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The problem of chemotherapy treatment optimization can be defined in order to minimize the size of the tumor without endangering the patient's health; therefore, chemotherapy requires to achieve a number of objectives, simultaneously. For…

Neural and Evolutionary Computing · Computer Science 2023-03-21 Mina Jafari , Behnam Ghavami , Vahid Sattari Naeini

Unblinded sample size re-estimation (SSR) is often planned in a clinical trial when there is large uncertainty about the true treatment effect. For Proof-of Concept (PoC) in a Phase II dose finding study, contrast test can be adopted to…

Methodology · Statistics 2022-11-11 Qingyang Liu , Guanyu Hu , Binqi Ye , Susan Wang , Yaoshi Wu

In traditional dose-finding studies, dose-limiting toxicity (DLT) is determined within a fixed time observation window where DLT is often defined as a binary outcome. In the setting of oncology dose-finding trials, often patients in…

Applications · Statistics 2019-09-09 Lucie Biard , Bin Cheng , Gulam A. Manji , Shing M. Lee

The study of combinations of drugs/drug-schedules gained increasing attention in various therapeutic areas recently. In oncology, the aim of phase I combination clinical trial is to find the maximum tolerated combination (MTC). Many…

Methodology · Statistics 2025-05-09 Weishi Chen , Pavel Mozgunov

Optimization is offered as an objective approach to resolving complex, real-world decisions involving uncertainty and conflicting interests. It drives business strategies as well as public policies and, increasingly, lies at the heart of…

Artificial Intelligence · Computer Science 2023-08-01 Benjamin Laufer , Thomas Krendl Gilbert , Helen Nissenbaum

I study the minimax-optimal design for a two-arm controlled experiment where conditional mean outcomes may vary in a given set. When this set is permutation symmetric, the optimal design is complete randomization, and using a single…

Methodology · Statistics 2020-05-08 Nathan Kallus

We mathematically justify metronomic chemotherapy as the best strategy to apply most cytotoxic drugs in oncology for both curative and palliative approaches, assuming the classical pharmacokinetic model together with the Emax…

Optimization and Control · Mathematics 2021-10-15 Luis A. Fernández , Cecilia Pola , Judith Sáinz-Pardo

The era of big data is coming, and evidence-based medicine is attracting increasing attention to improve decision making in medical practice via integrating evidence from well designed and conducted clinical research. Meta-analysis is a…

Methodology · Statistics 2016-10-05 Dehui Luo , Xiang Wan , Jiming Liu , Tiejun Tong

We study sequential experiments where sampling is costly and a decision-maker aims to determine the best treatment for full scale implementation by (1) adaptively allocating units between two possible treatments, and (2) stopping the…

Econometrics · Economics 2025-05-06 Karun Adusumilli

The paper develops methods to construct a one-stage optimal design of dilution experiments under the total available volume constraint typical for bio-medical applications. We consider various design criteria based on the Fisher information…

Statistics Theory · Mathematics 2013-05-08 Maryam Zolghadr , Sergei Zuyev

In multi-stage radiotherapy, a patient is treated in several stages separated by weeks or months. This regimen has been motivated mostly by radiobiological considerations, but also provides an approach to reduce normal tissue dose by…

We propose a frequentist adaptive phase 2 trial design to evaluate the safety and efficacy of three treatment regimens (doses) compared to placebo for four types of helminth (worm) infections. This trial will be carried out in four…

We study the problem of learning individualized dose intervals using observational data. There are very few previous works for policy learning with continuous treatment, and all of them focused on recommending an optimal dose rather than an…

Methodology · Statistics 2022-02-25 Guanhua Chen , Xiaomao Li , Menggang Yu

Combination therapies are often needed for effective clinical outcomes in the management of complex diseases, but presently they are generally based on empirical clinical experience. Here we suggest a novel application of search algorithms,…

Models often need to be constrained to a certain size for them to be considered interpretable. For example, a decision tree of depth 5 is much easier to understand than one of depth 50. Limiting model size, however, often reduces accuracy.…

Machine Learning · Computer Science 2020-07-02 Abhishek Ghose , Balaraman Ravindran

Testing the equivalence of multiple quantiles between two populations is important in many scientific applications, such as clinical trials, where conventional mean-based methods may be inadequate. This is particularly relevant in bridging…

Methodology · Statistics 2025-10-21 Jun Wu , Stéphane Guerrier , Si Gou , Yogeshvar N. Kalia , Luca Insolia

A central goal in designing clinical trials is to find the test that maximizes power (or equivalently minimizes required sample size) for finding a false null hypothesis subject to the constraint of type I error. When there is more than one…

Methodology · Statistics 2022-09-21 Ruth Heller , Abba Krieger , Saharon Rosset

Optimal block designs in small blocks are explored when the treatments have a natural ordering and interest lies in comparing consecutive pairs of treatments. We first develop an approximate theory which leads to a convenient multiplicative…

Statistics Theory · Mathematics 2014-05-20 S. Huda , Rahul Mukerjee

Phase I dose-finding trials are increasingly challenging as the relationship between efficacy and toxicity of new compounds (or combination of them) becomes more complex. Despite this, most commonly used methods in practice focus on…

Machine Learning · Computer Science 2020-06-16 Cong Shen , Zhiyang Wang , Sofia S. Villar , Mihaela van der Schaar

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
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