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 individual patient preferences. 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 patient 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.
Bio: Ph.D., 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 is the Co-Director of the Stanford Center for Innovative Study Design, the Biostatistics Core of the Stanford Cancer Institute, and Stanford BERD for the SPECTRUM program. His research areas are method development and application in clinical trials in oncology, neurology, cardiology and radiology, statistical evaluation of medical diagnostic tests, meta-analysis, and medical decision making. He published more than 300 research papers in peer-reviewed journals, edited two books and numerous book chapters. Dr. Lu is an elected fellow of the American Association for the Advancement of Science (AAAS) and the American Statistical Association (ASA). He was Professor of Health Research and Policy at Stanford University and the director of VA Cooperative Studies Program Palo Alto Coordinating Center (2009-2016), Professor of Biostatistics and Radiology at the University of California, San Francisco (1994-2009) and assistant professor at the University of Miami School of Medicine (1990-1994). He served as the 2014 President of the International Chinese Statistical Association (ICSA), the 2020 President of the WNAR, and was a past member of the FDA Peripheral and Central Nervous System (PCNS) Advisory Committee. He is an associate editor of the JCO Precision Oncology and a Co-editor of Cancer Research Section of the New England Journal of Statistics and Data Sciences. Dr. Lu received his BS in Mathematics from Fudan University, MS in Applied Mathematics from Shanghai Jiao Tong University, and Ph.D. in Biostatistics, from the University of California, Berkeley.