Add to this the establishment of organisations which act as patient-driven alliances, such as the European Organisation for Rare Diseases (EURORDIS), and the International Niemann-Pick Disease Alliance (INPDA), and you have a solid base upon which together we as a rare disease community have, and will continue to, prosper.
With an estimated 6,000 rare diseases, and approximately 20-30 million people living with them in Europe alone – EURORDIS, a non-governmental patient-driven alliance of both individuals and patient organisations in the rare disease field, plays a crucial role. It represents more than 700 rare disease organisations spanning 63 countries across the world, and is one of the founders of Rare Disease Day. As the organisation enters its 20th year, we are able to look back on some of their fantastic achievements from across the years:
It is the awareness of rare diseases, such as Niemann-Pick, that push the conversation to the forefront – and unfortunately, 20 years ago society was living in relative ignorance of rare diseases. Now, they are thankfully better understood and their impact is felt through initiatives such as Rare Disease Day, which since 2008 has involved tens of thousands of separate events held by thousands of patient organisations in over 100 countries. This unity is what EURORDIS prides itself on, as collectively our numbers as a rare disease community are too large to ignore.
After awareness comes engagement, especially by those affected by rare diseases such as family members and friends. Thanks to EURORDIS developing advisory groups and committees that bring together patients, patient engagement in rare disease policy, research, healthcare and development of medicines has never been higher. In fact, patients have become an integral part of the policy development process at the European Medicines Agency (EMA), and through the establishment of 23 European Reference Networks, as well as the recently launched European Patient Advocacy Groups, collaboration between patients, clinicians, and policymakers, is set to further improve in coming years.
It can be incredibly difficult to obtain a correct diagnosis initially for rare disease patients, and this often serves as a huge obstacle that can often take years to overcome. However, progress has been made in this area over the past 20 years thanks to EURORDIS, due largely to the push for more funding and research from the rare disease community and their allies. Recently several groups came together in order to publish ‘International Joint Recommendations to Address Specific Needs of Undiagnosed Rare Disease Patients’ to further substantiate the need for more aid in this area.
Recently our very own Chief Executive, Toni Mathieson (pictured), was in attendance at the ‘EURORDIS Awards & Black Pearl Evening 2017’, which was both a celebration of Rare Disease Day, and also a meeting that hoped to display solidarity and hope for rare disease patients throughout Europe:
‘Every year the Black Pearl event raises awareness and celebrates the achievements of those working hard to serve people living with rare disorders. EURORDIS leads the field, making this year’s celebration of EURORDIS’ two decades of achievement especially worthwhile.’
– Mark Krueger, MPH, President, Mark Krueger & Associations Inc. USA.
‘The EURORDIS Awards and Black Pearl are part a unique joint effort to bring together all stakeholders in the fight for better treatment options in rare diseases.
It is important to remember that it was EURORDIS, along with its Council of National Alliances, who first launched Rare Disease Day back in 2008. Held on the last day of February, it seeks to raise awareness of the impact rare diseases can have on the lives of those affected by them – the Black Pearl event donates a portion of the funds generated towards organising this extensive and important awareness-raising campaign.
You can find out the latest news about the rare disease community on eurordis.org/enews. You can also sign up to EURORDIS eNews to receive updates twice a month. Now available in English, French, German, Italian, Portuguese, Russian, and Spanish.
