Optimal two-stage design for a series of pilot trials of new agents.
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.
Yao
T J
TJ
Department of Epidemiology and Biostatistics, Memorial Sloan-Kettering Cancer Center, New York, New York 10021, USA. yao@biost.mskcc.org
Venkatraman
E S
ES
D001699:Biometry / Q000379:methods*; D019496:Cancer Vaccines / Q000276:immunology / Q000627:therapeutic use; D002986:Clinical Trials as Topic / Q000706:statistics & numerical data*; D006801:Humans; D008545:Melanoma / Q000276:immunology / Q000628:therapy; D009369:Neoplasms / Q000276:immunology / Q000628:therapy; D010865:Pilot Projects; D018401:Sample Size
D019496:Cancer Vaccines