Optimal two-stage design for a series of pilot trials of new agents

Biometrics. 1998 Sep;54(3):1183-9.

Abstract

An approach to determine the appropriate sample sizes for a series of screening trials to identify promising new therapeutic agents was presented by Yao, Begg, and Livingston (1996, Biometrics 52, 992-1001). This approach is now improved to a two-stage design that further minimizes the time to identify a promising agent under fixed error rates. When applied to data from the historical experience of exploratory vaccination trials at Memorial Sloan-Kettering Cancer Center, the method demonstrates that relatively small individual screening trials are optimal. The reliability of the results is evaluated using the bootstrap.

Publication types

  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Biometry / methods*
  • Cancer Vaccines / immunology
  • Cancer Vaccines / therapeutic use
  • Clinical Trials as Topic / statistics & numerical data*
  • Humans
  • Melanoma / immunology
  • Melanoma / therapy
  • Neoplasms / immunology
  • Neoplasms / therapy
  • Pilot Projects
  • Sample Size

Substances

  • Cancer Vaccines