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Considerations for pediatric trial designs and analyses.

Authors
  • Cho, Meehyung1
  • Qiu, Zhiying1
  • Ye, Jenny1
  • Quan, Hui1
  • Zhao, Peng-Liang1
  • 1 a Biostatistics and Programming , Sanofi US , Bridgewater , NJ , USA.
Type
Published Article
Journal
Journal of Biopharmaceutical Statistics
Publisher
Informa UK (Taylor & Francis)
Publication Date
Sep 05, 2017
Pages
1–24
Identifiers
DOI: 10.1080/10543406.2017.1372771
PMID: 28872409
Source
Medline
Keywords
License
Unknown

Abstract

There are challenges in designing pediatric trials arising from special ethical issues and the relatively small accessible patient population. The application of conventional phase 3 trial designs to pediatrics is not realistic in some therapeutic areas. To address this issue, we propose various approaches for designing pediatric trials that incorporate data available from adult studies using James-Stein shrinkage estimation, empirical shrinkage estimation, and Bayesian methods. We also apply the concept of consistency used in multi-regional trials to pediatric trials. The performance of these methods is assessed through representative scenarios and an example using actual Type 2 diabetes mellitus (T2DM) trials.

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