Skip to main content

Probability monitoring procedures for sample size determination.

Publication ,  Journal Article
Huang, Z; Chow, S-C
Published in: J Biopharm Stat
2019

In clinical research, power analysis is often performed for sample size calculation. The purpose is to achieve a desired power of correctly detecting a clinically meaningful difference at a pre-specified level of significance if such a difference truly exists. However, in some situations such as (i) clinical trials with extremely low incidence rates and (ii) for rare disease drug development clinical trials, power analysis for sample size calculation may not be feasible because (i) it may require a huge sample size for detecting a relatively small difference and (ii) eligible patients may not be available for a small target patient population. In these cases, other procedures for sample size determination with certain statistical assurance are needed. In this article, an innovative method based on a probability monitoring procedure is proposed for sample size determination. The concept is to select an appropriate sample size for controlling the probability of crossing safety and/or efficacy boundaries. For rare disease clinical development, an adaptive probability monitoring procedure may be applied if a multiple-stage adaptive trial design is used.

Duke Scholars

Altmetric Attention Stats
Dimensions Citation Stats

Published In

J Biopharm Stat

DOI

EISSN

1520-5711

Publication Date

2019

Volume

29

Issue

5

Start / End Page

887 / 896

Location

England

Related Subject Headings

  • Statistics & Probability
  • Sample Size
  • Rare Diseases
  • Probability
  • Humans
  • Drug Development
  • Drug Approval
  • 4905 Statistics
  • 3214 Pharmacology and pharmaceutical sciences
  • 1115 Pharmacology and Pharmaceutical Sciences
 

Citation

APA
Chicago
ICMJE
MLA
NLM
Huang, Z., & Chow, S.-C. (2019). Probability monitoring procedures for sample size determination. J Biopharm Stat, 29(5), 887–896. https://doi.org/10.1080/10543406.2019.1657139
Huang, Zhipeng, and Shein-Chung Chow. “Probability monitoring procedures for sample size determination.J Biopharm Stat 29, no. 5 (2019): 887–96. https://doi.org/10.1080/10543406.2019.1657139.
Huang Z, Chow S-C. Probability monitoring procedures for sample size determination. J Biopharm Stat. 2019;29(5):887–96.
Huang, Zhipeng, and Shein-Chung Chow. “Probability monitoring procedures for sample size determination.J Biopharm Stat, vol. 29, no. 5, 2019, pp. 887–96. Pubmed, doi:10.1080/10543406.2019.1657139.
Huang Z, Chow S-C. Probability monitoring procedures for sample size determination. J Biopharm Stat. 2019;29(5):887–896.

Published In

J Biopharm Stat

DOI

EISSN

1520-5711

Publication Date

2019

Volume

29

Issue

5

Start / End Page

887 / 896

Location

England

Related Subject Headings

  • Statistics & Probability
  • Sample Size
  • Rare Diseases
  • Probability
  • Humans
  • Drug Development
  • Drug Approval
  • 4905 Statistics
  • 3214 Pharmacology and pharmaceutical sciences
  • 1115 Pharmacology and Pharmaceutical Sciences