Parliament calls for action plan to improve rare disease care in Europe
EURORDIS estimates 30M in EU live with conditions like AADC deficiency
Members of the European Parliament (MEPs) spanning party lines have called for a coordinated effort focused on improving the lives of the estimated 30 million people living with a rare disease in the European Union, according to a press release from EURORDIS – Rare Diseases Europe.
Such an initiative would include efforts to facilitate more accurate diagnoses, better treatment, and improved care for people living with rare conditions such as aromatic l-amino acid decarboxylase (AADC) deficiency.
“For me, there are around 30 million reasons why we need to take bold action now,” said Tilly Metz, an MEP from Luxembourg.
“Thirty million children and adults across Europe have to fight every day,” Metz added. “This is why a European Action Plan on Rare Diseases is needed — and is needed now.”
Need for cross-border care emphasized
The call to action was welcomed by EURORDIS, a nonprofit alliance of more than 1,000 rare disease organizations across 74 countries. Still, the organization’s CEO, Virginie Bros-Facer, PhD, voiced concerns that existing policies are outdated and opportunities to develop a better strategy are being missed.
“If we continue with only these outdated tools, we will fail to tackle the challenges our community faces in a coherent, forward-looking way,” Bros-Facer said. “What we need now is a comprehensive European Action Plan that connects the dots, sets measurable goals, and delivers tangible results.”
Rare diseases are defined in the EU as conditions affecting fewer than 5 in 10,000 people. Among them is AADC deficiency, a very rare condition that causes neurological problems early in life.
People with rare conditions commonly face diagnostic delays because these diseases are under-recognized in clinical communities. Treatment options can also be more limited and costly.
MEPs believe that a coordinated effort across the EU to address these challenges would improve outcomes for those with rare conditions. The debate surrounding the issue occurred at the European Parliament’s recent plenary session.
If we continue with only these outdated tools, we will fail to tackle the challenges our community faces in a coherent, forward-looking way. … What we need now is a comprehensive European Action Plan that connects the dots, sets measurable goals, and delivers tangible results.
At the start of the debate, the European commissioner of health and animal welfare, Olivér Várhelyi, discussed a variety of ongoing initiatives and reforms in the EU that could have benefits in the rare disease community.
“In the EU, we estimate that around 30 million people live with a rare disease,” Várhelyi said. “So while rare diseases are rare, patients living with them are not.”
“A strong European Health Union helps improve the health of all our citizens — no matter where they live, no matter their disease or complex condition,” he added.
Parliament members stressed that despite these efforts, there’s still a need for a clearly defined and coordinated framework to prevent health inequities and improve care for those with rare conditions across Europe.
They believe that patients who can’t receive the right care in their own country should be able to easily access cross-border care in other EU countries.
“We need a European strategy that helps us overcome the fragmented and piecemeal approach we currently have – one that ensures all patients can access treatment, regardless of which member state they come from,” noted Letizia Moratti, an MEP from Italy.
Parliament urged to accelerate research into rare disease in Europe
Others noted the need for the Parliament to commit to accelerating research into rare conditions, training more clinical specialists, and building care centers.
“This is necessary so that in the future, we can talk not about how many children we’ve lost — but about how many lives we’ve saved,” said András Tivadar Kulja, an MEP from Hungary.
EURORDIS last year published an open letter to the European Commission outlining a set of actionable priorities for improving rare disease care, which were also reflected in its #ActRare2024 campaign. That campaign was launched ahead of last year’s European elections.
The organization emphasized that EU member states should connect and align on rare disease strategies, in addition to pooling resources and expertise to make the EU a global leader in rare disease innovation.
Bros-Facer noted that if a new strategy isn’t implemented to address the challenges faced by the rare disease community, diagnostic delays, mental health challenges, and research paucities will persist.
“Thanks to efforts at both national and EU levels, many essential building blocks are already in place,” the CEO said. “What’s missing is the mortar – a strategy that binds these elements into a durable, impactful whole. Only with the ‘strategic mortar’ of a European Action Plan can we [mold] these disparate components into coordinated and transformative change.”
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