DIA-MHRA Global Summit

SESSION OVERVIEW:Rare diseases and advanced therapies sit at the frontier of medical innovation, bringing together powerful science, urgent patient need and complex product development and unique regulatory approval challenges. This session will explore how the UK and international partners can support more effective pathways for rare disease and advanced therapy development, while keeping access,?affordability?and implementation in view. The discussion will bring together patient, clinical,?regulatory,?and global perspectives to consider what makes development?feasible, what makes access sustainable, and how health systems can respond when innovation moves faster than established pathways.

SESSION IMPACT:Bringing together diverse voices from industry and regulatory authorities, this session will?provide?a unique opportunity to explore the realities of developing and delivering innovative therapies. Attendees will leave with a deeper understanding of how to engage regulators effectively,?anticipate?challenges and shape robust long-term strategies that can translate scientific breakthroughs into transformative outcomes for patients.