SCT Sponsored Webinar Series

  • Title: The DOOR is Open: Patient-Centric, Pragmatic Benefit: Risk Evaluation in Clinical Trials
  • Moderator: Scott Rushing, Director, Research Information Systems, Wake Forest University Medical Center
  • Faculty: 
    • Scott Evans, PhD, MS; Director, The Biostatistics Center; Professor and Founding Chair, Department of Biostatistics and Bioinformatics, George Washington University
    • Toshi Hamasaki; Professor, The Biostatistics Center and Department of Biostatistics and Bioinformatics, George Washington University
  • Date: June 26, 2024
  • Time: 1:00 – 2:00 pm ET
  • Description: 

    Randomized clinical trials are the gold standard for evaluating the benefits and harms of interventions but they often fail to provide the necessary evidence to inform medical decision-making. One reason for this is a failure to recognize the patient-centric benefitr:risk nature of medical-decision making. Typically in clinical trials efficacy and safety are evaluated in silos, one outcome at a time. However this approach: fails to incorporate associations between or the cumulative nature of multiple outcomes in individual patients, suffers from competing risk complexities during interpretation of individual outcomes, fails to recognize important gradations of patient responses, suboptimallyevaluates treatment effect heterogeneity based on a single endpoint rather than benefit:risk considerations, and since efficacy and safety analyses are often conducted on different populations, generalizability is unclear. In recognition of this, the Council for International Organizations of Medical Sciences (CIOMS) recently recommended: (1) transitioning benefit-risk evaluation as a post-hoc exercise to incorporating benefit-risk considerations into clinical trial design, and (2) a pragmatic patient-centric approach to benefit-risk assessment to ensure proper evaluation of the benefits and harms as experienced by patients. The desirability of outcome ranking (DOOR) is a paradigm for the design, analysis, and interpretation of clinical trials based on a comprehensive patient-centric benefit-risk evaluation developed to address these limitations and the CIOMS recommendations, and advance clinical trial science. In this paradigm outcomes are used to analyze patients rather than patients being used to analyze outcomes. The experiences of trial participants in different treatment arms are compared by the desirability of the overall patient outcome, increasing pragmatism and addressing the most important “real world” question to aid clinical decision-making: how do resulting patient experiences, when comprehensively considering benefits and harms, compare between therapeutic alternatives? The DOOR paradigm, and freely available online tools for design and robust analyses are discussed, andillustrated using examples.

    By the end of this webinar, participants should be able to: 
    • To understand the challenges of benefit: risk evaluation and the rational for the DOOR paradigm
    • To understand the basic concepts and principles of the DOOR approach 
    • To design and analyse clinical trials utilizing the DOOR methodology using free online tools