Phase II dose–response trials: A simulation study to compare analysis method performance under design considerations

ABSTRACT Phase II trials are intended to provide information about the dose–response relationship and to support the choice of doses for a pivotal phase III trial. Recently, new analysis methods have been proposed to address these objectives, and guidance is needed to select the most appropriate analysis method in specific situations. We set up a simulation study to evaluate multiple performance measures of one traditional and three more recent dose-finding approaches under four design options and illustrate the investigated analysis methods with an example from clinical practice. Our results reveal no general recommendation for a particular analysis method across all design options and performance measures. However, we also demonstrate that the new analysis methods are worth the effort compared to the traditional ANOVA-based approach.

• Publication year

  2017

• Venue

  Journal of Biopharmaceutical Statistics

• Publication date

  2017-03-31

• Fields of study

  Mathematics, Medicine, Computer Science

• Identifiers
  DOI 10.1080/10543406.2017.1293078PMID 28362145
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar, PubMed

• No claims are published for this paper.

• No concepts are published for this paper.

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