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

Development of value adding drugs demands a clearly positive Benefit-risk balance during development, approval and life cycle of a medicinal product. Various regulatory relevant documents such as clinical trial applications and Development Safety Update Reports produced by sponsors during the development phase require an explicit statement on benefit-risk. This session will address the challenges for companies to map out and continuously update and benchmark the expected key benefits and risks, whilst maintaining consistency in the relevant documents provided to the regulatory authorities. It will also take into account available regulator's perspectives on how dialogue on the expected benefit-risk balance could be formalized already during development, e.g., during Scientific Advice/End of Phase 2 meetings, and what models or frameworks could be acceptable in this context. Patient centric drug development demands the elicitation of the patient´s perspective at an early stage, preferably prior to finalizing the Phase 3 design, and concrete examples for potential approaches will be provided.