Professor Ying Lu (Professor of Biomedical Data Science at Stanford University) will be talking about his work on a composite endpoint for treatment benefit according to patient preference.
Abstract:
Complex disorders usually affect multiple symptom domains measured by several outcomes. The importance of these outcomes is often different among patients. Current approaches integrate multiple outcomes without considering patient preferences at the individual level. In this talk, I presented a survey study of ALS patients to show the heterogeneity of functional symptom domains of ALSFRS-R from patient perspectives. I will then introduce a new composite Desirability of Outcome Ranking (DOOR) that integrates individual level ranking of outcome importance and define a winning probability measuring the overall treatment effect. A new composite endpoint named Patient-ranked Order of Function (PROOF) has been proposed for evaluation of efficacy of amyotrophic lateral sclerosis (ALS) clinical trials. We use both theoretical and empirical methods to examine the statistical properties of our method and to compare with conventional approaches. We conclude that the proposed composite DOOR properly reflects patient-level preferences and can be used in pivotal trials or comparative effectiveness trials for a patient-centered evaluation of overall treatment benefits.
van Eijk RPA, van den Berg LH, Lu Y. Composite endpoint for ALS clinical trials based on patient preference: Patient-Ranked Order of Function (PROOF). J Neurol Neurosurg Psychiatry. 2021 Dec 17:jnnp-2021-328194. doi: 10.1136/jnnp-2021-328194. Epub ahead of print. PMID: 34921121
Professor Ying Lu is a Professor of Biomedical Data Science in the Department of Biomedical Data Science, and by courtesy in the Department of Radiology and Department of Epidemiology and Population Health, Stanford University School of Medicine. He co-directs the Stanford Center for Innovative Study Design, BERD Research Methods Core of the SPECTRUM, and the Biostatistics Core of the Stanford Cancer Institute.
Dr. Lu is also an associate biostatistics editor of the JCO Precision Oncology, the co-editor of the Cancer Research Section of the New England Journal of Statistics in Data Science. He was the 2014 President of the International Chinese Statistical Association (ICSA) and the 2019 President of IBC WNAR. Dr. Lu is an elected fellow of the American Statistical Association and the American Association for the Advancement of Science, and recipient of the ICSA Outstanding Service Award, 2016, Healthstar Osteoporosis Medical Research (HOMA) Award, 2003, Evelyn Fix Memorial Award from the Department of Statistics at UC Berkeley, 1990
His research areas are biostatistics methodology and applications in clinical trials, statistical evaluation of medical diagnostic tests, radiology, osteoporosis, meta-analysis and medical decision making. He has published 300 research papers in peer-reviewed journals, edited two books and numerous book chapters.