On a Stepwise Quantitative Approach for Benefit-Risk Assessment

• Activity Number: 570 - More Emerging Topics in Benefit-Risk Assessment in Clinical Development Decision Making
• Type: Topic Contributed
• Date/Time: Wednesday, August 2, 2017 : 2:00 PM to 3:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Weili He* and Yaxuan Sun and Qing Li and Sabrina Wan
• Companies: Merck & Co., Inc. and Department of Statistics, Iowa State University, Ames, IA, USA and Merck & Co., Inc. and Merck & Co., Inc.
• Keywords: Benefit-risk assessment ; standardized scores ; probability of technical success ; framework ; joint evaluation ; benefit-risk trade-offs

Abstract

The field of structured benefit-risk assessment has evolved rapidly in the last few years with a great deal of regulatory and industry-wide initiatives. The available structured approaches to benefit-risk assessments exhibit and share many common elements in terms of defining decision problem and therapeutic context, identifying key benefit and risk factors, and interpreting and communicating benefit-risk findings. However, there is limited guidance with these initiatives that is specific to metrics and methods to conduct benefit-risk assessment. Although we agree that benefit-risk metrics and analysis methods may be specific to individual clinical settings, we believe that there are general principles that can be followed. In this article, we propose a stepwise quantitative approach for benefit-risk assessment. We applied the stepwise quantitative approach to three real case studies.