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An accurate estimation of the dose-response relationship is important to determine the optimal dose. For this purpose, a dose finding trial in which subjects are randomized to a few fixed dose levels is the most commonly used design. Often,…

Methodology · Statistics 2025-08-07 Jixian Wang , Zhiwei Zhang , Ram Tiwari

In this article, we propose a phase I-II design in two stages for the combination of molecularly targeted therapies. The design is motivated by a published case study that combines a MEK and a PIK3CA inhibitors; a setting in which higher…

Methodology · Statistics 2025-05-21 José L. Jiménez , Mourad Tighiouart

Dose-response models express the effect of different dose or exposure levels on a specific outcome. In meta-analysis, where aggregated-level data is available, dose-response evidence is synthesized using either one-stage or two-stage models…

Subclassification and matching are often used in empirical studies to adjust for observed covariates; however, they are largely restricted to relatively simple study designs with a binary treatment and less developed for designs with a…

Methodology · Statistics 2022-01-28 Bo Zhang , Emily J. Mackay , Mike Baiocchi

In longitudinal studies, we observe measurements of the same variables at different time points to track the changes in their pattern over time. In such studies, scheduling of the data collection waves (i.e. time of participants' visits) is…

Methodology · Statistics 2020-04-30 Li Xing , Xuekui Zhang , Ardo van den Hout , Scott Hofer , Graciela Muniz Terrera

To estimate casual treatment effects, we propose a new matching approach based on the reduced covariates obtained from sufficient dimension reduction. Compared to the original covariates and the propensity score, which are commonly used for…

Methodology · Statistics 2017-02-03 Wei Luo , Yeying Zhu

An objective of phase I dose-finding trials is to find the maximum tolerated dose; the dose with a particular risk of toxicity. Frequently, this risk is assessed across the first cycle of therapy. However, in oncology, a course of treatment…

Applications · Statistics 2021-05-03 Helen Barnett , Oliver Boix , Dimintris Kontos , Thomas Jaki

Active search is a learning paradigm for actively identifying as many members of a given class as possible. A critical target scenario is high-throughput screening for scientific discovery, such as drug or materials discovery. In this…

Machine Learning · Computer Science 2018-11-27 Shali Jiang , Gustavo Malkomes , Benjamin Moseley , Roman Garnett

Bayesian models quantify uncertainty and facilitate optimal decision-making in downstream applications. For most models, however, practitioners are forced to use approximate inference techniques that lead to sub-optimal decisions due to…

Machine Learning · Statistics 2019-09-12 Tomasz Kuśmierczyk , Joseph Sakaya , Arto Klami

An important objective in biomedical risk assessment is estimation of minimum exposure levels that induce a pre-specified adverse response in a target population. The exposure/dose points in such settings are known as Benchmark Doses…

Methodology · Statistics 2014-02-18 Qijun Fang , Walter W. Piegorsch , Susan J. Simmons , Xiaosong Li , Cuixian Chen , Yishi Wang

Modern computers enable methods for design optimization that account for uncertainty in the system---so-called optimization under uncertainty. We propose a metric for OUU that measures the distance between a designer-specified probability…

Optimization and Control · Mathematics 2015-08-10 Pranay Seshadri , Paul Constantine , Gianluca Iaccarino , Geoffrey Parks

The last decade has seen an explosion in data sources available for the monitoring and prediction of environmental phenomena. While several inferential methods have been developed that make predictions on the underlying process by combining…

Methodology · Statistics 2023-03-06 Eun-Hye Yoo , Andrew Zammit-Mangion , Michael G. Chipeta

Systems with both quantitative and qualitative responses are widely encountered in many applications. Design of experiment methods are needed when experiments are conducted to study such systems. Classic experimental design methods are…

Methodology · Statistics 2023-04-24 Lulu Kang , Xinwei Deng , Ran Jin

With the development of novel therapies such as molecularly targeted agents and immunotherapy, the maximum tolerated dose paradigm that "more is better" does not necessarily hold anymore. In this context, doses and schedules of novel…

Adaptive enrichment trials aim to identify and recruit participants most likely to benefit from treatment based on evolving biomarker evidence, with the goal of informing individualized treatment recommendations. Bayesian methods are well…

Methodology · Statistics 2026-03-11 Lara Maleyeff , Shirin Golchi , Erica E. M. Moodie

Clinical trials are an integral component of medical research. Trials require careful design to, for example, maintain the safety of participants, use resources efficiently and allow clinically meaningful conclusions to be drawn. Adaptive…

Methodology · Statistics 2025-07-17 James M. McGree , Antony M. Overstall , Mark Jones , Robert K. Mahar

For biological experiments aiming at calibrating models with unknown parameters, a good experimental design is crucial, especially for those subject to various constraints, such as financial limitations, time consumption and physical…

Applications · Statistics 2014-07-22 Xiao Lin , Gabriel Terejanu

Matching is a commonly used causal inference study design in observational studies. Through matching on measured confounders between different treatment groups, valid randomization inferences can be conducted under the no unmeasured…

Methodology · Statistics 2024-09-20 Jeffrey Zhang , Siyu Heng

We consider the classical problem of selecting the best of two treatments in clinical trials with binary response. The target is to find the design that maximizes the power of the relevant test. Many papers use a normal approximation to the…

Statistics Theory · Mathematics 2011-03-22 David Azriel , Micha Mandel , Yosef Rinott

The efficacy of robust optimization spans a variety of settings with uncertainties bounded in predetermined sets. In many applications, uncertainties are affected by decisions and cannot be modeled with current frameworks. This paper takes…

Optimization and Control · Mathematics 2018-03-29 Omid Nohadani , Kartikey Sharma
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