The launch of European reference networks for rare disease

16 February 2017

On March 1, the European Commission announced the launch of 24 European Reference Networks (ERNs) for rare diseases.
This was the culmination of considerable effort on the part of the European Commission, EURORDIS (The European Rare Diseases organisation of which EAT is a member), and the health authorities in the Member States of the EU (and the EEA) who have worked together to develop the concept of ERNs following the 2011 EU Directive concerning cross-border healthcare (which provides a legal right for patients to seek appropriate care in any EU country).

These networks involve leading hospitals, referred to as HCPs (Health Care Providers) who will tackle complex and/or rare diseases that require specialised treatment and where benefit is best derived from a concentration of knowledge and resources.
Each ERN covers a group of Rare Diseases and in total there are over 900 health care units from 313 hospitals in 25 countries across the ERNs.
One of the ERNs is focused on rare gastro-intestinal diseases and is known as ERNICA (ERN for Rare Inherited and Congenital Diseases); significantly for EAT, oesophageal atresia (OA) falls within its remit.
On March 9 -10 the European Commission held an ERN launch conference attended by over 500 delegates including the Commissioner for Health & Food Safety (Vytenis Andruikaitis ), and presided over by DG SANTE Director-General (Xavier Prats Monné).
Two EAT Board members, Anke Widenmann-Grolig and Graham Slater, were invited to attend this conference in official capacities.

In 2016, Anke was elected by EURORDIS member organisations as an ‘ePAG’ (European Patient Advisory Group) and she represents EURORDIS in the ERNICA management structure. In addition, Graham has been appointed as a member of ERNICA’s Advisory Board.

Patient involvement is a core element in the governance of all ERNs and guarantees of a ‘patient voice’ in the management of ERNICA was key to its approval by the Board of Member States. The guidance for ERNs is clear that “patients and patient representatives should play an integral role in the decision and opinion making process in rare disease ERNs and be involved in structural and clinical network activities”.

Twenty of Europe’s leading HCPs (hospitals) are members of ERNICA and its Cordinator (i.e its Leader) is Professor Rene Wijnen – Head of Paediatric Surgery at Erasmus MC–Sophia Children’s Hospital in Rotterdam (The Netherlands). The Deputy Cordinator is Professor Tomas Wester, Head of Paediatric Surgeon at Karolinska University Hospital, Stockholm (Sweden).

Why are ERNs so important (and why is ERNICA of benefit to OA patients)?

Once established the ERNs will support the exchange of knowledge and expertise between HCPs. Wherever possible it is envisaged that expertise will ‘travel virtually’ rather than the patients traveling physically, but this does not rule out the opportunity, where appropriate, for a patient to actually move to the expertise.
Imagine that a clinician is struggling to determine the best way to treat a patient with a complex form of OA. Supported by ERNICA this clinician will, in future, be able to ‘refer’ this patient not only to a recognised centre in her own country but to the most appropriate centre in the entire network.
Using a dedicated IT platform, essential clinical information could be shared with the appropriate experts, and in this way ERNICA will facilitate the chances of finding the best surgery or course of treatment for that patient.
Additionally ERNs will help realise a ‘critical mass’ of data through the extended use of registries, facilitate research, drive innovation and – critically – provide another means to promulgate existing guidelines and ‘best practice’ which often exists today but is poorly implemented.

Concluding remarks
It is important to note that member states remain responsible for national health policy and that they had (and continue to have) the responsibility to endorse HCPs as national recognised centres of excellence, a prerequisite for their membership of an ERN.
But, to quote Commissioner Vytenis Andruikaitis, ‘no country alone has the knowledge and capacity to treat all rare and complex conditions’, and the benefits of ERNs include their ‘potential to unlock tangible benefits for patients with and complex presentations of disease’ (quote: Matt Bolz-Johnson, Director Healthcare & Research, EURORORDIS).

Graham Slater.

Leave a Reply

Your email address will not be published.