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.
Original language | English (US) |
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Pages (from-to) | 118-132 |
Number of pages | 15 |
Journal | Statistical methods in medical research |
Volume | 25 |
Issue number | 1 |
DOIs | |
State | Published - Feb 1 2016 |
Keywords
- "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