Yann Le Cam
Chief Executive Officer, EURORDIS-Rare Diseases Europe
A clear, uniform and proactive strategy across Europe, supported by every country in the region, is required to optimise the way that rare diseases are diagnosed, treated, cared for and researched.
Due to the nature of rarity, the challenges that face the 30 million people living with a rare disease in Europe can only be addressed through cooperation and solidarity. However, these cornerstones of success do not just happen. There is currently no overarching action for rare diseases at a European level and policies have not kept pace with new technologies.
This is why we are calling for the adoption of Europe’s action plan for Rare Diseases 2030 to address the unmet needs and inequalities. This would serve as a cross-cutting strategy across geographic borders and sectors to consistently support national activities to ensure that no one living with a rare disease in Europe is left behind.
Rare diseases have become pioneers in tomorrow’s transformations and creative solutions for research and healthcare.
A goal-oriented approach
Unlike other public health priorities, such as cancer and obesity, rare diseases currently have no overarching goals that, as a society, we can work towards. By setting such targets at a European level, we are presented with a roadmap for all European countries to ensure that inequalities in addressing the challenges faced by people living with a rare disease are not exacerbated by their country of residence. In turn this could see 100 million years of life gained across Europe.
This should include goals to:
- Reduce diagnostic delays for 90% of people living with a rare disease from years or decades to within six months.
- Reduce premature death of children under five by 30%.
- Extend life expectancy by an average of three years across diseases thanks to better management and integration of medical and social care.
- Reduce psychological, social and economic vulnerability of people living with a rare disease and their families by a third.
- Ensure the approval of 1,000 innovative (symptomatic or transformative) treatments for people living with a rare disease.
Investing in a wider society
Although our call is directed at the European Union institutions, we know that such a strategy would have benefits across Europe at large: rare diseases know no borders and require collaboration across sectors.
Rare diseases have become pioneers in tomorrow’s transformations and creative solutions for research and healthcare. During the COVID-19 pandemic, we have seen how techniques, such as the RNA vaccines, are based on gene editing techniques, refined over decades to find cures for rare diseases, 72% of which are genetic.
Europe can become a world leader in rare and precision medicines. It can and should defend a model of care that leaves no one behind.
