TITLE: C’mon in … The DOOR is Open: Pragmatic Benefit:Risk Evaluation Using Outcomes to Analyze Patients Rather than Patients to Analyze Outcomes
SPEAKER: Scott Evans and Toshimitsu Hamasaki, George Washington University


Randomized clinical trials are the gold standard for evaluating the benefits and harms of interventions. However, they often fail to provide the necessary evidence to inform medical decision-making. The important implications of these deficiencies are largely absent from discourse in medical research and biostatistical communities. Typical analyses of clinical trials involve intervention comparisons for each efficacy and safety outcome. Outcome-specific effects are estimated and marginal effects are potentially combined in benefit:risk analyses. It is widely believed that such analyses provide comprehensive information regarding the intervention effects on patients. However such approaches do not incorporate associations between outcomes of interest, suffer from competing risk challenges when interpreting outcome-specific results, do not recognize the cumulative nature of multiple outcomes on individual patients, and since efficacy and safety analyses are often conducted using different analysis populations, the population to which such benefit:risk analyses apply, is unclear. This deficit will be remedied in future clinical trials using thoughtful approaches with pragmatic foci. The desirability of outcome ranking (DOOR) and partial credit methodologies offer more informed patient-centric evaluation of intervention effects, providing greater utility for informing clinical decision-making. Critical components of this vision include: (i) revising the typical data analysis arithmetic to use outcomes to analyze patients rather than patients to analyze outcomes, (ii) sensitivity and robustness analyses to patient values, and (iii) identifying and evaluating subgroups based on patient-centric benefit:risk. Crucial to this approach entails improved understanding of how to analyze one patient before analyzing many. We discuss issues on the design, conduct, analyses, and reporting of clinical trials using these methods including sample size considerations, missing data and censoring considerations, and estimating subgroup effects.


Instructors’ Biography:

Professor Evans interests include the design, monitoring, analyses, and reporting of and education in clinical trials and diagnostic studies. He is the author of more than 150 peer-reviewed publications and three textbooks on clinical trials including Fundamentals for New Clinical Trialists. He is the Director of the Statistical and Data Management Center (SDMC) for the Antibacterial Resistance Leadership Group (ARLG), a collaborative clinical research network that prioritizes, designs, and executes clinical research to reduce the public health threat of antibacterial resistance. Listen to Dr. Evans on NPR talk about his work with superbug infections: Stats and Stories Podcast

Professor Evans is a former member of the Board of Directors for the American Statistical Association (ASA) and formerly for the Society for Clinical Trials (SCT) and Mu Sigma Rho, the National Honorary Society for Statistics. He is a former member of an FDA Advisory Committee, a member of the Steering Committee of the Clinical Trials Transformation Initiative (CTTI), and a member of the Execution Committee for the Analgesic, Anesthetic, and Addiction Clinical Trial Translations, Innovations, and Networks (ACTTION). He serves as the co-Chair of the Benefit-Risk Balance for Medicinal Products Working Group of the Council for International Organizations of Medical Sciences (CIOMS).

Professor Evans is the Editor-in-Chief of Statistical Communications in Infectious Diseases (SCID), and the Co-Editor of a Special Section of Clinical Infectious Diseases (CID) entitled Innovations in Design, Education, and Analysis (IDEA). He is the former Editor of CHANCE.

Professor Evans is a recipient of the Mosteller Statistician of the Year Award, the Robert Zackin Distinguished Collaborative Statistician Award for contributions to the AIDS Clinical Trials Group (ACTG), an elected member of the International Statistical Institute (ISI), and is a Fellow of the ASA, SCT, and the Infectious Disease Society of America (IDSA).


Toshimitsu Hamasaki is a Research Professor of the George Washington University (GWU) Biostatistics Center and the Department of Biostatistics and Bioinformatics. Prior to joining GWU, he worked at Shiogoni, Pfizer, Osaka University and National Cerebral and Cardiovascular Center. His research interests include the design, monitoring, analyses, and reporting of clinical trials. He is the author of more than 200 peer-reviewed publications and four textbooks on statistical methods in clinical trials including “Sample Size Determination in Clinical Trials with Multiple Endpoints” and “Group-Sequential Clinical Trials with Multiple Co-Objectives”.

Dr. Hamasaki is the Editor-in-Chief of Statistics in Biopharmaceutical Research, an Official Publication of the American Statistical Association (ASA). He also currently serves as an Editor for Statistical Communications in Infectious Diseases and an Editorial Board Member for Contemporary Clinical Trials. He served as an Associate Editor or Editor for several international journals including Statistics in Biopharmaceutical Research, Journal of Biopharmaceutical Statistics, CHANCE, and so on.

Dr. Hamasaki is an elected member of International Statistical Institute (ISI) and a fellow of the ASA. He received the Distinguished Article Award from the Japanese Society of Computational Statistics and Hida-Mizuno Prize from the Behaviormetric Society of Japan.

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