Orphan Drugs & Rare Diseases Global Congress Europe 2025
19 March 2025 – 20 March 2025
Copthorne Tara Hotel London Kensington, London, United Kingdom
The 23rd Orphan Drugs & Rare Diseases Global Congress 2025 Europe is a two-day congress organised by Facilitate Live at the Copthorne Tara Hotel Kensington in London. It covers drug development, treatment availability and access for rare diseases, with plenary sessions, keynotes, sponsored spotlights and panel discussions on policy, research and development, patient engagement and market access. It is co-located with the 8th Expanded Access Programmes World Congress 2025 Europe, with a shared exhibit and networking area.
32 sessions · 34 speakers
Organiser (1)
Sponsors (4)
WEP CLINICAL · Bionical Emas · OPEN Health · Pulse Infoframe
Speakers (34)
- Stefano Benvenuti Head of Public Affairs and Market Access, fondazione telethon
- Alexander Natz EUCOPE
- Prof James Levine President, Fondation Ipsen
- Daniel O’Connor Director, Regulatory Policy and Early Access, ABPI
- Francis Pang SVP Global Market Access and Geographic Expansion, Orchard Therapeutics
- Stacey Minton SVP, Head of Corporate Affairs, Kyowa Kirin International
- Amanda Bok Chief Partnership Officer, The Synergist
- Carole Scrafton Director & Co-Founder, Flutters and Strutters
- Cpt. Luke Rosen Founder, Rescue7: Firefighters for Patients
- Dr Alberto Malerba Lecturer in Gene therapy, Gene Medicine Laboratory for Rare Diseases, Royal Holloway, University of London
- Dr Lucy McKay Chief Executive Officer, Medics4RareDiseases
- Dr Peter Davis Chair, British Paediatric Surveillance Unit Scientific Committee
- Dr Usman Khan Chair, Motor Neurone Disease (MND) Association
- Dr. Femida Gwadry-Sridhar Founder and CEO, Pulse infoframe Inc.
- Dr. Rüdiger Schulze Vice President and General Manager, DACH, Ultragenyx
- Jennifer Schranz Senior Vice President and Global Head of Rare Diseases, R&D, Ipsen
- Lindsay Randall Mother, Founder, CEO, Arthur’s Quest
- Manel Cascalló General Director EU Subsidiary & Founder, Theriva Biologics
- Nick Sireau CEO and Chair of Trustees, AKU Society
- Prof Guillaume Canaud Translational Medicine & Targeted Therapies Unit, Necker Enfants Malades
- Surabhi Verma Chief Medical Officer, Leadiant Biosciences
- Dante Di Iulio Public Affairs Lead, Chiesi GRD
- Dr Pooja Takhar Joint Chief Executive, Tuberous Sclerosis Association
- Alicia Granados Global Head Scientific Advocacy & Insights, Rare Diseases, Sanofi
- Anna Kole Global Patient Engagement Lead - MG, UCB
- Dan Lewi Head of Business Development and Patient Advocacy; Co-founder, CATS Foundation, Pulse infoframe Inc.
- Dr Chiuhui Mary Wang Global Programme Director, Rare Diseases International
- Dr Kirsten Johnson Chair of the Council, Rare Diseases International
- Enrico Piccinini Head of Europe & International, Rare Diseases, Chiesi Group
- Flaminia Macchia Head, Global Rare Disease Policy, AstraZeneca
- Josie Godfrey Co-Founder and CEO, Realise Advocacy
- Lindsay Birrell Co-CEO, Realise Advocacy
- Robert Mitchell-Thain CEO, PBC Foundation
- Sophie H. Turner Initiative Coordinator, Empowered By Us
Agenda
- Chairpersons Welcome and Opening Remarks 19 March 2025 · Auditorium 1
- Progressing Rare disease diagnosis, care and treatment to improve health outcomes for patients with rare diseases 19 March 2025 · Auditorium 1
- Update on England's Rare Diseases Action Plan 19 March 2025 · Auditorium 1
- Reflections on the EU’s future rare disease policy environment 19 March 2025 · Auditorium 1
- The DACMAR Advantage: This approach helps biotech companies position themselves for potential acquisition or public offering, with a focus on creating sustainable impact and preparing for long-term success 19 March 2025 · Auditorium 1
- Commercialisation of Orphan Drugs: Top issues facing the community today 19 March 2025 · Auditorium 1
- Navigating the complexity of Rare Diseases Clinical Trials 19 March 2025 · Auditorium 1
- Answering the million dollar question: Who really owns the data? 19 March 2025 · Auditorium 1
- Early access to Orphan Drugs in Europe 19 March 2025 · Auditorium 1
- Preclinical development of gene therapy products for muscular dystrophies. 19 March 2025 · Auditorium 1
- VCN-01 oncolytic adenovirus development in pediatric retinoblastoma 19 March 2025 · Auditorium 1
- Scientific journey from coal to diamond: myths and misperceptions in rare disease innovation 19 March 2025 · Auditorium 1
- Mesenchymal stem cells as a potential treatment for osteogenesis imperfecta 19 March 2025 · Auditorium 1
- Drug repositioning in overgrowth syndrome and vascular malformations 19 March 2025 · Auditorium 1
- Promises and Pitfalls of using Real World Data and Evidence (RWD/E) in Orphan Drug Development. 19 March 2025 · Auditorium 1
- Rare Disease Research: Its Importance for Orphan-Drug Development Success 19 March 2025 · Auditorium 1
- Chairperson's Closing Remarks 19 March 2025 · Auditorium 1
- Chairpersons Welcome and Opening Remarks 20 March 2025 · Auditorium 1
- Rare Diseases and Orphan Drugs: The importance of Patient engagement strategies to overcome challenges in Orphan Drug Development 20 March 2025 · Auditorium 1
- Reframing the patient in an age of precision medicine 20 March 2025 · Auditorium 1
- Defining rare in the era of personalised medicine 20 March 2025 · Auditorium 1
- Rare diseases – not just the “what” but the “who” 20 March 2025 · Auditorium 1
- Challenges and opportunities for mRNA therapy for rare genetic diseases 20 March 2025 · Auditorium 1
- Critical role of patient organisations in the development of new therapies 20 March 2025 · Auditorium 1
- How to engage every community and empower patients to participate in clinical research. 20 March 2025 · Auditorium 1
- The WHO resolution on social participation in health and well-being and what its implementation could mean for patients, society and health systems 20 March 2025 · Auditorium 1
- Global Rare disease groups collaborate for a parent led observational study 20 March 2025 · Auditorium 1
- Importance of Patient Advocacy for Clinical Research 20 March 2025 · Auditorium 1
- Awareness, Accessibility and Access: the reality of treating rare disease 20 March 2025 · Auditorium 1
- Optimizing pricing and access to rare disease drugs 20 March 2025 · Auditorium 1
- Global collaborations and innovations shaping the future of Rare disease treatment 20 March 2025 · Auditorium 1
- Session Chair's Closing Remarks 20 March 2025 · Auditorium 1