On the occasion of the 25th anniversary of EURORDIS, we highlight the major achievements and successes over the past quarter-century. We’ve watched our community grow and become stronger than ever before, so we would like to reflect on why this work matters and how it should move forward.
“Thanks to the collective effort of our 1000 member organisations, partners and stakeholders, we have succeeded over the past 25 years in taking rare diseases from ignorance and indifference to knowledge and hope as a health priority area at the global, European, and national levels.”
Yann Le Cam, EURORDIS Chief Executive Officer
European advocacy – frameworks to incentivise research, coordinate policies and connect knowledge around Europe
With the adoption of the EU Regulation on Orphan Medicinal Products in 1999, EURORDIS has contributed to establishing a centralised procedure for the designation of orphan medicinal products and putting in place incentives for their research and development.
Almost a decade after that, in 2008, the European Commission adopted the Communication “Rare Diseases: Europe’s Challenges“ and a year later in 2009, the EU Council adopted a “Recommendation on an action in the field of rare diseases”. These two milestones paved the way for an integrated EU strategy on issues including diagnosis, treatment, and care, and encouraged national plans to support the 30 million Europeans living with a rare disease.
Since then, we’ve seen the establishment of the 24 European Reference Networks and increased rights of people with rare diseases, following the adoption of the EU Cross-border health care Directive.
Elevating the patient voice through Rare Barometer
To ensure that our advocacy is meeting the needs of the patient community and is heard in Europe and internationally, EURORDIS launched in 2015 Rare Barometer, a key EURORDIS initiative, which engages with rare disease patients to help them influence healthcare policies that affect them. This interactive survey panel collects the experiences of over 15 000 people that are living with or affected by a rare disease, to make the voice of rare disease patients stronger and promote patient-centred policy.
The past 25 years of taking forward the patient voice to EU Institutions have given us the progress that we would not have had otherwise. But the work is not yet finished, so we will put our best foot forward to work toward establishing a European strategy for rare diseases that works for all.
Health care cannot take a ‘one size fits all’ approach, which is even more true when it comes to 6000 rare diseases, each of which requires individual diagnostic and management strategies. Addressing the challenges of personalised care and finding the right expert for a rare disease is essential.
Other ways of catalysing networks, exchanging experience, coproducing knowledge and cooperating with members of the wider rare disease community – researchers, pharmaceutical companies, and policy makers – also include the EURORDIS Round Table of Companies (ERTC) and the European Conference on Rare Diseases and Orphan Products (ECRD).
Making sure the patient voice sits at the core of healthcare organisation through ePAGs
For this reason, EURORDIS, together with the rare disease patient community, has created 24 European Patient Advocacy Groups (ePAGs), to bring together rare disease patient advocates and facilitate their active involvement in the work, development and governance of the European Reference Networks (ERNs). Each of these groups corresponds to the disease grouping of one of the 24 ERNs, aligning patient organisations, clinicians, and researchers working on the same rare or complex diseases.
Creating space for industry to discuss the most pressing issues at ERTC
Additionally, with the creation of the EURORDIS Round Table of Companies in 2004, we have been bringing together corporate health sector representatives to share their expertise in engaging public and private institutions involved in the rare disease and orphan drug fields. Over 30 ERTC workshops have been organised to date, and its members include some 70 entities, from start-up biotechs to mid-sized and large pharmaceutical companies, based in Europe and the United States. The importance and added value of this platform have been enormous, with health sector companies understanding better the patient perspective and taking measures to tackle issues shaping care and access to treatment for rare diseases.
Driving dialogue, learning and networking for the whole community through ECRD
The year 2001 saw the first European Conference on Rare Diseases & Orphan Products taking place in Copenhagen. Since then, every two years EURORDIS has been gathering together over 1000 stakeholders in the rare disease community to shape goal-driven rare disease policies and allow for important and innovative discussions on a national and an international level to take place. ECRD has built momentum for rare diseases over the past years: policy makers, clinicians, and industry are now taking our concerns and aspirations seriously, and what was once a rare sight in decision-making is now becoming more and more common as rare disease awareness shifts.
Engaging various stakeholders in collaborative action has allowed members of our community to coordinate their efforts and has been one of the key strategic initiatives EURORDIS has executed to elevate the needs of the 30 million people living with a rare disease in Europe. And while great progress has been made, we will continue to work towards more sustainable, needs-led and result-oriented public-private rare disease partnerships and keep strengthening the patient voice through effective communication between EURORDIS, national alliances, members and other organisations.
Throughout the years, we have encouraged people living with a rare disease, their carers and families to be active participants in their own lives, to improve their experience and have better healthcare outcomes.
As our community has grown to unite over 1000 patient organisations in 74 countries, we recognise the importance of patient engagement – not only for those who have one of the 6000 plus rare conditions but also for clinicians, companies and public institutions to understand and respond to the needs of those living with a rare disease. And so, we need to keep building on this nurture and grow the rare disease ecosystem for the next 25 years.
Bringing the community together on Rare Disease Day
Thanks to Rare Disease Day, which has grown to become a global movement encompassing 100 countries, we have succeeded in connecting people and creating a dialogue between patient advocates, researchers, policy makers and industry. This campaign, soon to be in its 15th year, has been hugely influential in helping people who felt isolated or marginalised to recognise that there was a community that they didn’t know existed – a large and strong community of 300 million people.
Empowering people with rare diseases to influence EU initiatives
Rare Disease Week has fostered the involvement of patient advocates in EU decision-making, allowing for a direct line of communication with Members of the European Parliament and other EU representatives. By engaging people with rare diseases in EMA committees or the ERNs through the European Patient Advocacy Groups (ePAGs) and numerous task forces, we’ve facilitated communication and collaboration between these communities in order to contribute to regulatory processes or shape research directions that are in keeping with the interests of those living with a rare disease.
Training patient advocates to be experts in medicine development through the EURORDIS Open Academy
As part of the EURORDIS Open Academy, established in 2018, we’ve helped over 608 people affected by a rare disease to have the confidence and knowledge needed to bring their expertise to discussions on health care, research and medicines development with policy makers, industry and scientists. Empowering people with rare diseases with inclusive and easy-to-use digital tools is also an important part of our work, and is in itself a must-do in today’s world. Through the Open Academy online training, we’ve helped people with rare diseases and their families navigate the regulatory maze leading to drug development, successfully advocate for more funding for rare disease research, build partnerships, and engage their local communities, among other things. To this date, over 1800 people have taken one of the 20 online courses, and this number continues to grow.
Check out our achievements and milestones by year.
Watch a series of videos with the Board of Directors members sharing their thoughts on the major achievements of the rare disease community over the past 25 years.