Utility and Challenges of Applying Quantitative Benefit-Risk Assessment for Regulatory Decision Making

• Activity Number: 631 - Clinical Trial Design- 7
• Type: Contributed
• Date/Time: Thursday, August 2, 2018 : 8:30 AM to 10:20 AM
• Sponsor: Biopharmaceutical Section
• Author(s): Weili He and Bo Fu* and John Scott
• Companies: AbbVie and Astellas Pharma Inc. and FDA
• Keywords: Benefit-risk assessment; quantitative BR assessment ; qualitative BR assessment; PDUFA-V; Common Technical Document

Abstract

In recent years, there has been a proliferation of regulatory and industry-wide initiatives on structured benefit-risk (BR) assessment. Examples of structured BR frameworks include the PrOACT-URL from EMA Work Package 3, FDA CDRH)/ CBER Benefit-Risk Guidance, and FDA CDER/CBER Draft Prescription Drug User Fee Act (PDUFA-V) Implementation Plan on Structured Approach to BR Assessment in Drug Regulatory Decision-Making, to name a few. In June 2016, the ICH Expert Working Group finalized the Common Technical Document (CTD) Section 2.5.6 on Benefit-Risk Evaluations. As a result of these efforts, the uptake and utilization of structured benefit-risk (BR) assessments has been increasing. However, the aforementioned BR frameworks are mostly qualitative in nature, and the utility of quantitative BR approaches has not been systemically explored, creating uncertainty about settings in which quantitative BR assessment could be optimally applied. In this talk, we will first discuss settings and case studies where quantitative BR approaches would provide clarity on BR trade-offs, and articulate challenges around utility.