SC-06 - Weighing the Benefits & Risks: The Science and Methods of Benefit-Risk Assessment in Drug Development

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 an introductory 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 drug development process 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 benefit-risk assessment including roles for preference studies
• Develop an understanding of basic methods for structured and quantitative benefit-risk assessment
• Develop an understanding of how patient preference methods can inform benefit-risk assessment
• Develop an understanding of how to successfully structure and present benefit-risk information for drug development decision-making and regulatory interactions
4. Syllabus Outline
Part 1: Intro and Benefit-Risk Assessment Didactic 85 minutes
• Introduction and Objectives for Course 5 minutes
• Overview of current regulatory background/guidances (FDA/EMA/CIOMS/ICH) 20 minutes
o What were the recent changes in regulations related to BR, what are the main similarities and differences
• Overview of benefit-risk methods 30 minutes
o What is a structured benefit-risk framework, overview of most common frameworks, what is difference between quantitative and qualitative, when quantitative is appropriate
o Example Framework Steps: FDA Framework, CIRS-BRAT Framework
• Industry perspective: Implementing benefit-risk assessment
o 30 minutes
 Understanding the challenges, the process, the methods used across product development (including early development), and success factors
 Use case of situations where benefit-risk assessment had the biggest impact
Part 2: Benefit-Risk

Assessment: Interactive Activity in small groups (includes time for break) 45 minutes

• Practicing benefit-risk assessment: Decision context, value tree development

Part 3: Patient Preference and their Role in Benefit-Risk Assessment Didactic 35 minutes
• Introduction to patient preference
o What does patient preference mean? How to measure patient preference ?
o How can patient preferences be integrated into benefit-risk ?
o Example of patient preference to support benefit-risk assessment

Part 4: Patient Preference Interactive Activity and Wrap Up 45 minutes

• Evaluating patient preference: collect and interpret preference data 35 minutes
• Wrap up, key takeaways and final questions 10 minutes