SOP 09: Statistical Design and Analysis

Statistical design and analysis of a CESAR study on the efficacy and/or safety of an anticancer drug should comply with ICC-GCP guidelines on statistical principles for clinical trials. Therefore, when not stated otherwise explicitly, the ICH-E9 guideline [1] and its rules will be applied to a CESAR study. The subsequent paragraphs will give specific guidance to special issues of CESAR trials. The respective ICH-E9 guideline will be cited below where applicable in its issue from February 1998. The biometric center (BC) of CESAR is responsible for contributing biostatistical expertise during the planning and the analysis stage of a CESAR study. Its mission is to support and to promote the scientific advancement of anticancer drug research. The tasks of the BC are guided by the primary objective(s) of a study and the goal of accomplishing a successful clinical trial. Therefore, the BC contributes especially to – The statistical design of the study and the determination of the minimal number of patients required, – The standardized documentation and the recording of the study data in accordance with the trial protocol, – The computerized storage, processing, and statistical analysis of the data, – The interpretation and reporting of the trial results in cooperation with the sponsor, the monitor, the Principal Investigator, and the clinical investigators. Specifically the BC is committed – For phase I studies to the Determination of appropriate dose ranges and identification of the maximal tolerable dose (MTD), Drug safety analysis; – For phase II studies to the Determination of the anticancer activity of the trial drug in terms of objective (measurable) criteria, Drug safety analysis; – For phase III studies to the Determination of a treatment effect in comparative randomized clinical trials on anti-cancer drug efficacy, Drug safety analysis.