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 (United States of America)
• Alexandra Heumber Perry, Rare Diseases International (France)

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

Session Chair: Franz Schaefer, Heidelberg University Hospital

• European Medicines Agency (The Netherlands) (TBC)
• Cécile Ollivier, Critical Path Institute (The Netherlands)
• Prof. Thomas Klockgether, University of Bonn (Germany)
• Pat Furlong, Parent Project Muscular Dystrophy

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

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

Session Chair: Yanis Mimouni (Associacte Directo, Regulatory Science, C-Path)

• Tudor Groza, Agency for Science, Technology and Research A*STAR, (Singapore)
• Yong Chen, Lancet Commission on Rare Diseases, (United States of America) TBC
• TBC, Leiden Medical University (The Netherlands)
• Alexander Hoischen, Groningen Medical University (The Netherlands)
• Dustin O’Dell, SymetryML (USA)

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, INSERM, Monica Drum, Rare Diseases International (RDI)

• Chris Hendriksz, A Rare Cause (South Africa)
• Roberto Giugliani, Federal University of Rio Grande do Sul (Brazil)
• Robin Sarfati, Tekkare (France)
• Prof. Mohamed Hassany, Assistant Minister of Health for Projects and Public Health Initiatives, Ministry of Health and Population (Egypt)

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

Session Chairs: Samantha Parker IRDiRC vice chair; Heidrun Hildebrand, Pediatric Development Alliance Manager

• Mark Turner, CEO conect4children Stichting
• Cécile Ollivier, Managing Director, cPATH Europe
• Begoña Nafria Escalera, Head of Patient Engagement in Research Area, Paediatric Cancer Center Barcelona – PCCB, Institut de Recerca Sant Joan de Déu
• Rebecca Leary, Senior Project Manager, Newcastle University, The John Walton Muscular Dystrophy Research Centre
• Together4Rare (TBC)

10.00 – 11.30 a.m. – Coffee and networking break

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

• Alexandra Heumber Perry, Rare Diseases International (France)
• Daria Julkowska, European Rare Diseases Research Alliance (France)
• Heidrun Hildebrand, European Federation of Pharmaceutical industries and associations (Germany)

12.30 a.m. – 13.30 p.m. – Lunch break

13.30 a.m. – 3.00 p.m. – Plenery 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, Rare Diseases International

• Dr. Sudheendra Rao, Organisation 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 Guigliani, Federal University of Rio Grande do Sul (Brazil)

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

3.30 – 5.00 p.m. – Plenery session 5: Global Networks and Models of Care in high income and LMIC – The Duchenne experience

Session Chair: Gareth Baynam, Rare Care Center

• Mark Turner, Conect4Children Stichting (United Kingdom)
• Anna Thetford, Rare Care Center, (Australia)
• Karolína Podolská, Program manager of Accredited Duchenne Centers Program

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