Abstract
Biomarkers play a key role in the development of personalized medicine. Cancer clinical trials with biomarker should be appropriately designed and analyzed reflecting the various factors, such as the phase of trials, the type of biomarker, the study objectives, and whether the used biomarker is already validated or not. In this paper, we demonstrate design and analysis of two phase II cancer clinical trials, one with a predictive biomarker and the other with a prognostic biomarker. A statistical testing method and its sample size calculation method are presented for each of the trials. We assume that the primary endpoint of these trials is a time to event variable, but this concept can be used for any type of endpoint with associated testing methods. The test statistics and their sample size formulas are derived using the large sample approximation based on the martingale central limit theorem. Using simulations, we find that the test statistics control the type I error rate accurately and the sample sizes calculated using the formulas maintain the statistical power specified at the design stage.
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