2008-12-10 Erhan Bayraktar , Masahiko Egami

This paper proposes a new non-parametric bootstrap method to quantify the uncertainty of average treatment effect estimate for the treated from matching estimators. More specifically, it seeks to quantify the uncertainty associated with the…

统计方法学 · 统计学 2024-08-21 Jing Li

While the use of combination therapy is increasing in prevalence for cancer treatment, it is often difficult to predict the exact interactions between different treatment forms, and their synergistic/antagonistic effects on patient health…

最优化与控制 · 数学 2021-01-29 Jayanth Pratap

We study treatment-effect estimation using panel data. The treatment may be non-binary, non-absorbing, and the outcome may be affected by treatment lags. We make a parallel-trends assumption, and propose event-study estimators of the effect…

计量经济学 · 经济学 2026-05-13 Clément de Chaisemartin , Xavier D'Haultfœuille