November 30, 2023 Webinar
Our Most Important Discovery: the Question
Scott Evans, PhD
Abstract
Randomized clinical trials are the gold standard for evaluating the benefits and harms of interventions, though often fail to provide the practical evidence to inform medical decision-making. A primary reason is failure to recognize the most important questions for treating patients in clinical practice, and using this as the motivation for the design, monitoring, analysis, and reporting of clinical trials. Standard approaches synthesizing information obtained from separate marginal analysis of each outcome, fail to incorporate associations between outcomes and recognize the cumulative nature of outcomes in individual patients, suffer from competing risk complexities during interpretation of individual outcomes, and since efficacy and safety analyses are often conducted on different populations, generalizability of benefit-risk is unclear. A most promising opportunity is identifying and placing increased interest on questions of a pragmatic origin to match their clinical importance and making corresponding adjustments to the design and analysis of clinical trials. The utility of clinical trials is enhanced by: (i) using outcomes to analyze patients rather than patients to analyze the outcomes, and (ii) incorporating benefit:risk evaluation into standard trial design and conduct, rather than being conducted as a post-hoc exercise. The desirability of outcome ranking (DOOR), a new paradigm for the design, analysis, and interpretation of clinical trials based on a comprehensive patient-centric benefit-risk evaluation, is described to address these issues and advance clinical trial science.
Short Bio
Dr. Scott Evans is the Director of The Biostatistics Center, and a Professor and the Founding Chair of the Department of Biostatistics Bioinformatics at the Milken Institute School of Public Health of the George Washington University. He is the: Director of the SDMC for the Antibacterial Resistance Leadership Group (ARLG); PI of the Coordinating Center for the ENICTO Consortium; Co-Chair of the Benefit-Risk Balance for Medicinal Products Working Group of the Council for International Organizations of Medical Sciences (CIOMS); Editor of a mini-Series on DSMBs for the NEJM Evidence, and the President-elect of the Society for Clinical Trials. He is a recipient of the Mosteller Statistician Award, the Zackin Distinguished Collaborative Statistician Award, the American Statistical Association's (ASA) Founders Award, and is a Fellow of the ASA, SCT, ISI, and the IDSA.