ERDERA – the European Rare Diseases Research Alliance – is pleased to announce the upcoming 2nd International Conference on Clinical Research Networks (CRNs) for Rare Diseases, co-organised in collaboration with Rare Disease International and the International Rare Diseases Research Consortium (IRDiRC).

Details:

• Date: 9–10 December 2025
• Format: Hybrid – online and in-person participation available
• Registration: here.
• Venue: Marsilius College, Heidelberg, Germany
• Programme: see below.

 

Programme

Times are in CET.

 

Tuesday December 9th, 2025

 

12.00 – 1.00 p.m. –  Light lunch reception

1.00 – 2.00 p.m. – Plenary session: Opening Session – Setting the scene

• David Pearce, International Rare Diseases Research Consortium (IRDiRC) Professor Department Pediatrics, Sanford School of Medicine of the University of South Dakota (United States of America)
• Alexandra Heumber Perry, Chief Executive Officer, Rare Diseases International (France)
• Daria Julkowska, Scientific Coordinator of the European Rare Disease Research Alliance (ERDERA) and Assistant Director of the Themathic Institute of Genetics, Genomics & Bioinformatics (IT GGB) at INSERM (France)

2.00 – 3.30 p.m. –  Plenary session 1:  Global efforts in Real World Evidence (RWE) and data collection

Session Chair: Franz Schaefer, Hospital Franz Schaefer, MD, Professor of Pediatrics Head, Division of Pediatric Nephrology, Heidelberg University Coordinator, European Reference Network for Rare Kidney Disease (ERKNet) Co-Lead, Clinical Research Network of the European Rare Disease Research Alliance (ERDERA)

• Carla Jonker, Scientific Administrator, Real World Evidence workstream (RWE); Data Analytics and Methods Task Force (TDA); European Medicines Agency (EMA) (The Netherlands)
• Cécile Ollivier, Vice President Global Affairs, Critical Path Institute (The Netherlands)
• Prof. Thomas Klockgether, German Center for Neurodegenerative Diseases (DZNE) (Germany)
• Pat Furlong, President, Parent Project Muscular Dystrophy (United States of America)

3.30 – 4.00 p.m. – Coffee and networking break

4.00 – 6.00 p.m. – Plenary session 2: New approaches in diagnostics & clinical research

Session Chair: Yanis Mimouni (Associate Director, Regulatory Science, C-Path)

• Tudor Groza, Co-Lead AI and Data Science, Maternal and Child Health Research Institute, KK Women’s’ and Children’s Hospital Singapore, Principal Scientist, Bioinformatics Institute, A*STAR (Singapore)
• Sergi Aguiló Castillo, RadboudUMC (The Netherlands)
• Alexander Hoischen, Groningen Medical University (The Netherlands)
• Ramona Walls, Data Collaboration Center Director, C-Path (United States of America)
• Dustin O’Dell, Cofounder & CEO, SymetryML Inc and Decentra Health (United States of America)
• Chris Hendriksz, A Rare Cause (South Africa)

7.00 – 8.30 p.m.  – Networking reception

 

Wednesday December 10th, 2025 

 

9.00 – 11.00 a.m. – Parallel workshops

PARALLEL WORKSHOP 1:  Learnings from Low and Middle-Income Countries: Ensuring Representation in Clinical Research Data and Registries 

Session Chairs: Daria Julkowska, Scientific coordinator of the European Rare Diseases Research Alliance (ERDERA) and Assistant Director of the Thematic Institute for Genetics, Genomics & Bioinformatics (IT GGB) at INSERM, Monica Drum, Senior Global Programmes Manager, Rare Diseases International (RDI)

• Chris Hendriksz, A Rare Cause (United Kingdom / South Africa)
• Roberto Giugliani, Professor of Genetics at the Federal University of Rio Grande do Sul (UFRGS), Chief of the Clinical Research Group in Clinical Genetics at Hospital de Clinicas de Porto Alegre (HCPA), and Executive Director of Casa dos Raros, in Porto Alegre (Brazil)
• Robin Sarfati, CTO at Tekkare (France)
• Prof. Mohamed Hassany, Assistant Minister of Health for Projects and Public Health Initiatives Professional Affiliation: Ministry of Health and Population (Egypt)

PARALLEL WORKSHOP 2: Rules of engagement in multi-stakeholder pre-competitive environments

Session Chairs: Samantha Parker Patient Engagement Lead Rare Diseases at Italfarmaco, Vice-Chair IRDiRC; Heidrun Hildebrand, Alliance Manager Pediatric Development; Bayer Pharmaceuticals

• Ricardo Fernandes, Chief Medical Officer at conect4children-stichting
• Cécile Ollivier, Vice President Global Affairs, Critical Path Institute (The Netherlands)
• Begoña Nafria Escalera, Head of Patient Engagement in Research Area, Paediatric Cancer Center Barcelona – PCCB, Institut de Recerca Sant Joan de Déu (Spain)
• Rebecca Leary, Senior Project Manager, Newcastle University, The John Walton Muscular Dystrophy Research Centre (United Kingdom)
• Kristina An Haack, MD, Senior Project Head Rare Development, Inherited NeuroMetabolic Diseases, Pediatric Network Lead (France)
• Victoria Hedley, Together4Rare (United Kingdom)

11.00 – 11.30 a.m. – Coffee and networking break

11.30 a.m. – 12.30 p.m. – Plenary session 3:  Reporting back from parallel workshops & discussion

• Monica Drum, Senior Global Programmes Manager, Rare Diseases International (France)
• Daria Julkowska, Scientific coordinator of the European Rare Diseases Research Alliance (ERDERA) and Assistant Director of the Thematic Institute for Genetics, Genomics & Bioinformatics (IT GGB) at INSERM (France)
• Heidrun Hildebrand, Alliance Manager Pediatric Development; Bayer Pharmaceuticals (Germany)

12.30 – 13.30 p.m. – Lunch break

13.30 – 3.00 p.m. – Plenary session 4:  Global trials, local impact: Empowering access and engagement in international clinical research, role of patients and broad partnerships

Session Chair: Alexandra Heumber Perry, Chief Executive Officer, Rare Diseases International (France)

• Dr. Sudheendra Rao, N R, Scientific Advisor, Organization for Rare Diseases India
• Kelly Du Plessis, Rare Diseases South Africa
• Begoña Nafria Escalera, Head – Patient Engagement in Research Area, Pediatric Cancer Center Barcelona, Sant Joan de Deu SJD, Spain; Conect4Children Stichting
• Dr Roberto Giugliani, Professor of Genetics at the Federal University of Rio Grande do Sul (UFRGS), Chief of the Clinical Research Group in Clinical Genetics at Hospital de Clinicas de Porto Alegre (HCPA), and Executive Director of Casa dos Raros, in Porto Alegre (Brazil)

3.00 – 3.30 p.m. – Coffee and networking break

3.30 – 5.00 p.m. – Plenary session 5: Global networks and models of care in high income and LMIC – The Duchenne experience

Session Chair: David Pearce, International Rare Diseases Research Consortium (IRDiRC) Professor Department Pediatrics, Sanford School of Medicine of the University of South Dakota (United States of America)

• Anna Thetford, Registered Nurse; Program Director (Clinical), Rare Care Centre, Perth, Western (Australia)
• Karolína Podolská, MD, Internal physician and coordinator of Center for adults with muscular dystrophy, General University Hospital in Prague, Czech Republic, Accredited Duchenne Centers Program Manager

5.00 – 5.30 p.m.  – Closing of the conference