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- 09:00
Topic: Building an ecosystem for rare disease trial readiness

Maurizio Scarpa, Coordinator of MetabERN

09:00- 09:15
Topic: The European Platform on rare disease registration: Connecting data, accelerating care pathways and enabling research

Dr. Andri Papadopoulou, Scientific Officer, Joint Research Centre, European Commission

09:15 – 09:30
Amplifying patient and public involvement in small rare diseases ecosystems

Mr. Spyros Polyviou, Director of the Cyprus Alliance for Rare Disorders

09:30- 09:45
Topic: National Biobanks for Population and Rare Disease Research

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

09:45 – 10:00
Considerations in the development of sensitive outcome measures needed to track the natural history of rare diseases

Dr. Luca Sangiorgi Director Rare Bone Disorders Dept. and Coordinator Rare Bone Diseases Centre Istituto Ortopedico Rizzoli, Coordinator of ERN-BOND

10:00- 10:15
Topic: Clinical trials from the patients’ perspective

Allan Fred Byamukama

10:15- 11:00
Panel discussion: Stakeholders working together for clinical trial preparation

Speakers:

  • Dr. Helene Le Borgne, Policy Officer, DG Research and Innovation
  • Dr. Maurizio Scarpa, Coordinator of MetabERN
  • Dr. Andri Papadopoulou, Scientific Officer, Joint Research Centre
  • Charalambos Papadopoulos, President of the Cyprus Alliance for Rare Disorders
  • Dr. Giedrė Kvedaravičienė, Director of the Lithuanian Population and Rare Diseases Biobank
  • Dr. Luca Sangiorgi Director Rare Bone Disorders Dept. and Coordinator Rare Bone Diseases Centre Istituto Ortopedico Rizzoli, Coordinator of ERN-BOND
  • Allan Fred Byamukama
  • Prof. Alexis Arzimanoglou, Coordinator of ERN EpiCARE

 

Moderator: Prof. Kleopas Kleopa, Senior Neurologist, Head of the Neuroscience Department, The Cyprus Institute of Neurology & Genetics

11:00-11:30
Coffee Break

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

11:30- 11:45
Topic: Infrastructure considerations for delivering therapies at scale: France’s example

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

11:45 – 12:00
Topic: Capacity building and interdisciplinary training for delivering novel therapies in secondary care comprehensive healthcare facilities

Prof. Nikolaos Zamboglou, German Medical Institute, Limassol

12:00- 12:15
Topic: Health Technology Assessment and Innovative Payment Models for therapies for rare diseases

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

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

Panikos Voskos, President of Muscular Dystrophy Association, Cyprus

12:30- 13:15
Panel discussion: Rare disease therapy delivery in the healthcare system: paths to sustainability

Speakers:

  • Dr. Anne-Sophie Lapointe, French representative in ERN Board of Member States
  • Prof. Nikolaos Zamboglou, German Medical Institute, Limassol
  • Dr. Christine Leopold, Pharmaceutical system and policy researcher, Utrecht Center for Pharmaceutical Policy and Regulation / WHO Collaborating Center
  • Panikos Voskos, President of Muscular Dystrophy Association, Cyprus
  • Marina Vasiliou, President and Managing Director, Biogen

 

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

 

13:15-13:30
Closing Lecture

The role of CING in caring for patients with rare diseases: clinical excellence and research innovation

Prof. Leonidas A. Phylactou, CEO & Medical Director of The Cyprus Institute of Neurology & Genetics

13:30 – 14:30
Lunch Break
14:30–17:15
Tour at the premises of The Cyprus Institute of Neurology & Genetics
17:15–20:00
Farewell networking party

The Cyprus Institute of Neurology & Genetics