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Related papers: A Likelihood Perspective on Dose-Finding Study Des…

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We study sequential change-point detection procedures based on linear sketches of high-dimensional signal vectors using generalized likelihood ratio (GLR) statistics. The GLR statistics allow for an unknown post-change mean that represents…

Machine Learning · Computer Science 2018-05-01 Yang Cao , Andrew Thompson , Meng Wang , Yao Xie

We develop and evaluate tolerance interval methods for dynamic treatment regimes (DTRs) that can provide more detailed prognostic information to patients who will follow an estimated optimal regime. Although the problem of constructing…

Methodology · Statistics 2017-04-26 Daniel J. Lizotte , Arezoo Tahmasebi

Introduction: Large language models (LLM) have shown great potential in clinical decision support. GPT-5 is a novel LLM system that has been specifically marketed towards oncology use. Methods: Performance was assessed using two…

Randomized Controlled Trials (RCT) are the current gold standards to empirically measure the effect of a new drug. However, they may be of limited size and resorting to complementary non-randomized data, referred to as observational, is…

Methodology · Statistics 2025-06-11 Ahmed Boughdiri , Julie Josse , Erwan Scornet

Rerandomization is an experimental design technique that repeatedly randomizes treatment assignments until covariates are balanced between treatment groups. Rerandomization in the design stage of an experiment can lead to many asymptotic…

Methodology · Statistics 2026-04-10 Antônio Carlos Herling Ribeiro Junior

Treatment of cancer has rapidly evolved over time in quite dramatic ways, for example from chemotherapies, targeted therapies to immunotherapies and chimeric antigen receptor T-cells. Nonetheless, the basic design of early phase I trials in…

Applications · Statistics 2024-12-04 Lukas Andreas Widmer , Sebastian Weber , Yunnan Xu , Hans-Jochen Weber

Benchmark dose (BMD; a dose associated with a specified change in response) is used to determine the point of departure for the acceptable daily intake of substances for humans. Multiple dose-response relationship models are considered in…

Computation · Statistics 2025-05-13 Sota Minewaki , Tomohiro Ohigashi , Takashi Sozu

Interval estimation of quantiles has been treated by many in the literature. However, to the best of our knowledge there has been no consideration for interval estimation when the data are available in grouped format. Motivated by this, we…

Applications · Statistics 2017-12-08 Dilanka S. Dedduwakumara , Luke A. Prendergast

Clinical trial matching is a key process in health delivery and discovery. In practice, it is plagued by overwhelming unstructured data and unscalable manual processing. In this paper, we conduct a systematic study on scaling clinical trial…

Predicting cancer treatment outcomes requires models that are both accurate and interpretable, particularly in the presence of heterogeneous clinical data. While large language models (LLMs) have shown strong performance in biomedical NLP,…

Computation and Language · Computer Science 2025-10-21 Raghu Vamshi Hemadri , Geetha Krishna Guruju , Kristi Topollai , Anna Ewa Choromanska

Phase I dose escalation trials in oncology generally aim to find the maximum tolerated dose (MTD). However, with the advent of molecular targeted therapies and antibody drug conjugates, dose limiting toxicities are less frequently observed,…

Methodology · Statistics 2025-08-19 Ayon Mukherjee , Jonathan L. Moscovici , Zheng Liu

Phase I oncology trials aim to identify a safe dose - often the maximum tolerated dose (MTD) - for subsequent studies. Conventional designs focus on population-level toxicity modeling, with recent attention on leveraging pharmacokinetic…

Applications · Statistics 2026-01-13 Kyong Ju Lee , Yuan Ji

The recently developed semi-parametric generalized linear model (SPGLM) offers more flexibility as compared to the classical GLM by including the baseline or reference distribution of the response as an additional parameter in the model.…

Methodology · Statistics 2024-04-09 Entejar Alam , Peter Müller , Paul J. Rathouz

Phase I dose-finding trials in oncology seek to find the maximum tolerated dose (MTD) of a drug under a specific schedule. Evaluating drug-schedules aims at improving treatment safety while maintaining efficacy. However, while we can…

Large pre-trained language models (LLMs) have been shown to have significant potential in few-shot learning across various fields, even with minimal training data. However, their ability to generalize to unseen tasks in more complex fields,…

Computation and Language · Computer Science 2023-04-24 Tianhao Li , Sandesh Shetty , Advaith Kamath , Ajay Jaiswal , Xianqian Jiang , Ying Ding , Yejin Kim

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

Traditionally, the major objective in phase I trials is to identify a working-dose for subsequent studies, whereas the major endpoint in phase II and III trials is treatment efficacy. The dose sought is typically referred to as the maximum…

Methodology · Statistics 2016-08-14 Mourad Tighiouart , André Rogatko

The linear Markov Decision Process (MDP) framework offers a principled foundation for reinforcement learning (RL) with strong theoretical guarantees and sample efficiency. However, its restrictive assumption-that both transition dynamics…

Machine Learning · Statistics 2025-06-03 Sinian Zhang , Kaicheng Zhang , Ziping Xu , Tianxi Cai , Doudou Zhou

Large Language Models (LLMs) are increasingly employed in real-world applications, driving the need to evaluate the trustworthiness of their generated text. To this end, reliable uncertainty estimation is essential. Leading uncertainty…

Machine Learning · Computer Science 2026-04-21 Lukas Aichberger , Kajetan Schweighofer , Sepp Hochreiter

We consider the problem of testing for a dose-related effect based on a candidate set of (typically nonlinear) dose-response models using likelihood-ratio tests. For the considered models this reduces to assessing whether the slope…

Methodology · Statistics 2015-10-22 Georg Gutjahr , Björn Bornkamp
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