title: A new statistical decision rule for single-arm phase II oncology trials.
abstract: Most single-arm phase II clinical trials compare the efficacy of a new treatment with historical controls through statistical hypothesis testing. One major problem with such a comparison is that the efficacy of the historical control is treated as a known constant, whereas in reality, it is never precisely known. This partially explains why many "Go" decisions made in single-arm phase II trials are shown to be incorrect in phase III trials. In this paper, we propose a new decision rule for an improved transitional decision for single-arm phase II oncology clinical trials with binary endpoints. This new decision rule is jointly based on the p value and a new statistical index named the testing confidence value. The testing confidence value reflects the uncertainty associated with the null value in the hypothesis testing of single-arm trials. Simulations are used to evaluate the operating characteristics of the new decision rule in comparison with the traditional decision rule and a widely used Bayesian decision rule. The application of the new decision rule is illustrated using a clinical trial on marginally resectable pancreatic cancer. A webpage http://www.yiyichenbiostatistics.com/TCV.html is available for readers to interactively compute the testing confidence value and to find the suggested decision based on the new decision rule.
authors.affiliation: Division of Biostatistics, Department of Public Health and Preventive Medicine, Oregon Health and Science University, Portland, OR, USA chenyiy@ohsu.edu.
authors.forename: Yiyi
authors.initials: Y
authors.lastname: Chen
authors.affiliation: Division of Biostatistics, Department of Public Health and Preventive Medicine, Oregon Health and Science University, Portland, OR, USA.
authors.forename: Zunqiu
authors.initials: Z
authors.lastname: Chen
authors.affiliation: Division of Biostatistics, Department of Public Health and Preventive Medicine, Oregon Health and Science University, Portland, OR, USA.
authors.forename: Motomi
authors.initials: M
authors.lastname: Mori
mesh_terms: D001499:Bayes Theorem; D056808:Biostatistics; D017322:Clinical Trials, Phase II as Topic / Q000706:statistics & numerical data*; D003661:Decision Support Techniques; D018572:Disease-Free Survival; D006801:Humans; D015233:Models, Statistical; D009369:Neoplasms / Q000628:therapy*; D010190:Pancreatic Neoplasms / Q000628:therapy; D016032:Randomized Controlled Trials as Topic / Q000706:statistics & numerical data