2nd Clinical Research Network Conference · Media Kit
The 2nd International Conference on Clinical Research Networks (CRNs) for Rare Diseases brings together global experts to strengthen coordinated research, improve diagnostics and trials, and advance equitable access for people living with rare conditions.
This media kit provides key messages and background on the role of CRNs in overcoming fragmentation in rare disease research, alongside the full event programme and practical details. It hopes to facilitate the work of media professionals and communications experts from the rare disease community.
For media enquiries, interview requests, additional context, or case studies, please contact the ERDERA Communications office.
2nd CRN Conference: Why It Matters
- Clinical Research Networks (CRNs) are structured collaborative grids of expert clinical sites, laboratories, patient organisations and other stakeholders that join forces to accelerate prevention, diagnosis, epidemiological studies, natural‑history studies and clinical trials for rare and groups of rare diseases.
- By pooling scarce patient populations, harmonising protocols and sharing high‑quality data, CRNs overcome the fragmentation that has long slowed progress in rare disease research.
- Through a dedicated service, ERDERA supports a pan-European Clinical Research Network to streamline and accelerate rare disease research across borders.
- The 2nd International Conference on Clinical Research Networks (CRNs) for Rare Diseases is designed to strengthen the global ecosystem that underpins clinical research in rare diseases, by bringing together researchers, clinicians, patient advocates, industry, policymakers and funders.
- The conference will take place 9–10 December 2025, in a hybrid format (Heidelberg, Germany, and online), with participants from across the globe. More on registration here.
- The event is organised by the European Rare Diseases Research Alliance (ERDERA), in collaboration with Rare Disease International (RDI) and the International Rare Diseases Research Consortium (IRDiRC), building on the success of the inaugural conference held in December 2022 in Paris.
- Its general objective is to gather experts from different continents to increase mutual knowledge on CRN structures and activities, and to identify concrete pathways to stimulate collaboration and interoperability of these networks worldwide.
- Core themes include global efforts to advance Real World Evidence (RWE) and data collection, new approaches in diagnostics and clinical research, medical devices in trials (e.g. for quality of life and movement evaluation), lessons learned from low‑ and middle‑income countries (LMICs), and models of care and global research networks across income settings. Find the programme below.
The Full 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)
- Baptiste Eluard, Senior Scientific Project Manager at the European Rare Disease Research Alliance (ERDERA)
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)
- Ramona Walls, Data Collaboration Center Director, C-Path (United States of America)
- 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, Radboudumc Nijmegen (The Netherlands)
- Cécile Ollivier, Vice President Global Affairs, Critical Path Institute (The Netherlands)
- Dustin O’Dell, Cofounder & CEO, SymetryML Inc and Decentra Health (United States of America)
- Chris Hendriksz, A Rare Cause (South Africa)
- Yong Chen, Ph.D., FASA, FACMI, Professor of Biostatistics, Director of the Penn Computing, Inference and Learning lab, Founding Director of the Center for Health AI and Synthesis of Evidence (CHASE), Perelman School of Medicine at the University of Pennsylvania (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, 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)
- Volker Straub, Harold Macmillan Professor of Medicine; Director, The John Walton Muscular Dystrophy Research Centre; Deputy Dean, Translational and Clinical Research Institute, Faculty of Medical Sciences
Newcastle University and Newcastle Hospitals NHS Foundation Trust (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
- Christine Mutena, Co-Founder at Rare Disorders Kenya
- 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
- Ricardo Fernandes,Chief Medical Officer at conect4children-stichting
- Karolina Śledzińska,MD, PhD, Pediatrician, Clinical Geneticist, Department of Pediatrics, Hematology and Oncology, Medical University of Gdansk, Poland – ONLINE
5.00 – 5.30 p.m. – Closing of the conference
Useful Materials
Visual Identity and Logos
Event Details and Registration
The 2nd International Conference on Clinical Research Networks (CRNs) for Rare Diseases, is co-organised by ERDERA – the European Rare Diseases Research Alliance, Rare Disease International and the International Rare Diseases Research Consortium (IRDiRC).
Event Details:
- Date: 9–10 December 2025
- Format: Hybrid – online and in-person participation available
- Registration: here.
- Venue: Marsilius College, Heidelberg, Germany
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