CLINICAL TRIALS
Design and Analysis of Cancer Clinical Trials for Personalised Medicine Cancer clinical trials for personalised medicine should be appropriately designed and analysed reflecting the various factors. Biomarkers play a key role in the development of personalised medicine. In this article, we review the design and analysis of two phase II cancer clinical trials of personalised medicine, one with a predictive biomarker and the other with a prognostic biomarker. We discuss statistical testing method and its sample size calculation method for each of the trials. Sin-Ho Jung, Department of Biostatistics and Bioinformatics Duke University School of Medicine
P
ersonalised medicine for caner is a treatment module tailored to individual patients based on various factors including biomarkers. Different types of biomarkers are measured from the tumour, blood or urine using molecular, biochemical, physiological, anatomical, or imaging method
before treatment or during the course of treatment. Observed biomarkers can be used for the selection of personalised treatment for cancer patients. For example, predictive biomarkers are used to predict the response to a specific treatment and prognostic biomarkers are used to measure the aggressiveness
of a disease for patients with no or a non-targeted treatment. These biomarkers can be used to select a treatment for cancer patients. However, biomarkers should be validated before being used to select a treatment in clinical trials. If a biomarker has not been validated through a clinical trial yet, it can be used as a stratification factor of a randomised clinical trial. In such a case, the biomarker can be validated through the trial. The methods required for design and analysis of a clinical trial with a biomarker-guided personalised medicine can be different depending on the type of the biomarker. In this sense, it is impossible to present a unified design and analysis method for clinical trials of various type of personalised medicines. Various design issues of randomised clinical trials with biomarkers has been widely discussed by Freidlin et al. (2010). In this article, we discuss design and analysis methods for two cancer clinical
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