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Adaptive designs for clinical trials permit alterations to a study in response to accumulating data in order to make trials more flexible, ethical and efficient. These benefits are achieved while preserving the integrity and validity of the…

Adaptive designs have been proposed for clinical trials in which the nuisance parameters or alternative of interest are unknown or likely to be misspecified before the trial. Whereas most previous works on adaptive designs and mid-course…

统计方法学 · 统计学 2011-05-18 Jay Bartroff , Tze Leung Lai

We propose a powerful adaptive contrast test with ordinal constraint contrast coefficients determined by observed responses. The adaptive contrast test can perform using easily calculated contrast coefficients and existing statistical…

统计方法学 · 统计学 2021-09-23 Masahiro Kojima

A new approach to adaptive design of clinical trials is proposed in a general multiparameter exponential family setting, based on generalized likelihood ratio statistics and optimal sequential testing theory. These designs are easy to…

统计理论 · 数学 2011-05-25 Jay Bartroff , Tze Leung Lai

Dose-finding trials are a key component of the drug development process and rely on a statistical design to help inform dosing decisions. Triallists wishing to choose a design require knowledge of operating characteristics of competing…

统计计算 · 统计学 2025-03-11 Michael Sweeting , Daniel Slade , Dan Jackson , Kristian Brock

The graph based approach to multiple testing is an intuitive method that enables a study team to represent clearly, through a directed graph, its priorities for hierarchical testing of multiple hypotheses, and for propagating the available…

统计方法学 · 统计学 2025-01-07 Cyrus Mehta , Ajoy Mukhopadhyay , Martin Posch

FDA's Project Optimus initiative for oncology drug development emphasizes selecting a dose that optimizes both efficacy and safety. When an inferentially adaptive Phase 2/3 design with dose selection is implemented to comply with the…

应用统计 · 统计学 2024-12-12 Cong Chen , Mo Huang , Xuekui Zhang

An important issue for many economic experiments is how the experimenter can ensure sufficient power for rejecting one or more hypotheses. Here, we apply methods developed mainly within the area of clinical trials for testing multiple…

统计方法学 · 统计学 2021-08-06 Sebastian Jobjörnsson , Henning Schaak , Oliver Mußhoff , Tim Friede

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…

统计方法学 · 统计学 2025-07-17 James M. McGree , Antony M. Overstall , Mark Jones , Robert K. Mahar

Adaptive experiments use preliminary analyses of the data to inform further course of action and are commonly used in many disciplines including medical and social sciences. Because the null hypothesis and experimental design are…

统计方法学 · 统计学 2026-05-26 Tobias Freidling , Qingyuan Zhao , Zijun Gao

Randomized controlled trials (RCTs) can be used to generate guarantees on treatment effects. However, RCTs often spend unnecessary resources exploring sub-optimal treatments, which can reduce the power of treatment guarantees. To address…

计算机与社会 · 计算机科学 2024-10-16 Santiago Cortes-Gomez , Naveen Raman , Aarti Singh , Bryan Wilder

In this paper we consider two-stage adaptive dose-response study designs, where the study design is changed at an interim analysis based on the information collected so far. In a simulation study, two approaches will be compared for these…

统计方法学 · 统计学 2016-02-08 Emma McCallum , Björn Bornkamp

An early phase clinical trial is the first step in evaluating the effects in humans of a potential new anti-disease agent or combination of agents. Usually called "phase I" or "phase I/II" trials, these experiments typically have the…

统计方法学 · 统计学 2010-12-01 Peter F. Thall

Embedded systems interaction with environment inherently complicates understanding of requirements and their correct implementation. However, product uncertainty is highest during early stages of development. Design verification is an…

软件工程 · 计算机科学 2011-12-20 Hara Gopal Mani Pakala , Dr. Plh Varaprasad , Dr. Raju Kvsvn , Dr. Ibrahim Khan

Adaptive sample size re-estimation, early stopping, and trial re-design at interim analyses can reduce expected sample sizes in randomised trials. Cluster randomised trials, in which groups of participants are randomly allocated to…

统计方法学 · 统计学 2026-03-09 Samuel I. Watson , James Martin

This PhD thesis covers breakthroughs in several areas of adaptive experiment design: (i) (Chapter 2) Novel clinical trial designs and statistical methods in the era of precision medicine. (ii) (Chapter 3) Multi-armed bandit theory, with…

统计方法学 · 统计学 2022-05-20 Michael Sklar

While scaling test-time compute can substantially improve model performance, existing approaches either rely on static compute allocation or sample from fixed generation distributions. In this work, we introduce a test-time compute…

人工智能 · 计算机科学 2026-04-24 Bowen Zuo , Dongruo Zhou , Yinglun Zhu

Two useful strategies to speed up drug development are to increase the patient accrual rate and use novel adaptive designs. Unfortunately, these two strategies often conflict when the evaluation of the outcome cannot keep pace with the…

统计方法学 · 统计学 2018-07-24 Ruitao Lin , Ying Yuan

In adaptive clinical trials, the conventional confidence interval (CI) for a treatment effect is prone to undesirable properties such as undercoverage and potential inconsistency with the final hypothesis testing decision. Accordingly, as…

Mid-study design modifications are becoming increasingly accepted in confirmatory clinical trials, so long as appropriate methods are applied such that error rates are controlled. It is therefore unfortunate that the important case of…

应用统计 · 统计学 2014-05-08 Dominic Magirr , Thomas Jaki , Franz Koenig , Martin Posch
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