EURORDIS-Rare Disease Europe’s ERN and healthcare director, Inés Hernando, reflects on the progress of the European Reference Networks two years on.
Launched by the European Commission in 2017, the European Reference Networks (ERNs) represent an invaluable opportunity for Member States to come together to provide highly specialised treatment or care for patients with rare diseases – defined in Europe as conditions that affect fewer than one in 2,000 people.
The European Reference Networks, which currently number 24, comprise virtual networks involving healthcare providers across Europe who discuss complex cases via a dedicated IT platform and telemedicine – a cross-border solution to the challenges of small, scattered patient populations and a scarcity of rare disease expertise.
The non-governmental, patient-driven alliance EURORDIS, the voice of rare disease patients in Europe, played a crucial role in the formation and development of the European Reference Networks, and continues to be involved in their ongoing operation, particularly in relation to ensuring that patients are adequately represented within the networks.
Here, the organisation’s ERN and healthcare director, Inés Hernando, tells Health Europa more about the progress of the networks and the work still to be done.
EURORDIS has long advocated the establishment of European healthcare networks for rare diseases – two years on from their launch, have the European Reference Networks lived up to expectations?
The ERNs were launched in 2017 and already operate on a huge scale, comprising 900 care units within 300 hospitals and roughly 300 patient advocates, as well. The networks are really all about relationships, so in 2018 they were working a lot on getting to know each other because many of those involved hadn’t previously worked together. They have also been working to establish themselves and their governance structures.
EURORDIS and the rare disease community at large have very ambitious expectations of the ERNs. We believe the networks are ushering in a whole different way of organising care for rare disease patients. In November, at the annual ERN conference here in Brussels, we heard about how ERN virtual consultations could contribute to savings; while this is true, focusing on savings only, for us, is quite short-sighted, because the potential of this new structure goes beyond savings.
Efficiency in relation to the ERNs is about changing the way healthcare is delivered to achieve better health outcomes, treatment and quality of life for patients with rare diseases while using public money in the smartest way. Of course, this is a large-scale initiative that sets up a whole new structure for rare diseases healthcare delivery and research, so it will take some time for the ERNs to achieve their full potential.
Nonetheless, we’ve already seen some good activity in terms of training, with some healthcare providers arranging training fellowships for younger doctors. That’s great because it means that we can sustain the capacity and expertise that is needed.
Progress has also been made in identifying gaps in clinical practice guidelines and research, which is great because it builds the foundation for future work. Some 250 virtual consultations have taken place on the most complex cases, in which clinicians have come together over a secure IT platform to discuss a case and decide – where possible – on a diagnosis or treatment.
Several networks have also done good work around recommendations for physicians and GPs who treat patients with rare diseases when they go to the doctor for a normal thing. Another positive step is that all the networks have established their governing boards and patients are represented in all of them.
These are the kinds of things that are already going on and are setting the foundations to enable the ERNs to realise their full ambition of better health outcomes for rare disease patients across Europe, no matter what disease they are living with or where they live.
How can the long-term sustainability of the European Reference Networks be achieved?
The first goal is simply to consolidate the ERNs and co-ordinate their expansion in order to ensure that they cover not only all the Member States but also all the diseases. There are approximately 6,000 rare diseases, so the networks must expand their disease coverage – some more than others. We refer to this as the enlargement process. This year, we will see new healthcare providers joining the ERNs, and this process must be carefully managed.
In addition to that, we need to consider the long-term financial planning of the ERNs and also put in place some kind of steering group to decide on their strategic priorities.
At the moment, Member State representatives and the ERN co-ordinators come together in different working groups to discuss certain key aspects such as the integration of ERNs into national health systems, but what we are lacking is a group where all relevant stakeholders can discuss progress and build a shared vision.
These two things are very much interlinked: once there is a clear strategic roadmap, we can decide where to put the money and how to finance the plans. It is therefore very important to establish a steering group because right now the different stakeholders have different goals and are pulling in different directions.
It is also very important that we can measure the performance of the ERNs. A set of 18 basic indicators that will measure the activity of the ERNs has already been published, and this year each network will have to identify some outcome indicators of their own. These are necessary in order to demonstrate the value of the ERNs, which is vital to securing financial support and building the credibility of this new structure. There needs to be a strong commitment from the Member States, European Commission and ERN co-ordinators on this.
Finally, the networks need to establish a strong governance structure to keep them manageable. Expertise moves – the healthcare providers that are part of the ERNs today might not be part of them tomorrow, and, of course, new healthcare providers might join in the future. There needs to be clear rules around this.
What role is there for Member State governments in ensuring the longevity and proper functioning of the European Reference Networks?
At the moment, the ERNs are reliant on the voluntary co-operation of clinicians and patients, which is not sustainable. The model will only really deliver its full potential if it is fully supported by the Member States. This is why EURORDIS has recently launched recommendations on how to integrate ERNs into national healthcare systems.
In this document we have outlined a number of actions that need to be taken by Member States, including the development of ERN referral pathways to ensure timely access to ERNs’ advice and the revision of their rare disease national strategies to anchor this new structure into their own health systems. Importantly, the ERNs need to respond to the requirements and demands of the national health systems.
For this to happen, Member States need to take ownership of the ERNs and identify clearly their demand of ERN services. Member States need to define how their health systems are going to benefit from the ERNs: do they want to be able to refer a certain number of patient cases or are they more interested in the guidelines or training? Member States will need to commission the ERNs to meet their demands.
They also need to play a major role in raising awareness at national level, because apart from those already involved in the networks, many clinicians and experts in the Member States are not actually aware that this new structure exists, and that makes it difficult for the knowledge that is being pooled and generated in the networks to filter down to national level.
It will also be very important that the ERNs are networks of networks. Some Member States have done a good job of setting up networks for rare diseases at national level and grouping centres of expertise and experts on different diseases, which will make it much easier for them to link with the ERNs.
What are the main barriers to aligning European Reference Networks with national health systems and how might these be overcome?
Possibly right now the main barrier is securing adequate funding. The centres that are co-ordinating each of the networks receive some funding from the European Commission for the co-ordination activities, but this is not enough. The healthcare providers that participate in these networks need to be supported at the national level, which comes back to the hospital managers, who need to support the clinicians involved in the ERNs.
This, obviously, is difficult, as hospitals face many financial hardships. As a result, a lot of clinicians are devoting extra hours to virtual consultations and to the networks’ working groups and activities – none of which is being reimbursed. We need to come up with a mechanism to fund this, otherwise it won’t scale. Ideally, for the virtual consultations this mechanism would have some kind of solidarity fund built into it, because many of the cases that are being referred come from countries with a lower GDP, who don’t necessarily have the capacity needed at national level to treat or diagnose these rare disease patients.
We need to make sure that we are not putting an additional financial burden on these Member States as they will not refer their patients if they cannot afford to. Adequate funding to sustain the involvement of patient advocates in the ERNs is also crucial.
Very much linked to this, our greatest enemy is disengagement. If the doctors and patients involved in the ERNs don’t see things moving, they might start to withdraw.
Finally, in order to fully take advantage as a national health system of the knowledge, guidelines and recommendations being generated by the ERNs, Member States really need to invest at national level in the mechanisms that are needed to translate, own and adopt that knowledge within their own health systems, in order to reap the benefit of their involvement in the networks.
This is why, for example, they will need to develop a mechanism to recognise and adopt at national level the guidelines adopted and reviewed by the ERNs.
Please note, this article will appear in issue 8 of Health Europa Quarterly, which will be published in February 2019.