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Applications of artificial intelligence (AI) in drug development continue to increase at a rapid pace. Regulatory authorities have provided increasingly clear perspectives on the use of AI in regulated applications, including recent draft…

Applications · Statistics 2026-05-25 Aaron M. Smith , Tala Fakhouri , Run Zhuang , Jonathan R. Walsh

In this paper, a Bayesian approach is developed for simultaneously comparing multiple experimental treatments with a common control treatment in an exploratory clinical trial. The sample size is set to ensure that, at the end of the study,…

Statistics Theory · Mathematics 2019-11-14 John Whitehead , Faye Cleary , Amanda Turner

A recent method using Dynamic Linear Models to improve preferred treatment allocation budget in random allocation models was proposed by Lee, Boone, et al (2020). However this model failed to include the impact covariates such as smoking,…

Applications · Statistics 2020-09-24 Albert H. Lee

We consider the utilization of a computational model to guide the optimal acquisition of experimental data to inform the stochastic description of model input parameters. Our formulation is based on the recently developed consistent…

Computation · Statistics 2021-05-04 Scott N. Walsh , Tim M. Wildey , John D. Jakeman

To generalize inferences from a randomized trial to the target population of all trial-eligible individuals, investigators can use nested trial designs, where the randomized individuals are nested within a cohort of trial-eligible…

The Design of Experiments (DOEs) is a fundamental scientific methodology that provides researchers with systematic principles and techniques to enhance the validity, reliability, and efficiency of experimental outcomes. In this study, we…

Machine Learning · Statistics 2025-07-22 Miao Huang , Hongqiao Wang , Kunyu Wu

In a recent paper Dette et al. (2014) introduced optimal design problems for dose fnding studies with an active control. These authors concentrated on regression models with normal distributed errors (with known variance) and the problem of…

Methodology · Statistics 2014-11-03 Holger Dette , Katrin Kettelhake , Frank Bretz

Adaptive experimental designs have gained popularity in clinical trials and online experiments. Unlike traditional, fixed experimental designs, adaptive designs can dynamically adjust treatment randomization probabilities and other design…

Methodology · Statistics 2025-08-19 Wenxin Zhang , Mark van der Laan

A fundamental issue in causal inference for Big Observational Data is confounding due to covariate imbalances between treatment groups. This can be addressed by designing the data prior to analysis. Existing design methods, developed for…

Methodology · Statistics 2022-03-17 Yumin Zhang , Arman Sabbaghi

To maximize clinical benefit, clinicians routinely tailor treatment to the individual characteristics of each patient, where individualized treatment rules are needed and are of significant research interest to statisticians. In the…

Methodology · Statistics 2021-11-23 Trinetri Ghosh , Yanyuan Ma , Rui Song , Pingshou Zhong

The statistical efficiency of randomized clinical trials can be improved by incorporating information from baseline covariates (i.e., pre-treatment patient characteristics). This can be done in the design stage using stratified (permutated…

Methodology · Statistics 2025-02-04 Zhiwei Zhang

A/B testing is critical for modern technological companies to evaluate the effectiveness of newly developed products against standard baselines. This paper studies optimal designs that aim to maximize the amount of information obtained from…

Methodology · Statistics 2023-11-07 Ting Li , Chengchun Shi , Jianing Wang , Fan Zhou , Hongtu Zhu

A case-cohort design is a two-phase sampling design frequently used to analyze censored survival data in a cost-effective way, where a subcohort is usually selected using simple random sampling or stratified simple random sampling. In this…

Methodology · Statistics 2024-11-06 Kaeum Choi , Sangwook Kang

There has been a split in the statistics community about the need for taking covariates into account in the design phase of a clinical trial. There are many advocates of using stratification and covariate-adaptive randomization to promote…

Methodology · Statistics 2011-02-21 William F. Rosenberger , Oleksandr Sverdlov

When a new treatment is considered for use, whether a pharmaceutical drug or a search engine ranking algorithm, a typical question that arises is, will its performance exceed that of the current treatment? The conventional way to answer…

Machine Learning · Computer Science 2016-10-27 Nir Rosenfeld , Yishay Mansour , Elad Yom-Tov

We develop a set of algorithms to solve a broad class of Design of Experiment (DoE) problems efficiently. Specifically, we consider problems in which one must choose a subset of polymers to test in experiments such that the learning of the…

Quantitative Methods · Quantitative Biology 2024-08-06 Swagatam Mukhopadhyay

Medical research has evolved conventions for choosing sample size in randomized clinical trials that rest on the theory of hypothesis testing. Bayesians have argued that trials should be designed to maximize subjective expected utility in…

Methodology · Statistics 2018-12-11 Charles F. Manski , Aleksey Tetenov

The sequential treatment decisions made by physicians to treat chronic diseases are formalized in the statistical literature as dynamic treatment regimes. To date, methods for dynamic treatment regimes have been developed under the…

Methodology · Statistics 2022-02-22 Janie Coulombe , Erica E. M. Moodie , Susan M. Shortreed , Christel Renoux

Selecting causal inference models for estimating individualized treatment effects (ITE) from observational data presents a unique challenge since the counterfactual outcomes are never observed. The problem is challenged further in the…

Machine Learning · Computer Science 2021-02-15 Trent Kyono , Ioana Bica , Zhaozhi Qian , Mihaela van der Schaar

An important objective in the development of targeted therapies is to identify the populations where the treatment under consideration has positive benefit risk balance. We consider pivotal clinical trials, where the efficacy of a treatment…