Edition of 04 February 2017

Several OrphaNews editorials this year have charted progress along the road to European Reference Networks, or ERNs. In essence, ERNs are networks connecting expert centres in the field of rare disease and highly specialised healthcare. On 15^th December 2016, the Board of Member States of ERNs voted to approve the 23 ERN proposals submitted in June of last year (the 24^th ERN proposal will be evaluated early in 2017). The concept of ERNs has been developing and maturing over the past five years, since the publication of the Directive on the application of patients’ rights in cross border healthcare; consequently, the approval of the Networks constitutes a watershed moment for all stakeholders in highly specialised healthcare. The development marks a major innovation in care for Europe’s millions of rare disease patients: although pan-European structures exist in the research domain, this is the first such enterprise in the health sphere.

Europe now moves from an era of hypothetical ERNs to the actual implementation of the Networks. It will likely take several years for the ERNs to truly reach their potential, and multistakeholder support -as well as political will and commitment- will remain essential over the coming months and years:

• Patients must continue to strive for meaningful input to all aspects of Network operations, which should be greatly facilitated by the ePAGS (European Patient Advocacy Groups), an initiative spearheaded by EURORDIS (which was instrumental in developing the concept of ERNs almost a decade ago);
• Member States must continue to explore how best to link ERNs with existing national structures and pathways for patients, to benefit fully from this new resource;
• The European Commission will hopefully continue to support the ERNs as hitherto, ensuring collaboration across the various units of DG Sante (and beyond) and maintaining its robust–indeed, often tireless- support for a true European innovation;
• RD-Action, the Joint Acton for Rare Diseases, will continue to work with –and for- the ERNs, to support them in exploring and addressing their shared challenges around policy issues such as data-sharing, registration, clinical guidelines, therapy development etc.
• Last but not least, it is essential that the 23 (soon to be 24) ERN coordinators are granted time and all possible support, to embrace the huge task of implementing these (brand new) Networks created across broad thematic areas (http://www.rd-action.eu/european-reference-networks-erns/coordination-of-rare-disease-erns/)   

The launch of the ERNs will be celebrated in the 3^rd European Commission Conference on ERNs, in Vilnius, Lithuania from 9-10^th March 2017. The second day of the conference will be dedicated to individual Kick-Off-Meetings for each of the ERNs.

Meanwhile, as the ERNs commence work and agree their short, medium and long-term priorities, it will be important to capitalise on the fruits of past investment and efforts, wherever possible: the Networks will need to share good practices relating to data collection and standardisation, to optimise the value of precious patient information for improved one-to-one care, whilst also creating a critical mass of data to propel the field forwards; greater awareness of the state of the art in terms of clinical guideline generation and implementation should make it easier to identify what works best for patients, in terms of interventions and care management, and by pointing patients towards their optimal -perhaps personalised- care pathways, precious health system resources may be deployed more wisely and advantageously in future; ERNs should be able to access existing pan-European resources to facilitate research (from bench to bedside) including cutting-edge platforms to integrate and analyse ‘big-data’ and hopefully provide a diagnosis which otherwise might never materialise.

The ambitions of the Networks are substantial, and the various elements making-up the ‘ideal’ ERN will not fall into place overnight. December 15^th brought an early Christmas present to all Europeans living -and working- with rare diseases (and indeed any condition requiring a particular concentration of specialised expertise); for this reason, 2016 will always constitute a special milestone on the road to ERNs – but it will be in 2017 that the Networks take their first steps.