This note explores the possibility of allowing an efficacy claim for a new drug based upon the results of two pivotal clinical trials if one trial is significant in the usual sense while the other just shows a trend in the right direction. We show that it is possible to select the type I errors that enable the individual trials to achieve this goal without a substantial increase in the overall type I error.
US Food and Drug Administration.Guidance for Industry: Providing Clinical Evidence for Effectiveness for Human Drug and Biological Products.Rockville, MD: US Department of Health and Human Services, Food and Drug Administration; 1998.
2.
Committee for Proprietary Medicinal Products.CPMP Points to Consider on Application with 1. Meta-analyses: 2. One Pivotal Study.London, England: European Agency for the Evaluation of Medicinal Products, Committee for Proprietary Medicinal Products; 2001.
3.
FisherRAStatistical Methods for Research Workers.4th edition.London, England: Oliver & Boyd; 1934.
