Inés Hernando joined EURORDIS as ERN and Healthcare Director in February 2018. In this role she is responsible for monitoring the European landscape on rare disease healthcare from a policy and advocacy perspective, and managing all related activities. Inés supports the establishment of ERNs by leading the EURORDIS team that manages the 24 European Patient Advocacy Groups (ePAGs) that were set up by EURORDIS in 2016 to ensure the meaningful engagement of patient advocates across all ERN activities. Previously she worked for 2 years at COCIR as eHealth Senior Manager where she led the organisation’s work on all topics related to digital health, focusing specifically on issues such as data protection, interoperability and cross-border data flows. She also worked for 8 years in the Spanish Electronic Health Record initiative as eHealth Coordinator where she provided overall programme management and helped build the programme partners’ ecosystem that included all Regional Health Authorities and the Ministry of Health. Inés holds a Master’s in Health Economics, Policy and Management from the London School of Economics.

Can you please introduce what EURORDIS does to make the voice of patients affected by rare diseases heard by policy-makers, researchers and the society in general?

EURORDIS-Rare Diseases Europe is a unique, non-profit alliance of 894 rare disease patient organisations from 72 countries that work together to improve the lives of the 30 million people living with a rare disease in Europe. By connecting patients, families and patient groups, as well as by bringing together all stakeholders and mobilising the rare disease community, EURORDIS strengthens the patient voice and helps to shape research, policies and patient services.

We advocate at European and international levels to make rare diseases a public health priority and have created numerous initiatives to make the voice of patients affected by rare diseases heard by policy-makers, researchers and society. These initiatives include a network of parliamentary advocates for rare diseases which is made up of MEPs and MPs, Rare Disease Day, the EURORDIS Open Academy and the NGO Committee for Rare Diseases at the UN, to name just a few.

How does EURORDIS support the work of the European Reference Networks?

For years, EURORDIS has led the rare disease patient community’s advocacy efforts to create ERNs and has worked hand in hand with all stakeholders to develop the policy recommendations that have helped to shape the ERNs’ structure and scope. Since the ERNs were launched, EURORDIS has worked to ensure patients are engaged in the governance, strategic and operational delivery of the ERNs by creating 24 European Patient Advocacy Groups, which bring together over 200 patient advocates who are actively involved in the ERNs. These patient advocates are the bridge between the Networks and the wider rare disease patient community. They gather the needs of the rare disease patient community and discuss with clinicians how the Networks can best address these needs and, at the same time, they share with that community the information on the activities, services and knowledge that the ERNs are producing and curating. 

Through the EURORDIS Leadership School, we provide patient representatives participating in the ERNs with the knowledge and skills they need to engage and partner effectively with the clinicians in ERN activities. We also support national alliances around Europe to actively help in integrating ERNs into national health systems.

Is there a specific message you would like to pass on concerning rare diseases patients?

Some ERNs still need to build the space and mechanisms for patient representatives and clinicians to exchange and work together on a regular basis. We also still need to identify where the input of patient representatives is most valuable for the ERNs so that their involvement can be targeted to these areas, and identify the most effective tools for their involvement. At the moment, for example, we are having very good results with the patient journeys as a tool to collect and present patients’ needs throughout the care pathway in a structured way. They allow patient representatives to convey very valuable information on the needs of the wider patient community that can then be used to inform some of the Networks’ core activities.

On a positive note, we are very pleased with the support that all the Networks will receive to develop new and review existing clinical practice guidelines and other clinical decision support tools. Access to evidence-based highly specialised healthcare is what will make all the differencein improving health outcomes and the lives of people living with a rare disease. Of course, we would like to see meaningful (including financial) support to enable patients to get involved in this activity in all ERNs. The involvement both of patient representatives and clinicians in the ERNs remains voluntary, and will at some point evaporate due to a lack of support for these volunteers.

Another aspect that is critical for the patient community at this moment is the assessment of the centres that want to join the networks. Poor quality care negatively affects the health and wellbeing of our community, therefore we need to ensure all the applications are rigorously assessed. One that is transparent and provides clear assurance to our community that the right experts are connected under a given ERN. In order to be effective, the assessment system needs to be well resourced and supervised by the European Commission to ensure that the technical assessment process is duly executed end-to-end. It is very important that the technical part related with the expertise and experience of the candidates is performed by the Board of the networks, as they are the best placed to assess and peer review this set of specific criteria. To maintain public confidence, independent validation of a proportionate sample of the applications remains critical.

Finally, at this stage the networks still require strong political support at EU level and a more concrete commitment at national level to integrate ERNs into national health systems. They also require acceptance by national authorities and hospital managers that clinicians need time and resources to work on ERN collaborative activities.

Those are four messages, rather than one! But they can be summed up in one sentence: we need better patient-clinician partnership, quality assurance and sustained leadership.