Following the 11 December 2025 political agreement, EU institutions are moving towards formal adoption of a new Directive and Regulation that will reshape how medicines—including orphan and paediatric medicines—are developed, authorised and supplied across the European Union.
The two-day event will focus on translating research and policy initiatives into tangible benefits for Rare Disease patients through better prevention, diagnosis, and treatment.
With over 300 million people affected by rare diseases, timely access to effective therapies is critical. Despite strong alignment in regulatory expectations, patients in many regions face years of delays highlighting the need for improved international coordination.
Bringing together global experts, healthcare professionals, patient advocates, and policymakers to advance knowledge and strengthen international collaboration in the field of rare diseases.
