Day 2 – 17/06/2026
08:00- 08:30
Registration and Coffee
Session 4: Becoming Trial-Ready for Rare Diseases Therapies
08:30- 08:45
Introducing Initiatives to Bring more Clinical Trials to Europe
08:45- 09:00
The European Platform on Rare Disease Registration: Connecting Data, Accelerating Care Pathways and Enabling Research
09:00 – 09:15
Amplifying Patient and Public Involvement in Small Rare Diseases Ecosystems
09:15- 09:30
National Biobanks for Population and Rare Disease Research
09:30 – 09:45
Considerations in the Development of Sensitive Outcome Measures Needed to Track the Natural History of Rare Diseases (Virtually)
09:45- 10:00
Clinical Trials From the Patients’ Perspective
10:00- 10:45
Panel Discussion: Stakeholders Working Together for Clinical Trial Preparation
10:45-11:15
Coffee Break
Session 5: Rollout of Efficacious Therapies: Access and Infrastructure Considerations
11:15- 11:30
Infrastructure Considerations for Delivering Therapies at Scale: France’s Example
11:30- 11:45
Mind the Access Gap: Cost-Effectiveness Aspirations Against Scientific Realities
11:45-12:00
Health Technology Assessment and Innovative Payment Models for Therapies for Rare Diseases (Virtually)
12:00-12:15
Capacity Building and Interdisciplinary Training for Delivering Novel Therapies in Secondary Care Comprehensive Healthcare Facilities
12:15- 12:30
Rare Diseases, Research, Therapies and Social Impact
12:30- 13:15
Panel Discussion: Rare Disease Therapy Delivery in the Healthcare System: Paths to Sustainability
13:15-13:25
Closing Remarks
13:30 – 14:30
Networking Cocktail & Farewell at The CING Premises