Status Quo of European Reference Networks
2016 is an exciting year for the rare disease community: this is the year which saw the first call for European Reference Networks (ERNs) and the first proposals submitted. (An ERN is -or will be- a network connecting providers of highly specialised healthcare, for the purposes of improving access to diagnosis, treatment and high-quality care for patients with conditions requiring a particular concentration of resources or expertise.) The five years since the publication of the Directive on the Application of Patients’ rights in cross-border healthcare (Directive 2011/24/EU) have been filled with concerted efforts and hard work by thousands of rare disease stakeholders across Europe, to move the ERN concept into reality.
The fruits of these efforts are the 24 Network proposals submitted to the European Commission in June and July. The potential Coordinators of the proposed Networks are based in 7 EU Member States: Austria; France; Germany; Italy; Netherlands; Spain; United Kingdom. However, as over 800 healthcare providers (centres with specialised expertise) have applied to partner in these proposals, there is strong pan-European participation. At present, the ERN proposals and the accompanying applications for membership are being reviewed and assessed according to the formal DG Sante procedure. The successful Networks should be approved by the end of this year.
The ERN deadline coincided with the end of the 1st year of RD-ACTION, the Joint Action for rare diseases, a key focus of which has been in fact to support the rare disease field in developing and implementing robust ERN proposals. For instance, the RD-ACTION team at Newcastle University established a ’Matchmaker’ resource to support RD experts in organising themselves into collaborative –as opposed to competing- proposals, adhering to the rare disease Groupings recommended by the Commission Expert Group on Rare Diseases. The team worked closely with clinical groups and existing networks to develop single, comprehensive proposals in each area. The resulting proposals –none of which have yet been approved, of course- are summarised here . Meanwhile, RD-ACTION partner EURORDIS initiated the European Patient Advisory Groups (ePAGs) to support meaningful patient participation in ERNs.
With the ERN proposals now submitted, implementation of the first Networks becomes ever closer and several relevant activities are converging:
• The Tender concerning the study on the services to be provided by the ERNs and their members, awarded to PricewaterhouseCoopers, will hold its final workshop in September, and will shortly after deliver its final outputs (including a proposed catalogue of healthcare services to be provided by the future ERNs, along with cost drivers)
• 19th July was the deadline for ’Requests to Participate’ in the Tender for the ERNs’ ’IT platform’ (formally called ’the Scalable Software as a Service for a clinical patient management system to support ERNs in the diagnosis and treatment of rare or low prevalence complex diseases or conditions across national borders’. A period of competitive dialogue will now commence.
• The Board of Member States of ERNs will convene next in September, and will meet with the potential Coordinators to discuss key issues such as how to involve all Member States & EEA countries in each Network (particularly important for the smaller countries) to ensure meaningful pan-European access.
• RD-ACTION Policy workpackage will follow-up this important meeting with the first in a series of workshops designed to support the Networks and generate guidance on key topics such as standardising and sharing data for care, integrating with rare disease research infrastructures, supporting the development of Orphan Medicinal Products and more. This will help to ensure that the ERNs embody the good practices already defined and deployed by the broader rare disease field, to complement the state of the art in care and research.