Eurordis - Rare Disease Europe

The eight final recommendations covering diagnosis, treatment, care, research, data and European and national infrastructures sets out the roadmap for the next decade of rare disease policies.

 

Recommendation 1: A European policy framework guiding the implementation of consistent national plans and strategies, monitored and assessed by a multistakeholder body on a regular basis.

 

 

 

Recommendation 2: Earlier, faster and more accurate diagnosis of rare diseases through better and more consistent use of harmonised standards and programmes across Europe, new technologies and innovative approaches driven by patient-needs.

 

 

Recommendation 3: A highly specialised healthcare ecosystem, with political, financial and technical support at European and national levels, that leaves no person living with a rare disease in uncertainty regarding their diagnosis, care or treatment.

 

 

Recommendation 4: Guarantee the integration of people living with a rare disease in societies and economies by implementing European and national actions that recognise their social rights.

 

 

Recommendation 5: A culture encouraging meaningful participation, engagement and leadership of people living with a rare disease in both the public and private sectors.

 

 

Recommendation 6: Rare disease research maintained as a priority – across basic, clinical, translational and social research.

 

 

 

Recommendation 7: Data used to its maximum to improve the health and well-being of people living with a rare disease.

 

 

 

Recommendation 8: Improve the availability, accessibility and affordability of rare disease treatments, by attracting investments, fostering innovation and collaboration across countries, to address inequalities.

 

 

Watch a discussion about the Rare 2030 Recommendations in focus with a panel of experts.

To prepare a better future for people living with a rare disease the Rare 2030 Foresight Study called upon Europe’s most dedicated doctors, companies, researchers, young citizens, advocates, patients, payers and policy makers. 

Together they comprised several expert panels of over 250 key thought leaders and thousands of people living with a rare disease who have all participated in this iterative, participatory and inclusive study between January 2019 and March 2021. 

EURORDIS led the study, which had nine partners from across Europe.

It followed a four stage process using foresight:

1. 1. Establishing what we already know through workshops, surveys, research and interviews, to develop in eight Knowledge Base Summaries setting out the current policy development and needs across different areas. This was the bedrock of the work
   2. Forecasting current and future trends, identifying 12 trends clustered around changing values and changing technology that will affect rare disease policy
   3. Preparing four potential scenarios based on the trends that could play out by 2030, and deciding on the preferred scenario “Investment for social justice”, prioritising patient needs-led innovation and collaboration

• Develop policy recommendations based on the preferred scenario.

Using foresight 

Foresight helps to prepare for the future. This exercise is extensively used across policy development in spheres such as energy, public transport and geo-politics. They are more rarely used in health. By analysing current and future trends and applying them to various scenarios, foresight expands our thinking and uncovers inevitable or near-inevitable futures. 

 

This allows challenge to  conventional wisdom and enables the design of strategic pathways. It also is a process of co-production. Starting from the same evidence based, people from across the field and beyond can decide, together, on the recommendations. Actors are all on the same page, and can therefore move in the same direction. 

The COVID-19 pandemic has shown that foresight in health, and particularly for the most vulnerable of Europe’s citizens, is proving instrumental in preparing resilient and strong health and social sectors.

Trends 

Through a series of consultations held during meetings in 2019 (the EURORDIS Membership Meeting, the Panel of Experts Workshop, the EURORDIS Council of National Alliances and Council of European Federation meeting), the Rare2030 Panel of Experts summarised and validated 12 trends that have been identified as instrumental to shaping the future of rare disease policy. 

Two clusters of trends emerged and their progressions created four possible future scenarios varying in degrees of solidarity and drivers of innovation. 

Scenarios 

With the information collected through the knowledge base and trends identified through the Rare2030 Foresight study, the Panel of Experts put together four possible future scenarios depicting what the world may be like for people living with a rare disease in 2030.

Watch the video to see the four scenarios: https://youtu.be/WBvsj9E0U-g 

The four scenarios left the rare disease community with a choice on which direction they wanted to move in for a better future. Their choice: a future based on “INVESTMENT FOR SOCIAL JUSTICE”, defined by patient-need driven innovation; high collective responsibility and performance based goals. This is driven by approaches prioritising human rights, multi-stakeholder and patient partnerships, technology and the holistic view to care and research. Importantly, this highlights the key principles justifying a coordinated European, and even an international approach, to address the challenges of rare diseases.

The Rare 2030 Foresight Study took a truly multi-stakeholder approach with hundreds of experts and thousands of patients feeding into the study through workshops, interviews meetings, consultations and surveys. 

A multi-stakeholder Panel of Experts comprised of over 250 thought leaders in the field of rare diseases and health served as the central consultative body during the Foresight study.

Project Partners 

EURORDIS-Rare Diseases Europe led the Rare 2030 Foresight Study. Nine project partners, from academic institutions, patient organisations and clinical networks worked extremely closely on delivering the project. 

Research Advisory Board

The Rare 2030 Research Advisory Board represented one panel of key opinion leaders involved in the Rare 2030 Foresight Study. It is composed of high level experts in the field of rare diseases, in health and research policy in general, emerging technologies and foresight studies – complementing the governance of the project.