Speaking in Parliament on Tuesday at a major event on rare diseases, Fajon spoke of the “frustration” about the “widespread inequalities” facing people who have a rare disease.
Fajon has a rare disease herself, having been diagnosed 20 years ago with Chronic myelogenous leukemia (CML), a cancer of the white blood cells.
She said, “We need to push hard as policymakers to get better policies in place to deal with this issue.”
She was speaking during a full day of activities in Parliament organised by EURORDIS - Rare Diseases Europe, which aimed to put a spotlight on the issue.
It included the relaunch of a “Network of Parliamentary Advocates for Rare Diseases” which Fajon said she “enthusiastically supports.”
An estimated 32 million people in Europe, and ten times that globally, live with at least one of the 6,000 known rare diseases.
The event kicked off with an exhibition showcasing artwork created by patients such as artist Phil Herold, who has Spinal Muscular Atrophy (SMA) and an opera performance by Noemie Desquiotz-Sunnen, a singer with Amyotrophic Lateral Sclerosis (ALS).
“We need to push hard as policymakers to get better policies in place to deal with this issue” Tanja Fajon MEP
She said, “Of course my future with ALS scares me a lot. The most terrible thing for me will be to lose my voice. That’s why I don’t want to look back at what I have lost and I also don’t want to look too far into the future; I am living now in the present and for now I can still sing.”
This was followed later on Tuesday by “Reframe Rare”, a policy event which was an opportunity for MEPs, patient organisations, medical professionals and representatives from the world of academia and industry to discuss the reality of living with rare diseases along with “EU coordinated actions.”
Participants heard about the importance of early diagnosis and registries, orphan drug legislation and also highlighted some of the achievements and challenges in the field of rare diseases.
Another participant was Stelios Kympouropoulos, a Greek MEP, who has a “deep” personal experience of the issue of special needs as he was diagnosed with spinal muscular atrophy as a child.
The EPP member said he hopes the Network will help tackle specific challenges faced by people living with a rare disease and ensure stronger EU-wide action through targeted support.
“My future with ALS scares me a lot. The most terrible thing for me will be to lose my voice. That’s why I don’t want to look back at what I have lost and I also don’t want to look too far into the future” Noemie Desquiotz-Sunnen
Another participant at the “fireside chat” discussion was Czech GUE member Kateřina Konečná. She too has thrown her weight behind the Network, saying that she hopes it will ensure that rare diseases are made an “integral” part of EU, national and regional programmes in health.
The meeting heard that last June, the European Court of Auditors recommended a review of current EU rare disease policy strategy, a recommendation endorsed by the Council, which has now led to the Commission announcing that it revise its policy by early 2023.
This has been welcomed by EURORDIS which, since 2008, has helped organise Rare Disease Day (29 February).
Yann Le Cam, Chief Executive Officer of EURORDIS, said “great progress” had been made in the last decade to make rare diseases a public health priority at the EU level and to put in place European and national policies that benefit people living with a rare disease.
He went on, “However, Europe’s challenge to address the needs of 30 million people living with a rare disease is far from won; only 10 years of action cannot do it all. We must build on the achievements of the last decade to build an EU policy framework in the next decade that helps achieve equity for patients and families.”
“Europe’s challenge to address the needs of 30 million people living with a rare disease is far from won; only 10 years of action cannot do it all. We must build on the achievements of the last decade to build an EU policy framework in the next decade” Yann Le Cam, Chief Executive Officer of EURORDIS
“We need to build progress that will translate into meaningful health, social and improved well-being for people affected by rare diseases.”
Le Cam said that with the support of the Parliamentary Advocates for Rare Diseases, “we call on all stakeholders - from the European institutions and national governments, the NGO community, academic and research institutions, as well as the private sector - to join and move forward the conversation about a renewed EU framework for rare diseases.”
The event was told that for the vast majority of cases of the 6,000 rare diseases that are known to exist, there is no cure and few treatments available.
Globally, 300 million people have a rare disease, 30 million of whom are in Europe, representing 5 percent of Europe’s population (the equivalent of the population of Belgium and the Netherlands combined).
The event heard that rare diseases “seriously impacts” on everyday life with, for example, eight out of ten patients and carers having difficulties completing basic daily tasks such as preparing meals, shopping and doing household chores.