Evaluating benefit-risk. Clinical trials and handling missing data.

Evaluating benefit-risk, handling missing data, and a universal sample size formula for clinical trials.

Speakers: Jeetu Ganju, Ganju Clinical Trials, LLC, Independent Consultant | Ron Yu, Senior Director, Biostatistics, Gilead Sciences

Abstract: Combining endpoints hierarchically enables the addressing of various problems in clinical trials, ranging from (a) providing a single metric for evaluating benefit and risk, (b) handling missing data by accounting for the reason for missingness, and (c) accounting for relative priorities of multiple efficacy endpoints. For (c), we provide a universal sample size formula for parallel-group trials of fixed or variable length that holds across any kind of endpoint. The method is based on the Mann-Whitney U test of comparing patient data in a pairwise manner, and its extensions by other authors for combining endpoints hierarchically.

A CAPS Methods Core and Biostatistics and Bioinformatics Seminar.

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