Benefit‐Risk Analysis for Decision‐Making: An Approach

The analysis of benefit and risk is an important aspect of decision‐making throughout the drug lifecycle. In this work, the use of a benefit‐risk analysis approach to support decision‐making was explored. The proposed approach builds on the qualitative US Food and Drug Administration (FDA) approach to include a more explicit analysis based on international standards and guidance that enables aggregation and comparison of benefit and risk on a common basis and a lifecycle focus. The approach is demonstrated on six decisions over the lifecycle (e.g., accelerated approval, withdrawal, and traditional approval) using two case studies: natalizumab for multiple sclerosis (MS) and bedaquiline for multidrug‐resistant tuberculosis (MDR‐TB).