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Novembro 2022

The Momentum Builds: European Economic and Social Committee calls for an action plan on rare diseases


Ensuring straong european solidarity for rare disease patients

“Having heard evidence from people right across the patient community and civil society, we learned that there is huge, unrealised potential for EU policy to make sure scientific, technological, clinical and social advances reach every European citizen living with a rare disease”, explains Alain Coheur, the Rapporteur who led latest the European Economic and Social Committee’s (EESC’s) latest Opinion on Ensuring strong European solidarity for rare disease patients

Speaking to EURORDIS, Mr Coheur insists that a “European action plan and establishing a solidarity fund would be key to delivering equitable and affordable access to medicines, and solidarity with the rare disease community.” 

 The EESC’s Opinion states that the Committee “deeply regrets that, more than ten years after the adoption of its Opinion, it has to reiterate its call for a comprehensive European approach that takes into account all the needs of people with rare diseases”. Crucially, the EESC adds yet another voice to the growing chorus of calls for a European action plan on rare diseases, accompanied by a series of SMART goals. 

Although the EESC laments the fragmented and inequitable policy approaches pursued thus far, it also shines a light on the EU’s opportunities to create a better tomorrow for the millions living with a rare disease across Europe. 

 We take a look at some of the EESC’s recommendations, and explain why their adoption would make tangible improvements to the lives of people living with a rare disease. 

What has the EESC recommended? 

Establishing measurable goals 

The EU doesn’t just need the promise of actions. The EU requires SMART goals – goals which are Specific, Measurable, Achievable, Relevant and Time-bound, and attainable by 2030. We had called for the adoption of SMART goals in the EU-funded, EURORDIS-led Rare 2030 Foresight Study

These goals would help make sure that the EU is held accountable and compelled to ensure that all patients enjoy equal opportunities to receive diagnoses, treatments, and more integrated care. 

Having carefully listened to voices from across society, including those from our rare disease community, the EESC is absolutely correct to assert that comprehensive policy action must be driven by specific goals. Without such goals, patients risk being left disappointed by a lack of genuine progress.

Yann Le Cam, Chief Executive Officer, EURORDIS

Optimising the use of health data 

The Opinion rightly states that a European Health Data Space (EHDS) is “necessary” for faster diagnoses and can make “new, safer, personalised treatment accessible sooner”. 

The EHDS, which the European Commission has been endeavouring to set up, is of particular importance in the field of rare diseases, as better ways of using data hold great potential to advance scientific research and improve clinical practices for conditions where medical knowledge and expertise are limited. 

Our position paper on the EHDS, published last month, cites the finding from our 2019 Rare Barometer survey that 95% of rare disease patients are not only willing to share their data for primary health uses, which directly improve services for whom the data relates; they are also keen to share their data for secondary health purposes, facilitating health providers, researchers and regulators to use data for the purposes of advancing medical research and improving policymaking. 

The challenge of collecting enough data on specific rare conditions affecting small patient populations at national level can be addressed by building safe and reliable systems for the collection and use of data at the European level. The successes of Orphanet and the European Reference Networks have long demonstrated the value of improved data-sharing to healthcare specialists and those living with rare conditions.

Valentina Bottarelli, Public Affairs Director & Head of European and International Advocacy, EURORDIS

Speeding up diagnoses 

The EESC stresses the importance of “accessing a rare disease diagnosis during perinatal or neonatal screening”, and among the specific SMART goals recommended is an aim for patients to receive a diagnosis within one year. 

Given that an estimated 72% of rare diseases are of genetic origin, the EESC is right to highlight the unparalleled opportunity newborn screening offers to improve the health and well-being of people living with a rare disease. 

Screening for rare conditions in newborns holds the potential to lengthen, improve, and even save many lives. We want to see Europe take action, to reduce existing inequalities in newborn screening between countries and increase the quality of life of all children, no matter where they are born.

Gulcin Gumus, Research & Policy Project Manager, EURORDIS 

Support for – and beyond – the European Reference Networks 

Similarly to EURORDIS’ Recommendations to achieve a mature ERN system by 2030, the EESC calls for the optimised functioning of the European Reference Networks (ERNs) and their “integration into the entire EU and healthcare systems of the Member States”. 

We welcome the EESC’s call for ERNs’ further integration into healthcare systems, which EURORDIS has been actively supporting, as ERNs should provide a key advisory role in cross-border decision-making for rare disease cases.  

The EESC also recognises the needs of those with conditions not covered by ERNs and suggests a European solidarity fund to prevent rare disease patients from incurring unbearable costs for their care. 

Solidarity and greater European cooperation must drive policy action to ensure access to cross-border healthcare is based on needs, rather than people’s ability to afford travelling to a different country where specialist knowledge and healthcare is available.

Ines Hernando, ERN & Healthcare Director, EURORDIS 

Improving access to orphan drugs

The EESC states that the upcoming Revision of the EU’s Orphan Medicinal Products (OMP) and Paediatric Regulation requires an “ambitious approach” and should consider “models of joint purchasing and contributions” to extend Member States’ and patients’ access to treatments. 

As reported in our latest Proposal on the upcoming Revision of the Regulation, too many rare disease patients cannot get the treatments they need because they are not available where they live. We firmly agree with the EESC that the EU must use the Revision to create a robust regulatory framework that drives innovation and expands treatment access. 

We welcome the strong support coming from the EESC to ensure inequalities in access to therapies are addressed through solidarity instruments. That’s exactly what the rare disease community needs, and it shows concretely the added value of European concerted action.

Simone Boselli, Public Affairs Director, EURORDIS 

The need for a European action plan

The EESC’s Opinion includes many more recommendations, from empowering patient advocates to delivering greater support for informal carers. But they all fall under one, overarching recommendation: committing to a goals-based European action plan on rare diseases. Through our #30MillionReasons campaign, EURORDIS has long called for an action plan. 

The EESC Opinion follows hot on the heels of the Czech Presidency of the EU having supported EURORDIS’ call for an action plan, and makes an appeal to the next trio-presidency of the Council – Spain, Belgium and Hungary – to keep rare diseases high on the agenda. 

Successive Council presidencies, Members of the European Parliament, and now the European Economic and Social Council, have all allied themselves to the call for an action plan.  

It’s time for the European Commission to sit up, listen, and act on the unmet needs of people living with rare diseases. 


Julien Poulain, Communications Manager