Building a path for rare diseases in the European Health Data Space

Data sharing through the proposed European Health Data Space (EHDS) has the potential to improve health care services, policymaking and health system administration, as well as research. It will also make the lives of every patient easier, especially rare diseases patients. Imagine being able to share medical data with healthcare professionals in other European countries. This will soon become the reality right across Europe.

Our latest report “Building a path for rare diseases in the European Health Data Space” highlights how the rare diseases community can serve as a model case for the health data governance framework in the European Union.

“The needs of people living with a rare disease could be met through the proposed EHDS and, conversely, […] the long-term experience of data sharing within the rare diseases community via the European Reference Networks (ERNs) and other mechanisms can inform the governance, structure and operability of the EHDS,” writes Tamsin Rose, Senior Fellow for Health at Friends of Europe.

The report outlines four key principles that not only optimise the use of rare diseases data but could be used more widely for all medical data within the EHDS:

Principle 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

• Recommendation 1: Implement a federated, interoperable common access interface for all health data
• Recommendation 2: Create common rules for access to data

Principle 2: Build partnerships for rare diseases and leverage existing initiatives such as the European Reference Networks (ERNs)

• Recommendation 3: Include the European Reference Networks (ERNs) to pilot the EHDS

Principle 3: Develop digital skills for the rare diseases community

• Recommendation 4: Develop a pipeline of data specialists
• Recommendation 5: Support increased data literacy for healthcare professionals and the public

Principle 4: Build on experiences from the rare diseases community to empower health data users through systems of trust

• Recommendation 6: Build systems of trust through a clear legislative framework for health data governance
• Recommendation 7: Support patients’ data custodianship

Each recommendation includes concrete actions for guidance. This report is based on the outcomes of three working group sessions organised by Friends of Europe in 2021, which brought together a wide range of stakeholders from healthcare institutions, international organisations and industry to healthcare providers and patients.