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Open AccessJournal ArticleDOI

Two-stage designs for Phase 2 dose-finding trials.

TLDR
A Bayesian adaptive two-stage design for the efficient estimation of the maximum dose or the minimum effective dose in a dose-finding trial that allocates subjects in stage two according to the posterior distribution of the target dose location.
Abstract
We propose a Bayesian adaptive two-stage design for the efficient estimation of the maximum dose or the minimum effective dose in a dose-finding trial. The new design allocates subjects in stage two according to the posterior distribution of the target dose location. Simulations show that the proposed two-stage design is superior to equal allocation and to a two-stage strategy where only one dose is left in the second stage.

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Optimal designs for estimating the interesting part of a dose-effect curve

TL;DR: Compared with a traditional balanced design for this trial, it is shown that the optimal design is substantially more efficient, which implies either a gain in information, or essential savings in sample size.
Journal ArticleDOI

Implementation of maximin efficient designs in dose-finding studies.

TL;DR: This paper exemplifies the maximin efficient approach by considering a sigmoid Emax model describing a dose-response relationship and compare inferential precision with that obtained when using a uniform design.
Journal ArticleDOI

An Opioid for Depression

TL;DR: Buprenorphine is a somewhat unusual opioid, and its unusual properties led to its becoming a very important treatment agent for over 3 million opioid-dependent patients in the United States since it received approval from the Food and Drug Administration for this indication in 2002.
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Characterization of dose-response for count data using a generalized MCP-Mod approach in an adaptive dose-ranging trial.

TL;DR: A 'shapebased' two-stage adaptive trial design where the doses to be tested in the second stage are determined based on the correlation observed between efficacy of the doses tests in the first stage and a set of pre-specified candidate dose-response profiles is presented.
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Dose finding when the target dose is on a plateau of a dose–response curve: comparison of fully sequential designs

TL;DR: Which of the several known dose‐finding methods developed for oncology phase I trials is the most suitable when the dose–response curve plateaus, and which tends to spread the allocation among the doses on the plateau.
References
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Journal ArticleDOI

Bayesian data analysis.

TL;DR: A fatal flaw of NHST is reviewed and some benefits of Bayesian data analysis are introduced and illustrative examples of multiple comparisons in Bayesian analysis of variance and Bayesian approaches to statistical power are presented.
Journal ArticleDOI

Combining multiple comparisons and modeling techniques in dose-response studies.

TL;DR: A unified strategy to the analysis of data from dose- response studies is described which combines multiple comparison and modeling techniques and assumes the existence of several candidate parametric models and uses multiple comparison techniques to choose the one most likely to represent the true underlying dose-response curve, while preserving the family-wise error rate.
Journal ArticleDOI

Testing and estimation in flexible group sequential designs with adaptive treatment selection.

TL;DR: This paper review adaptive treatment selection based on combination tests and propose overall adjusted p-values and simultaneous confidence intervals and point estimation in adaptive trials is considered.
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