Optimal sample size for a series of pilot trials of new agents Journal Article


Authors: Yao, T. J.; Begg, C. B.; Livingston, P. O.
Article Title: Optimal sample size for a series of pilot trials of new agents
Abstract: A new approach is presented for determining the appropriate sample sizes for a series of screening trials to identify promising new therapeutic agents. The formulation of the problem is motivated by recognition of the fact that screening of new agents is a continuing process. Consequently, it does not seem ideal to fix the overall total sample size, as previous authors have done. Instead we fix the error rates and optimize the individual sample sizes to minimize the time to identify a promising agent, using an empirical Bayes formulation. When applied to data from the large historical experience of exploratory vaccination trials at Memorial Sloan Kettering Cancer Center, the method demonstrates that relatively small individual screening trials are optimal in this setting. The reliability of the results is evaluated using bootstrapping techniques.
Keywords: neoplasms; melanoma; bayes theorem; statistics; cancer vaccines; pilot projects; models, statistical; vaccination; screening test; clinical trials; biometry; sample size; sample; empirical bayes; humans; article; bootstrap estimation; screening trials
Journal Title: Biometrics
Volume: 52
Issue: 3
ISSN: 0006-341X
Publisher: Wiley Blackwell  
Date Published: 1996-09-01
Start Page: 992
End Page: 1001
Language: English
PUBMED: 8805764
PROVIDER: scopus
DOI: 10.2307/2533060
DOI/URL:
Notes: Article -- Export Date: 22 November 2017 -- Source: Scopus
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  1. Colin B Begg
    306 Begg
  2. Tzy-Jyun Yao
    59 Yao