European Reference Networks for Rare Diseases – The Neuromuscular Field

Background:

In Europe a rare disease is defined as a disease that affects less than 1/ 2000 people. Neuromuscular diseases (NMD) are among these rare diseases. They are complex, progressive, multi-system disorders, mostly of genetic origin but also acquired and they can present from childhood to adulthood.

Most of the NMD need multidisciplinary care and due to their rarity and diversity pose specific challenges both for healthcare provision and for research. Networks of highly specialised healthcare professionals, arranged either in a formal or informal way, have proven to be a way to address this problem. The TREAT-NMD Alliance, for instance, is an excellent example of what can be achieve with networking and exchange of knowledge. Other projects like NEUROMICS or RD-CONNECT also reinforce this aspect.

There have been many efforts at EU level to improve the profile of rare diseases and to develop mechanisms which will allow care for patients with RD to be better organised. One of these initiatives is the creation of European Reference Networks (ERNs). In the neuromuscular field much has already been done regarding networking however, most of the work was done on a research basis and it is now time for the field to join efforts and organize ourselves in a ERN both for care and research.

Important documentation:

European Reference Networks (ERNs) have been a priority of the EU Health policy since the publication of the Directive on Patient’s Right in Cross-Border Healthcare in 2011.  ERNs main aim is the promotion of equal access to the most up-to-date care and advice to all individuals, regardless of where they live in Europe.
The Commission has adopted in 2014 the Delegated Decision 2014/286/EU setting out the Criteria and Conditions for ERNs and their Members and the Implementing Decision 2014/287/EU setting out the Criteria for Establishing and Evaluating the ERNs and their Members. In the same year the Commission also published the Rules of Procedures of the Board of Member States of the European Reference Networks.

Important steps in the near future:

  • The European Commission is seeking to support EU Member States in the development of ERNs and is presently putting together a series of initiatives. Among those initiatives is the 2nd European Reference Networks Conference: from planning to implementation that will be held on the 8-9 October 2015 in Lisbon.
  • The Member States are due to be identifying and connecting healthcare providers and centres of expertise at a national level and also nurturing the participation in the ERNs.

The first call for Networks is now open.


General aspects regarding ERNs:

  • ERNs are a mechanism of cooperation between healthcare providers of highly specialised services all over Europe
  • ERNs should improve access to diagnosis, treatment and provision of high-quality healthcare
  • ERNs should act as hubs for medical training, research and dissemination of information
  • Healthcare providers will benefit from an improvement in knowledge, international recognition and leadership in their area of expertise
  • ERNs will be built around thematic groups; so that every RD is covered.
  • Healthcare providers are expected to collaborate amongst themselves in a dedicated field of expertise in order to establish a Network.
  • The Members of a network will work in accordance with the legislation of their Member State.
  • The Members of a network are expected to take full responsibilities, tasks and participation in all technical and governance bodies of the ERN
  • They should act as a hub between the national healthcare providers and the ERN
  • ERNs are an attempt to formalise existing informal networks and bring together highly specialised healthcare providers in different Member States, for conditions that require a high concentration of resources and expertise.
  • All ERNs will have a common EC logo and registered trademark.
  • Each Network will be required to report to the European Commission on its activities.
  • The participating organisations should ensure they can dedicate sufficient time and resources to the Network.
  • Key elements for the establishment of the Networks:
  • Should be patient centred and clinically lead
  • Should include a minimum of 10 Healthcare providers in at least 8 Countries
  • ERNs will be submitted to an independent assessment, by an independent assessment body
  • ERNs must fulfil the criteria set  by the Commission Implementing Decision
  • Healthcare providers must fulfil the criteria to become a Member of the ERN and must have the endorsement and approval by National Authorities

Networks criteria and capacities

  • knowledge and expertise to diagnose, follow and manage patients
  • proved evidence of good outcomes
  • multi-disciplinary approach
  • capacity to produce good practice guidelines and to implement outcome measures and quality control
  • research, teaching and training
  • collaborate with other centres of expertise and networks

Timeline for the Setting up of the ERNs:

  • 2nd half 2015 Call for selection of independent bodies to evaluate ERN
  • January-May 2016 Call for Networks
  • 2nd half 2016 Establishment of ERN
  • End of 2016 Network is operational
  • 2020 Evaluation

Opportunities for the Neuromuscular Field:

This collaboration will help with the adoption and spread of innovations in medical science and health technologies.

A NMD ERN can act as a focal point for medical training and research, helping to establish an European CV, helping to disseminate standards of care and general knowledge about NMD.

It is expected that ERNs will have a high profile and recognition in their particular domain. This will attract the best experts and more patients and although ERNs are supposed to facilitate the cross-border health care it is expected that the pooling of resources and the e-Health solutions will decrease the burden of travelling for the patients.

The Networks and their members will be in a good position to apply for more research funding and further develop and accelerate basic and translational research.  

 
12 Apr 2016