Together For Rare Diseases and ERICA hosted a webinar on European Reference Networks (ERNs), detailing their core missions, expertise, diversity and benefits of collaboration with the private sector.
This webinar aimed to bridge the knowledge gaps between ERNs and the pharmaceutical and biotech industries, fostering collaborations that can accelerate research and development in rare diseases, ultimately benefiting patients and advancing medical science.
Bringing together 50+ people from the private sector as well as from the European research community and patients/their representatives and policymakers, the webinar showcased how ERNs can work with pharmaceutical and biotech companies to leverage the strengths of both sectors: the public health focus and patient access of ERNs, combined with the research capabilities and resources of industry. These public-private collaborations accelerate research and development in rare diseases, ultimately benefiting patients and advancing medical science.
Together For Rare and ERICA’s next joint webinar will be on 16 January 2025, articulating the contribution of the private sector in public-private partnerships in rare disease research.
You can also watch the webinar recording here.
Highlights
Alongside our Chair, Sheela Upadhyaya, our panelists outlined how and where ERNs have emerged as invaluable research partners. With over 1,600 specialised healthcare units from 382 hospitals across 27 EU Member States and Norway, ERNs provide access to a vast network of clinical experts and patient data. This concentration of expertise allows researchers to tap into a diverse pool of knowledge and experience, which is particularly crucial when studying rare diseases that may have limited cases in any single country.
Understanding ERNs
Alberto Pereira (Professor of Medicine at UMC Amsterdam and coordinator of Endo-ERN (the ERN for Rare Endocrine Conditions)) and Franz Schaefer (Professor of Paediatrics at Heidelberg University Hospital and coordinator of ERKNet ( the European Rare Kidney Disease Reference Network)) outlined how and where ERNs can serve as research partners, presenting the resources, data repositories and clinical expertise available within ERNs.
They offered specific examples of how ERNs have developed clinical data repositories, trial networks, biobanks and tissue samples, registries, standardised protocols and guidelines, telemedicine platforms, training and education resources, and data sharing initiatives that make ERNs invaluable for research. Clinical data repositories, such as ERN-EYE’s comprehensive database for over 900 rare eye conditions, enable large-scale studies by consolidating anonymized patient data across borders. Biobanks, like those in ERN EURO-NMD, house crucial biological samples for genetic and molecular research, facilitating insights into disease mechanisms and therapeutic development. ERNs also streamline clinical trial networks, exemplified by ERN PaedCan’s pan-European framework for swift patient recruitment in pediatric cancer studies. Standardized protocols and guidelines enhance data consistency, ensuring the reliability of multicentre studies. Patient registries, like ERN-RND’s, track longitudinal data on disease progression and treatment outcomes, aiding biomarker discovery and long-term analysis. Telemedicine platforms expand research inclusivity, enabling remote data collection and patient monitoring across diverse regions. ERNs further foster innovation through training and education resources, such as ERN RITA’s webinars and e-learning modules, which equip researchers with cutting-edge methodologies. Lastly, their commitment to data-sharing initiatives breaks silos, promoting collaboration and accelerating advancements in rare disease understanding and care. Together, these resources transform the research landscape, making ERNs critical drivers of progress in tackling rare diseases.
The RHINE Project
Katarzyna Mosiewicz (Medical Affairs, Novo Nordisk) and Prof. Schaefer presented insights into the RHINE Project, a public-private partnership in rare renal disease research. The project serves as an excellent case study of how public-private partnerships in rare disease research can develop, the capacity, skills and structures required, as well as challenges encountered in the project’s development.