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Statistical Considerations in Setting Quality Specification Limits Using Quality Data Read chapter Read first chapter

2019 | OriginalPaper | Chapter

Adaptive Three-Stage Clinical Trial Design for a Binary Endpoint in the Rare Disease Setting

Authors : Lingrui Gan, Zhaowei Hua

Published in: Pharmaceutical Statistics

Publisher: Springer International Publishing

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Abstract

A fundamental challenge in developing therapeutic agents for rare diseases is the limited number of eligible patients. A conventional randomized clinical trial may not be adequately powered if the sample size is small and asymptotic assumptions needed to apply common test statistics are violated. This paper proposes an adaptive three-stage clinical trial design for a binary endpoint in the rare disease setting. It presents an exact unconditional test statistic to generally control Type I error when sample size is small while not sacrificing power. Adaptive randomization has the potential to increase power by allocating greater numbers of patients to a more effective treatment. Performance of the method is illustrated using simulation studies.

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previous chapter Sample Size Determination Under Non-proportional Hazards
next chapter Clinical Trial Designs to Evaluate Predictive Biomarkers: What’s Being Estimated?
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Metadata
Title
Adaptive Three-Stage Clinical Trial Design for a Binary Endpoint in the Rare Disease Setting
Authors
Lingrui Gan
Zhaowei Hua
Copyright Year
2019
Book
Pharmaceutical Statistics

Print ISBN: 978-3-319-67385-1

Electronic ISBN: 978-3-319-67386-8

Copyright Year: 2019

DOI
https://doi.org/10.1007/978-3-319-67386-8_13

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