On a Stepwise Quantitative Approach for Benefit-Risk Assessment

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 the 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 3 real case studies.