The Challenge

In 2017 24 European Reference Networks (ERNs) were launched, virtual networks involving more than 900 highly specialised healthcare units from over 300 hospitals across 26 EU countries.

The aim of the ERNs is to improve care for patients with rare or low-prevalence complex diseases, by facilitating discussion on such conditions and concentrating knowledge and resources across Member States. As clinical research networks, ERNs hold an incredible amount of knowledge and data on rare diseases, which can support the research and development of new therapies and support greater understanding of these conditions. However, there is no overarching framework for ERNs to engage with developers to exchange and collaborate using this knowledge. Moreover, in 2019 the Board of Member States of ERNs published a Statement outlining the principles of ERN and industry collaboration, but there are restrictions on funding and governance arrangements which make collaboration practically challenging.

Stakeholders approached FIPRA with a clear ask: to address the challenges around ERN-third party collaboration, with a view to unlocking innovation for some of the 95% of rare diseases still without a dedicated treatment.

The FIPRA Approach

The challenge called for a collaborative approach, bringing together ERNs, industry, patients and EU decision makers. To this end, FIPRA helped establish agile 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: Together for Rare Diseases (Together4RD).

The multi-stakeholder initiative is led by a Steering Group, which provides strategic input to, and oversight of, the Together4RD workplan. Steering Group members include ERN Coordinators, representatives from EU research initiatives, patient association representatives and industry partners. DG SANTE acts as an observer to the Steering Group’s work.

Moreover, four cross-party MEP Champions from the European Parliament support awareness raising and advocacy efforts, with a view to overcoming the existing barriers to more collaboration, and setting out a vision for what a more collaborative rare disease ecosystem could look like.

The Outcome

Together4RD was launched in 2022, hosting a panel discussion at EURORDIS’ European Rare Disease Conference 2022 and publishing an article authored by the Steering Group Chair in The Parliament Magazine.

Since then, Together4RD has launched key policy asks at an event in the European Parliament (Brussels) and the World Orphan Drug Congress (Barcelona), with key actions needed to unlock ERN-industry collaboration. A White Paper, to be published in the academic journal of Orphanet, is due in 2023.

Throughout, FIPRA has implemented a communications and engagement strategy, to build support for the Steering Group’s work and advocate for concrete actions at EU level that are needed to bring change.