A global community of clinicians, patient leaders, regulators and researchers met in Heidelberg and online for the second International Conference on Clinical Research Networks (CRNs), organised by the European Rare Diseases Research Alliance (ERDERA), Rare Diseases International (RDI) and the International Rare Diseases Research Consortium (IRDiRC).
CRNs are structured collaborations of expert sites, laboratories, patient organisations and other stakeholders that accelerate prevention, diagnosis, observational studies and clinical trials for rare and groups of rare diseases.
The conference was held on 9-10 December and moved from evidence to action: regulatory grade real world evidence (RWE) and data collection; new diagnostics and clinical research methods; medical devices in trials; lessons from low and middle income countries (LMICs); and models of care within global networks.
“Interoperability — common standards, shared infrastructure and clear roles — can help data, diagnostics and trials move across borders and translate into earlier diagnoses and better care,” said Alexandra Heumber Perry, Chief Executive of RDI. IRDiRC Chair David Pearce added: “Connected centres of expertise turn research into better care. Our task is to make those connections routine.”
“We are not on a blank page, not starting from zero”, said ERDERA Scientific Coordinator Daria Julkowska while discussing the value of an existing collaborative ecosystem. She called for practical alignment for the future: “We are here to connect, to act, and to create the impact that none of us could achieve alone”.
Advancing evidence, diagnostics and clinical research
Plenary discussions clarified what regulators need from RWE and highlighted multinational efforts contributing to it — from ERKReg and CompCure to the Ataxia Global Initiative and Critical Path (C-Path) Institute’s RDCA‑DAP platform. “Real‑world evidence is powerful only when collected according to common standards and trusted by regulators,” said Franz Schaefer of Universitätsklinikum Heidelberg, a co lead under ERDERA’s Clinical Research Network work stream. “For families, it means everyday experiences inform guidelines and approvals. Our job is to link centres between countries, so data are comparable, high quality and usable.”
A second plenary outlined a practical pipeline: securely drawing information from electronic health records, turning symptoms into computable descriptions, using next generation diagnostics, and applying model informed methods and looking at privacy preserving, decentralised AI. “We are closing the loop between data capture, diagnosis and trial design — with approaches that also work in resource limited settings,” said Yanis Mimouni, Associate Director for Regulatory Science of Critical Path Institute (C-PATH). “In short, safeguarded clinical data can guide smarter studies and faster access to promising therapies”.
Workshops to boost collaboration and inclusion
One workshop, led by Daria Julkowska and RDI’s Senior Global Programmes Manager Monica Drum, underlined innovative solutions implemented in LMICs and set out further recommendations to strengthen LMICs capacities in new born screening, diagnosis, registries and trials — from enhancing of local knowledge and collaboration, through context adapted registries and ethical cross border data sharing to building sustainable, locally owned infrastructure and leveraging regional cooperation.
In parallel, Heidrun Hildebrand of the European Federation of Pharmaceutical Industries and Associations and IRDiRC Vice‑Chair Samantha Parker convened partners to discuss rules of engagement for multistakeholder CRNs — networks that bring together patients, clinicians, laboratories, regulators, funders and industry to run studies and optimise data use for all purposes across countries and stakeholders. “Clear rules and shared incentives build trust — and trust accelerates trials,” said Samantha Parker. “Transparent frameworks make collaboration predictable: who leads what, how decisions are taken, how data are governed, and how benefits are shared.”
Both workshops fed into a session on global trials with local impact, examining enabling policy frameworks and patient led models for access. Speakers underlined that patients must be a starting point of all processes and showed how national rules and global guidance can make international studies easier to run while protecting fairness in recruitment and generating results regulators can use.
The conference closed with the Duchenne muscular dystrophy experience, showing how connected networks of centres of expertise support day to day care and open routes into research. “We must match diagnostic gains with treatment access and coordinated, holistic care,” said David Pearce. “When networks are fragmented, families face long waits, duplicated tests and missed chances to join studies; when they are connected, pathways are clearer and outcomes improve.”
Next steps already defined
The three organisers will publish a concise conference summary and a fuller report and a publication.
Each session produced a list of actions — from data standards work to practical steps that make trials more inclusive — which together will form a shared roadmap. ERDERA, RDI and IRDiRC will use this to track progress and will revisit the commitments at the next CRN Conference planned for 2027.
