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Sample size estimation of multiregional clinical trials with heterogeneous variability across regions.

Publication ,  Journal Article
Wu, Y-J; Tan, T-S; Chow, S-C; Hsiao, C-F
Published in: J Biopharm Stat
2014

In recent years, global collaboration has become a conventional strategy for new drug development. To accelerate the development process and shorten approval time, the design of multiregional clinical trials (MRCTs) incorporates subjects from many countries around the world under the same protocol. After showing the overall efficacy of a drug in all global regions, one can also simultaneously evaluate the possibility of applying the overall trial results to all regions and subsequently support drug registration in each of them. Several statistical methods have been proposed for the design and evaluation of MRCTs. Most of these approaches, however, assume a common variability of the primary endpoint across regions. In practice, this assumption may not be true, due to differences across regions (e.g., differences in ethnic factors and/or medical culture/practice). In this article, we use a random-effect model for modeling heterogeneous variability across regions for the design and evaluation of MRCTs. We also address consideration on the determination of the number of subjects in a specific region to establish the consistency of treatment effects between the specific region and the entire group.

Duke Scholars

Published In

J Biopharm Stat

DOI

EISSN

1520-5711

Publication Date

2014

Volume

24

Issue

2

Start / End Page

254 / 271

Location

England

Related Subject Headings

  • Treatment Outcome
  • Statistics & Probability
  • Sample Size
  • Research Design
  • Randomized Controlled Trials as Topic
  • Internationality
  • Humans
  • Double-Blind Method
  • 4905 Statistics
  • 3214 Pharmacology and pharmaceutical sciences
 

Citation

APA
Chicago
ICMJE
MLA
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Wu, Y.-J., Tan, T.-S., Chow, S.-C., & Hsiao, C.-F. (2014). Sample size estimation of multiregional clinical trials with heterogeneous variability across regions. J Biopharm Stat, 24(2), 254–271. https://doi.org/10.1080/10543406.2013.859150
Wu, Yuh-Jenn, Te-Sheng Tan, Shein-Chung Chow, and Chin-Fu Hsiao. “Sample size estimation of multiregional clinical trials with heterogeneous variability across regions.J Biopharm Stat 24, no. 2 (2014): 254–71. https://doi.org/10.1080/10543406.2013.859150.
Wu Y-J, Tan T-S, Chow S-C, Hsiao C-F. Sample size estimation of multiregional clinical trials with heterogeneous variability across regions. J Biopharm Stat. 2014;24(2):254–71.
Wu, Yuh-Jenn, et al. “Sample size estimation of multiregional clinical trials with heterogeneous variability across regions.J Biopharm Stat, vol. 24, no. 2, 2014, pp. 254–71. Pubmed, doi:10.1080/10543406.2013.859150.
Wu Y-J, Tan T-S, Chow S-C, Hsiao C-F. Sample size estimation of multiregional clinical trials with heterogeneous variability across regions. J Biopharm Stat. 2014;24(2):254–271.

Published In

J Biopharm Stat

DOI

EISSN

1520-5711

Publication Date

2014

Volume

24

Issue

2

Start / End Page

254 / 271

Location

England

Related Subject Headings

  • Treatment Outcome
  • Statistics & Probability
  • Sample Size
  • Research Design
  • Randomized Controlled Trials as Topic
  • Internationality
  • Humans
  • Double-Blind Method
  • 4905 Statistics
  • 3214 Pharmacology and pharmaceutical sciences