Next steps for addressing unmet medical need: Leveraging policy to boost ERN-industry collaboration
The multi-stakeholder group Together for Rare Diseases (Together4RD) has published a position statement calling for more effective collaboration between the European Reference Networks (ERNs) and industry. In this paper, Together4RD is advocating for an approach which would allow Europe to maintain its status as a pioneer in research and innovation, and most importantly, address the high level of unmet medical need in the rare disease community.
When the ERN system was introduced in 2017, it was with the intention of addressing the various, multi-faceted challenges faced by people living with rare diseases. However, the full realisation of this potential requires collaboration with a range of stakeholders, including industry, to meaningfully advance RD treatment and care. To date, this collaboration has been hampered by a lack of clarity on what level of industry involvement is permissible under the ERN Board of Member States’ policies.
Using evidence from case studies, stakeholder meetings, and steering group meetings, Together4RD presents a set of policy asks such as promoting transparent governance structures and creating a European Action Plan for Rare Diseases, all of which would help foster ERN-industry collaboration.
Fulfilling these asks would also move the rare disease community closer to fulfilling Recommendation #6 from the Rare 2030 Foresight Study on innovative and needs-led research and development, which calls in particular for clear rules to enable ERN-industry collaboration across a range of activities.
Together4RD is a multi-stakeholder initiative aimed at supporting ERNs to collaborate with stakeholders to pursue opportunities that will address unmet medical needs of people living with rare diseases. Launched in 2021, the initiative is led by a Steering Group made up of ERN coordinators, industry representatives, researchers, and EURORDIS.
