A new statistical decision rule for single-arm phase II oncology trials

Yiyi Chen, Zunqiu Chen, Motomi Mori

Research output: Contribution to journalArticlepeer-review

5 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)118-132
Number of pages15
JournalStatistical methods in medical research
Issue number1
StatePublished - Feb 1 2016


  • "Go/No Go" decision
  • Transitional decision
  • oncology trials
  • phase II clinical trials
  • testing confidence value

ASJC Scopus subject areas

  • Epidemiology
  • Statistics and Probability
  • Health Information Management


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