1. Course Aim Benefit-risk assessment is now a fundamental science for understanding and evaluating the development, approval, and continued marketing of drugs and medical devices. This course is aimed at introducing attendees to core concepts and tools of both the science and practice of benefit-risk assessment.
2. Requisites Statement This is a mid-level course designed to acquaint attendees with the current state of the art in the use of benefit-risk assessment methods and tools. Basic understanding of benefit-risk assessment concepts will be helpful for attendees to follow course content however there are no prerequisites for this course.
3. Course Objectives
Educational Objectives: • Develop an understanding of current regulatory policies regarding the use of structured and quantitative benefit-risk assessment including roles for preference studies • Develop an understanding of methods for structured and quantitative benefit-risk assessment • Develop an understanding of how pharmacoepidemiology and benefit-risk assessment intersects with preference methods • Develop an understanding of how to successfully structure and present benefit-risk information for drug development decision-making and regulatory interactions, including the use of visual tools 4. Syllabus Outline Tarek Hammad will focus on the “why” benefit-risk assessment is important – providing attendees with an overview of regulatory expectations and available guidance
Juhaeri Juhaeri will focus on the “how” of benefit-risk – providing attendees with an overview of existing frameworks for a structured benefit-risk assessment and overview of quantitative benefit-risk assessment methods (e.g., MCDA, SMAA)
Carol Mansfield will focus on how patient-preference information can be designed and applied to support benefit-risk assessment. Using case studies, the session will provide attendees with an overview of leading methods (e.g., discrete choice, direct elicitation, threshold technique) for the elicitation, analysis and presentation of patient preference information.
Bennett Levitan will focus on “putting it all together” – providing attendees with a conceptual overview and practical examples, including how patient preference information can be integrated with clinical data to inform decisions
Alicia Gilsenan will moderate the overall session as well as a 20-minute panel discussion focused on panel experience with structured benefit-risk assessments and opportunity for audience participation