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2021 Rare Diseases

The future of rare diseases starts today

James © Ceridwen Hughes : Same but different

Dr. Ana Rath

Director, Us14 Inserm – Orphanet

Yann Le Cam

Chief Executive Officer, EURORDIS

New policy recommendations from the rare disease community aim to improve the future of those living with a rare disease in Europe.

The health and future of 30 million people living with a rare disease in Europe should not be left to luck or chance. Today, 20 years after the first European rare disease legislation, many patients in Europe still have unmet needs; the road to a diagnosis is long, treatment options are lacking and limited support provided.

Success by design

A renewed policy framework is needed to ensure that the rare disease community’s vision for the future is made a reality and to ensure the best outcome whatever happens. This is the conclusion of two years of work and hundreds of rare disease experts through the Rare 2030 Foresight study, Rare 2030, funded by the European Parliament.

A renewed policy framework

The Rare 2030 Foresight study for rare disease policy has brought together over 200 stakeholders to define the rare disease community’s vision of the future and the actions needed to achieve this.

This desired future prioritises innovation responding to unmet needs, with a strong sense of collective accountability demonstrated by solidarity between citizens and countries, with diseases constituting a policy priority.

Making new policy recommendations

The project’s final recommendations for a new policy framework were presented at a final online conference on 23 February 2021 to over 800 participants, including Members of the European Parliament and representatives from the European Commission. These recommendations consider the full rare disease ecosystem, from public health to research and innovation, social affairs and technology.

Helping to inspire global change

Although intended to help shape a new European policy framework for rare diseases, these recommendations can also provide inspiration for countries outside the EU and other sectors. It is hoped that these recommendations will help pave the way to a brighter future where no person living with a rare disease is left behind.

Yann Le Cam, Chief Executive Officer, EURORDIS-Rare Diseases Europe and Rare 2030 project coordinator underlined that, “Rare diseases know no borders; all countries in Europe – including the UK – should see the Rare 2030 Recommendations as the direction we need to go, together, to continue to move to a better future.”

Ana Rath, Director of Orphanet and Rare 2030 project partner, highlighted that, “Now more than ever, European and national institutions have a vital role to play in improving the health of citizens across Europe. These recommendations provide a roadmap for further work over the next decade to improve the journey of living with a rare disease for patients and families.”

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