Evolving approaches to choice of outcomes in clinical trials: win ratio, repeat events, etc
Many clinical trials have a composite outcome which is conventionally analysed as time-to-first-event. Better alternatives can put the events in a clinical hierarchy, leading to a win ratio analysis and/or make use of repeat events, e.g. hospitalisations. These evolving techniques will be described with examples from recent trials in cardiovascular diseases.
Stuart Pocock, LSHTM
Stuart Pocock is Professor of Medical Statistics (since 1989) at the London School of Hygiene and Tropical Medicine, a leading European centre of excellence for biostatistical research and teaching.
His primary research interest concerns clinical trials, both as regards methodological developments and applied collaboration in major trials, mainly in cardiology. He also has interests in observational epidemiology especially pharmaco-epidemiology. His particular methodological areas of expertise include: standards for the statistical reporting of trials and epidemiological studies, the statistical ethical and organisational principles for data monitoring including early stopping guidelines, the win ratio approach to a hierarchical composite of outcomes with clinical priorities, adaptive designs, handling non-proportional hazards, the value of repeat-event analyses, use and misuse of propensity scores, the pros and cons of non-inferiority trials, problems of multiplicity in trial reporting, eg, subgroup analyses, multiple outcomes and covariate adjustment, and the development/validation of prognostic risk scores.
Professor Pocock and his colleagues run a statistical centre for the design, conduct, analysis and reporting of major clinical trials, especially in cardiovascular diseases. He is also a consultant statistician for a wider range of clinical trials in which expert statistical advice is needed, and serves as a statistical member of many trial data monitoring and steering committees.
He collaborates internationally especially with the Centro Nacional de Investigaciones Cardiovasculares in Madrid, and the Cardiovascular Research Foundation and Mount Sinai School of Medicine in New York. He is a frequent lecturer on a variety of clinical trials issues, and has published over 600 articles in peer-reviewed journals, and a popular textbook “Clinical Trials: a Practical Approach”.