Advancing Structured Benefit-Risk Assessment in FDA Review

As part of a broader effort to increase the predictability, transparency, and efficiency of its decision-making process, FDA developed and piloted a structured qualitative framework for benefit-risk assessment starting in 2012. This framework is designed to help characterize and communicate uncertainties about a drug’s benefits and risks, and to reflect the iterative nature of the benefit-risk assessment process at FDA, where new findings can be incorporated into the framework as more information becomes available in the post-market setting. With the qualitative framework now in place, many stakeholders have expressed interest in the use of more quantitative approaches to benefit-risk assessment. However, there are ongoing questions over when and how these approaches can provide the greatest value in supporting FDA’s decision-making.

To support further progress in this area, the Duke-Margolis Center for Health Policy convened this expert workshop in order to discuss: 1) when and how application of structured benefit-risk assessment approaches and tools can contribute the greatest value to support regulatory decision-making, 2) key considerations for ensuring that benefit-risk assessment approaches and tools are fit-for-purpose in FDA’s drug regulatory context, and 3) strategies for incorporating patient input (derived through both qualitative and quantitative methods) into structured benefit-risk assessment.

Funding for this conference was made possible in part by a cooperative agreement with the FDA’s Center for Drug Evaluation and Research. The views expressed in written conference materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services nor does mention of trade names, commercial practices, or organizations imply endorsements by the U.S. Government.