Day 2

08:00- 08:30
Registration and coffee

Session 4: Becoming trial-ready for rare diseases therapies

08:30- 08:45
Topic: Introducing initiatives to bring more clinical trials to Europe

Helene Le Borgne, Policy Officer, DG Research and Innovation

08:45- 09:05
Topic: Building an ecosystem for rare disease trial readiness

Maurizio Scarpa, Coordinator of MetabERN

09:05- 09:20
Topic: The European Platform on rare disease registration: Connecting data, accelerating care pathways and enabling research

Andri Papadopoulou, Scientific Officer, Joint Research Centre

09:20- 09:40
Topic: National Biobanks for Population and Rare Disease Research

Giedrė Kvedaravičienė, Director of the Lithuanian Population and Rare Diseases Biobank

09:40- 10:00
Topic: Clinical trials from the patients’ perspective

Allan Fred Byamukama

10:00- 10:30
Panel discussion: Stakeholders working together for clinical trial preparation

Moderator: Prof. Kleopas Kleopa

10:30- 11:00
Coffee Break

Session 5: Rollout of efficacious therapies: access and infrastructure considerations

11:00- 11:20
TBC
11:20- 11:40
Topic: Health economics of expensive therapies: considerations from the State

Neophytos Charalambides, Minister of Health, Republic of Cyprus

11:40- 12:00
Topic: Infrastructure considerations for delivering therapies at scale

Anne‑Sophie Lapointe, French representative in ERN Board of Member States

12:00- 12:20
Topic: Health Technology Assessment and Innovative Payment Models for therapies for rare diseases

Christine Leopold, Utrecht Center for Pharmaceutical Policy and Regulation / WHO

12:20- 12:40
Topic: Competitive (mis)pricing in small markets: an example of DMD therapies

Panikos Voskos, President MDA Cyprus

12:40- 13:10
Panel discussion: Rare disease therapy delivery in the healthcare system: paths to sustainability

Speakers:

· Neophytos Charalambides, Minister of Health, Republic of Cyprus

· Anne-Sophie Lapointe, French representative in ERN Board of Member States

· Christine Leopold, Pharmaceutical system and policy researcher, Utrecht Center for Pharmaceutical Policy and Regulation / WHO Collaborating Center

· Panikos Voskos, President MDA Cyprus

· Stella Kyriakides, former EC Health Commissioner

Moderator:

Dr Menelaos Pipis, Senior Neurologist, Head of the Neuropathology Department, The Cyprus Institute of Neurology & Genetics

 

13:10- 13:30
Closing Lecture

Leonidas Phylactou, CEO & Medical Director, CING