The European Health Data Space (EHDS) - Building a path for rare diseases and learning from its experience

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The European Health Data Space (EHDS) - Building a path for rare diseases and learning from its experience


Participants welcomed the report “Building a path for rare diseases in the European Health Data Space” produced by Friends of Europe during an event on 8 November and shared their views on how a European Health Data Space (EHDS) can benefit rare diseases.

“The last few months have shown a clear acceleration in telehealth,” said Tamsin Rose, Senior Fellow at Friends of Europe and the event moderator. “But is it permanent? Today we can see where the gaps are, as well as the opportunities for change.” (00:01:10)

Before opening the floor to participants Rose briefly summarised the report, in particular listing the four principles, namely:

  1. Ensure federated access to harmonised rare diseases health data in an interoperable continuum encompassing epidemiological, healthcare, research, quality of life and treatment-related data
  2. Build partnerships for rare diseases and leverage existing initiatives such as the European Reference Networks (ERNs)
  3. Develop digital skills for the rare diseases community
  4. Build on experiences from the rare diseases community to empower health data users through systems of trust

If EHDS can work for the rare diseases community, it will work for everyone.

Data can build knowledge

When it comes to rare diseases, sharing data is part of the DNA. Yet there is a risk that data related to rare diseases could be lost in the bigger picture of health-related data – so how do we ensure that the specific nature of rare data can be accommodated in the data space?

“The silo approach per disease or per country goes nowhere,” said Yann Le Cam, Chief Executive Officer of the European Organisation for Rare Diseases (EURORDIS). “It’s a deadlock. Only a standardised system can work.” (15:00)

“It’s true that for rare diseases we are less mature, but that may turn into an advantage as it gives us more flexibility to shape the system.” (16:16)


Ease of access to data is a prerequisite for a successful EHDS. “It is essential that it is easy to use for all stakeholders across Europe,” said Le Cam. “Data is knowledge, and knowledge is life.”

“Data can also build our knowledge base,” he continued, “which will enable us to learn about the causality of new diseases, which in turn will enable us to build better health strategies in the future.” (00:18:01)

“The question of access is not just for researchers and industry, but also for public bodies,” added Andrzej Ryś, Director at the European Commission Directorate-General for Health and Food Safety. (00:31:18)

Quality of data

“[The] EHDS will provide us with a huge opportunity to look at the bottom of the iceberg,” said Ana Rath, Director of ORPHANET.

“At the moment not all rare disease patients are reaching their expertise networks, and the burden of rare diseases is completely hidden and off the radar of policymakers,” she said.

“[The] EHDS would give us the evidence to enable us to make informed decisions that can change the lives of patients suffering from rare diseases,” she continued. “But for this to happen, we need access to data. We need common data models and a set of standards that are interoperable, and we need mapping systems that will mitigate the lack of quality in data sources.” (00:20:17)

Data sharing

“[The] EHDS is the future for management of data coming from hospital for the service of the patient,” said Maurizio Scarpa, Coordinator of the European Reference Network for Hereditary Metabolic Diseases (MetabERN), explaining that the European Reference Networks (ERNs) are formed from hospitals, but this in turn raises an issue. (00:36:54)

“Each hospital is a silo,” he explained. “Hospitals are generating billions of data, but it is unstructured, not of good enough quality and not interoperable. This limits the sharing of data.” (00:37:37)

“We need to develop projects to change the mentality of generating data,” he explained. “We need infrastructure to collect data and need to be led by our motto: share can cure.” (00:39:24)

Stelios Kympouropoulos MEP added that the European Parliament can “work towards creating a common governance to collect as much data as possible to promote the transfer of knowledge and best practices, with the ultimate objective of having a global community of knowledge.” (00:47:31)
Role of EU?

Juozas Olekas MEP proposed a stronger European health union. “We need to discuss the need to strengthen EU influence in healthcare,” he said. “We need to change the treaties to give more responsibility to the EU on rare diseases.” (00:53:22)

Speaking on behalf of the European Commission, Ryś said that a lot of the recommendations in the Friends of Europe report are on his organisation’s radar.

“We need to build on our success stories,” he said. “When we set out the future framework, we need to build on our experience and progress on the work we have done. For example, the digital skills agenda is very important, and hopefully we can invest more in this field.” (00:29:31)

He also spoke about the need for a “European trust agenda”, adding that this must be built into future legislation around the EHDS, for example around consent forms. (00:30:43)

Kympouropoulos agreed, stating that patients “must feel their data is secure and is treated in compliance with applicable data protection framework.” (00:48:55)

Responding to a comment from the moderator that there was often a gap between what was agreed at the EU level versus implementation on the ground, Ryś said that the individual needs of member states need to be understood.

“Member states are developing at different speeds,” he said. “Some are setting up future elements of [the] EHDS, and we want them to pilot the exchange of data. At the same time, we are continuing setting up national contact points, and countries are already sharing their experiences.” (00:33:24)

“ERNs are close to my heart,” said Tapani Piha, Senior Advisor at FI-Sitra. “We will need to build the EHDS from the elements that we have at hand, and ERNs are one of these.” (00:42:34)

Piha argued that Europe can create “an umbrella of different initiatives” and expand this cross-border exchange of data. (00:44:21)

“We need smoother access across borders, and this is where [the] EDHS can bring added value,” he added. (00:44:43)

“The ability of the EU to demonstrate its value has become very clear,” concluded Tamsin Rose. “If we can get the EDHS to work properly and effectively, this will have a huge impact for citizens, and they will start to see that Europe that works for them.” (00:59:52)



Conceived as an interlinked system that offers access to portable, comparable and interoperable health data from across the EU, the European Health Data Space (EHDS) aims to promote the reuse of health data, boost research and ensure that citizens have control over their own data.

In the context of the lead up to its launch at the end of the year, Friends of Europe has convened a working group building on the assumption that rare disease patients are the best rationale for the EHDS. A priority for the upcoming French presidency of the Council of the European Union, the limited number of rare disease patients and the scarcity of knowledge and expertise make rare diseases a field that greatly benefits from the European joined action.


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The European Health Data Space (EHDS) - Building a path for rare diseases and learning from its experience
Expand The European Health Data Space (EHDS) - Building a path for rare diseases and learning from its experience

The connectivity of everyday life continues to accelerate and the healthcare sector is part of this rapid digital transformation. The potential of data to deliver more effective and efficient care increases the imperative to build a shared EU health data environment.

COVID-19 has revealed the vital importance of accurate health data shared across the fragmented landscape of national and local agencies that have different ways of collecting information.

The new European Health Data Space (EHDS) will federate datasets to enable greater exchange and access to information. If well designed, this will unlock the value of data at hand into products and services to support innovation, create new medical insights and improve delivery of care for patients.

The field of rare diseases is ideally positioned to both contribute to and benefit from the EHDS. The value of learning from smaller populations is two-fold; addressing unmet medical needs as well as generating insights that can be applied to larger patient populations.

  • How will the European Health Data Space support better health outcomes for patients? Will the needs of people living with a rare disease be met through the proposed EHDS?
  • Will the European Health Data Space adequately address the differing requirements of patients, clinicians, industry and researchers?
  • How can the long experience of data sharing within the rare disease community via the European Reference Networks (ERNs) and other mechanisms inform the governance, structure and operability of the EHDS?


Andrzej Ryś

Director at the European Commission Directorate-General for Health and Food Safety

Stelios Kympouropoulos

Member of the European Parliament Special Committee on the COVID-19 pandemic: lessons learned and recommendations for the future

Yann Le Cam

Chief Executive Officer of the European Organisation for Rare Diseases (EURORDIS)


Juozas Olekas

Member of the European Parliament and former Lithuanian health minister

Ana Rath

Director Orphanet

Peter Arlett

Head, Data Analytics and Methods Task Force, European Medicines Agency

Diana Buzoianu

MP Romania

Maurizio Scarpa

Coordinator, European Reference Network for Hereditary Metabolic Diseases (MetabERN)

Wout Feitz

Coordinator eUROGEN

Celina Ramjoué

Deputy Head of Unit, DG CONNECT, European Commission

Peggy Maguire

Director General European Institute of Women’s Health

Bert Verdonck

Lead of Health Data Initiatives and GAIA-X, Philips

End of Debate


Yann Le Cam
Yann Le Cam

Chief Executive Officer of the European Organisation for Rare Diseases (EURORDIS)

Show more information on Yann Le Cam

Yann Le Cam has dedicated close to 30 year to raising awareness of rare diseases as a public health priority in Europe and beyond. In addition to co-founding and leading Eurordis, which gathers today 751 rare disease patient organisations in 66 countries, he has held a number of high-ranking positions in leading medical research NGOs and patient organisations. He is also recognised for his particular contribution to the adoption of many European regulations that impact the lives of rare diseases patients. From 2016 – 2019, Yann Le Cam served as a member of the European Medicines Agency’s (EMA) Management Board.

Andrzej Rys
Andrzej Ryś

Director at the European Commission Directorate-General for Health and Food Safety

Show more information on Andrzej Ryś

Andrzej Rys has held several key positions in local and national health institutions, notably serving as the Director of the Health Department of the City of Kraków, President of the Polish Public Health Foundation and Polish Deputy Minister for Health, a role which put him on the negotiation team for Poland’s accession to the EU. He later founded and led the School of Public Health and the Centre for Innovation Technology Transfer at Jagiellonian University. In 2006, Rys joined the European Commission as the Director for Public Health and Risk Assessment and was later appointed Director for Health Systems, Medical Products and Innovation at the Directorate-General for Health and Food Safety, where he works on improving EU healthcare systems for citizens.

Juozas Olekas
Juozas Olekas

Member of the European Parliament and former Lithuanian health minister

Show more information on Juozas Olekas

Juozas Olekas is a Member of the European Parliament and was recently nominated to the European Parliament’s Special Committee on the COVID-19 pandemic. He is the former Lithuanian minister of defence and minister of health, has served as a member of the Lithuanian parliament, and actively participated in the processes of restoring Lithuania’s independence. Prior to entering politics, Olekas led a distinguished career in the medical field for nearly two decades. A doctor by training, he was notably an associate professor at the Vilnius University Faculty of Medicine and the chief doctor of VU Žalgiris Hospital.

Photo of Maurizio Scarpa
Maurizio Scarpa

Coordinator, European Reference Network for Hereditary Metabolic Diseases (MetabERN)

Show more information on Maurizio Scarpa

Maurizio Scarpa has extensive expertise in the treatment of rare paediatric disorders. As coordinator of MetabERN, he oversees a comprehensive network that connects existing highly specialised healthcare providers across the European Union to improve the care and treatment of all patients affected by rare metabolic disorders. Scarpa is also the president of the Brains for Brains (B4B) Foundation, which seeks to promote an international network of health professionals in rare disorders, specifically in the field of rare neurological, paediatric diseases.



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