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Adaptive sampling algorithms are modern and efficient methods that dynamically adjust the sample size throughout the optimization process. However, they may encounter difficulties in risk-averse settings, particularly due to the challenge…

最优化与控制 · 数学 2025-02-17 Sandra Pieraccini , Tommaso Vanzan

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…

统计方法学 · 统计学 2021-11-23 Trinetri Ghosh , Yanyuan Ma , Rui Song , Pingshou Zhong

In adaptive clinical trials, the conventional end-of-trial point estimate of a treatment effect is prone to bias, that is, a systematic tendency to deviate from its true value. As stated in recent FDA guidance on adaptive designs, it is…

Drug combination trials are increasingly common nowadays in clinical research. However, very few methods have been developed to consider toxicity attributions in the dose escalation process. We are motivated by a trial in which the…

统计方法学 · 统计学 2018-08-23 Jose L. Jimenez , Mourad Tighiouart , Mauro Gasparini

Standard A/B tests in online experiments face statistical power challenges when testing multiple candidates simultaneously, while adaptive experimental designs (AED) alone fall short in inferring experiment statistics such as the average…

机器学习 · 计算机科学 2025-06-04 Qining Zhang , Tanner Fiez , Yi Liu , Wenyang Liu

Precision medicine aims to tailor therapeutic decisions to individual patient characteristics. This objective is commonly formalized through dynamic treatment regimes, which use statistical and machine learning methods to derive sequential…

机器学习 · 统计学 2026-03-23 Sophia Yazzourh , Erica E. M. Moodie

Increasingly, medical research is dependent on data collected for non-research purposes, such as electronic health records data (EHR). EHR data and other large databases can be prone to measurement error in key exposures, and unadjusted…

统计方法学 · 统计学 2020-05-13 Kyunghee Han , Thomas Lumley , Bryan E. Shepherd , Pamela A. Shaw

Adaptive therapy improves cancer treatment by controlling the competition between sensitive and resistant cells through treatment holidays. This study highlights the critical role of treatment-holiday thresholds in adaptive therapy for…

其他定量生物学 · 定量生物学 2025-07-15 Lanfei Sun , Haifeng Zhang , Kai Kang , Xiaoxin Wang , Leyi Zhang , Yanan Cai , Changjing Zhuge , Lei Zhang

We consider the problem of obtaining D-optimal designs for factorial experiments with a binary response and $k$ qualitative factors each at two levels. We obtain a characterization for a design to be locally D-optimal. Based on this…

统计理论 · 数学 2015-03-19 Jie Yang , Abhyuday Mandal , Dibyen Majumdar

We consider conditional estimation in two-stage sample size adjustable designs and the following bias. More specifically, we consider a design which permits raising the sample size when interim results look rather promising, and, which…

统计方法学 · 统计学 2018-08-27 Per Broberg , Frank Miller

The majority of response-adaptive randomisation (RAR) designs in the literature rely on efficacy data to guide dynamic patient allocation. However, their applicability becomes limited in settings where efficacy outcomes, such as survival,…

统计方法学 · 统计学 2026-02-23 Maria Vittoria Chiaruttini , Lukas Pin , Sofia S. Villar

In single-arm phase II oncology trials, the most popular choice of design is Simon's two-stage design, which allows early stopping at one interim analysis. However, the expected trial sample size can be reduced further by allowing…

统计方法学 · 统计学 2019-09-09 Martin Law , Michael J. Grayling , Adrian P. Mander

This study investigates the contextual best arm identification (BAI) problem, aiming to design an adaptive experiment to identify the best treatment arm conditioned on contextual information (covariates). We consider a decision-maker who…

机器学习 · 计算机科学 2025-06-23 Masahiro Kato , Kyohei Okumura , Takuya Ishihara , Toru Kitagawa

We develop a novel iterative algorithm for locally optimal experimental design under constraints, like budget or performance constraints. It is an adaptive discretization algorithm. In every iteration, a discretized version of the…

最优化与控制 · 数学 2026-04-21 Jochen Schmid , Philipp Seufert , Jan Schwientek , Tobias Seidel , Karl-Heinz Küfer

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…

统计方法学 · 统计学 2018-12-11 Charles F. Manski , Aleksey Tetenov

Optimal designs can help experimenters obtain more accurate parameter estimates with reduced experimental time and cost. In this paper, we characterize the Expected Weighted (EW) D-optimal designs as robust designs against unknown parameter…

统计方法学 · 统计学 2026-04-08 Siting Lin , Yifei Huang , Jie Yang

In a sequential multiple-assignment randomized trial (SMART), a sequence of treatments is given to a patient over multiple stages. In each stage, randomization may be done to allocate patients to different treatment groups. Even though…

统计方法学 · 统计学 2024-01-09 Rik Ghosh , Bibhas Chakraborty , Inbal Nahum-Shani , Megan E. Patrick , Palash Ghosh

This paper shows that sequential statistical analysis techniques can be generalised to the problem of selecting between alternative forecasting methods using scoring rules. A return to basic principles is necessary in order to show that…

统计理论 · 数学 2025-05-15 David T. Frazier , Donald S. Poskitt

Adaptive experimentation enables efficient estimation of causal effects, but existing methods are not designed for survival data with censoring, where event times are only partially observed (e.g., overall survival in cancer trials but with…

机器学习 · 计算机科学 2026-05-19 Yuxin Wang , Dennis Frauen , Jonas Schweisthal , Maresa Schröder , Emil Javurek , Stefan Feuerriegel

Recently, the strategy for dose optimization in oncology has shifted to conduct Phase 2 randomized controlled trials with multiple doses. Optimal biologic dose selection from Phase 1 trial data to determine candidate doses for Phase 2…

统计方法学 · 统计学 2023-02-14 Masahiro Kojima