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#ActRare2024: Optimised data for patient and societal benefit

Июнь 2024

In this latest article of our series on our #ActRare2024 policy recommendations, we explore with Professor Dipak Kalra, a former general practitioner and President of the European Institute for Innovation through Health Data (i~HD), why Europe’s health systems can, and must, make sure that health data is used to improve the lives of rare disease patients.

Professor Dipak Kalra on the opportunities to be seized:

Following the vote on the European Health Data Space (EHDS) legislation in April, we spoke with Professor Kalra about how enhanced health data usage could improve the lives of people with rare diseases. Drawing on his experiences, he pointed out that data can be effectively utilised in two main ways, providing immediate short-term advantages and offering medium- to long-term benefits.

Speaking of the short-term advantages of optimised health data management, Professor Kalra emphasised that the EHDS can play a fundamental role in ensuring continuity and safety of care. Patients with rare diseases often see specialists who know them or are familiar with their condition, but challenges arise when they need care in settings where their medical history is unknown, particularly during unplanned or urgent situations, such as while travelling.

“I think the prospect going forward, that attracts me for patients with rare diseases, is the ability for them to be confident that wherever they are, at least a modest summary of their health background information, and importantly, including their medication list, is available in a cross border interpretable way.”

The European Health Data Space would, therefore, ensure that essential health information, like diagnoses and medication lists, is accessible across borders, enabling healthcare providers to quickly understand a patient’s medical history.

In the long term, shared health data has immense research potential. Rare diseases require collaborative research beyond national boundaries. Professor Kalra noted that no single country has enough patients with rare diseases for comprehensive research, so a Europe-wide data-sharing approach could greatly accelerate research and innovation.

Enhanced data sharing would enable more effective research into care pathways, treatment efficacy, and even the development of new medicines and medical devices. For instance, monitoring tools and artificial intelligence applications could be developed to predict and manage disease flare-ups more effectively, thereby improving patient outcomes and quality of life.

Policy implications and the role of the EU institutions

Professor Kalra describes the current EHDS landscape as a «hurtlingly exciting opportunity space,» offering an unprecedented chance for Europe to advance rare disease diagnosis, treatment, care, and research.

However, he emphasises the importance of recognising the legislation’s limitations to ensure rare disease patients are not overlooked, especially regarding the secondary use of data for research purposes.

Professor Kalra argues that the current regulation favours public authorities’ access to data, often at the expense of industry and academic researchers, who are crucial for driving innovation. He advocates for a balanced framework that includes multi-stakeholder participation while protecting patient data.

At EURORDIS, we support industry access to data under strict criteria beyond profit-seeking, ensuring responsible and ethical use of patient data to drive innovation.

Additionally, according to Professor Kalra, current patient summaries are insufficient for rare disease patients, as they often lack hospital discharge reports due to patients receiving intensive care at clinics. He suggests adding “rare disease annexes” to these summaries to provide critical information to healthcare providers, facilitating better treatment across Europe, even when traveling abroad.

The need for ambitious advocacy

It is crucial that rare disease patients are not left behind in the EHDS and its legislation, and EURORDIS is calling for the next set of EU policymakers to #ActRare2024, to see that the development of the EHDS is inclusive and comprehensive with respect to people living with rare diseases to make sure no patient is left behind. This includes pushing for detailed implementation of regulations that support not only the ethical use of data but also the practicalities of data sharing and interoperability across borders.

As Prof. Kalra outlines, it’s the details that matter, especially for rare disease patients:

“It’s important that we focus on treatment innovation, diagnostic innovation, monitoring innovation and, absolutely the vitality of public-private collaboration on those innovations – areas the EHDS is currently not favouring enough.”

In conclusion, Prof. Kalra’s insights underscore the importance of turning words into action. Whilst manifesto pledges are important calls for officials to agree upon, it’s important that they are developed, and turned into concrete actions with robust ethical frameworks in place.

«While manifesto principles are great, the real question is, where do you go from there? It’s important to think more deeply about specific issues, like ethical use or data sharing, and develop detailed proposals or position papers with specific actions and considerations.”

It is the research, development and implementation of the EHDS which will really matter for patients with rare diseases.

EURORDIS’ call for optimised data for patient and societal benefit

Reflecting on the position of Professor Kalra, Jelena Malinina, Data Director at EURORDIS, emphasises the importance of the EHDS and its potential for rare disease patients. She describes the EHDS proposal as “highly ambitious and much needed”, stressing that it must be thoroughly managed and aligned with both a higher vision and the realities of Member States and their available resources.

“We were greatly encouraged by the Parliament’s adoption of measures that would ensure patients’ immediate access to health data,” recounts Jelena.

“These measures would empower patients by providing them with direct access to their personal health data processed within health services, including indispensable information like patient summaries and laboratory results. Such transparency and immediacy in accessing one’s own health information is critical, especially for the rare disease community where timely information can significantly influence treatment outcomes.”

Amongst EURORDIS’ eight manifesto points within the #ActRare2024 campaign is the call for the EU to support the harmonisation, optimisation, and interoperability of electronic health records to ensure safe and timely sharing of health data across countries, thereby facilitating cross-border healthcare.

We are calling for new data sharing governance to reflect patients’ needs – in terms of developing robust standards ensuring secure, ethical, and responsible data sharing, and allowing health data to be seamlessly shared across borders to benefit all people living with a rare disease.

As we are currently at an exciting moment within the landscape of European Health Data, we must ensure that the voices and needs of the rare disease community are not forgotten.

This is why the future EU policymakers must #ActRare2024 and implement strategies that will enhance the quality of life for the 30 million individuals impacted by rare diseases across Europe.


Millie Trsic, Communications Intern