Benefit-Risk Assessment from Inception to Maturation: Aligning Regulatory and Industry Goals

The culmination of drug and device development entails health authority assessment of a treatment’s benefit-risk balance. Sponsors’ application are increasingly expected to defend whether a treatment’s benefits outweigh its risks, accounting for not just clinical data but the nature of the medical condition, alternative treatments and risk mitigation plans. The growing body of regulatory and industry structured benefit-risk frameworks provide some guidance for this process, but sponsors are struggling with questions on whether and how to prespecify a benefit-risk approach, the balance between qualitative and quantitative approaches for the assessment, whether and how to incorporate patient perspectives, and how to concisely communicate a benefit-risk argument. This session will review principles and examples from health authority submissions, describing methods that have been successful and the processes and practical concerns sponsors need to address to conduct them.