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
Building an Ecosystem for Rare Disease Trial Readiness
09:00- 09:15
The European Platform on Rare Disease Registration: Connecting Data, Accelerating Care Pathways and Enabling Research
09:15 – 09:30
Amplifying Patient and Public Involvement in Small Rare Diseases Ecosystems
09:30- 09:45
National Biobanks for Population and Rare Disease Research
09:45 – 10:00
Considerations in the Development of Sensitive Outcome Measures Needed to Track the Natural History of Rare Diseases (Virtually)
10:00- 10:15
Clinical Trials From the Patients’ Perspective
10:15- 11:00
Panel Discussion: Stakeholders Working Together for Clinical Trial Preparation
11:00-11:30
Coffee Break
Session 5: Rollout of Efficacious Therapies: Access and Infrastructure Considerations
11:30- 11:45
Infrastructure Considerations for Delivering Therapies at Scale: France’s Example
11:45 – 12:00
Capacity Building and Interdisciplinary Training for Delivering Novel Therapies in Secondary Care Comprehensive Healthcare Facilities
12:00- 12:15
Health Technology Assessment and Innovative Payment Models for Therapies for Rare Diseases (Virtually)
12:15- 12:30
Competitive (mis)pricing in Small Markets: An Example of DMD Therapies
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 – 17:00
Networking Cocktail & Farewell at The CING Premises