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 (online)
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
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
13:15-13:30
Closing Lecture
The role of The Cyprus Institute of Neurology & Genetics in caring for patients with rare diseases: clinical excellence and research innovation
13:30 – 14:30
Lunch Break
14:30–15:30
Tour at the premises of The Cyprus Institute of Neurology & Genetics
15:30–17:00
Farewell networking party, The Cyprus Institute of Neurology & Genetics