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Rare Diseases - a turning point

An accumulation of pressure from patients, practitioners and key opinion leaders has riled up what can only be described as a perfect storm around rare disease policy.  It has been a long time coming as the last European rare disease strategy was over a decade ago, in 2009. Since then, the pandemic has mainlined health awareness across the world and with it, science, technology and most recently our approach to public health has changed. So what has been the drive behind this apparent movement towards reform? As is only right, at the heart lies the drive to improve the lives of the estimated 30 million people living with a rare disease in the EU. With this in mind, political momentum has been building up, from the European Parliament to the EU presidency of the Council.

Rare 2030 - a new study on rare diseases

In February 2021, the European Parliament, together with Eurordis (Rare Diseases Europe) presented a 2-year foresight study named Rare 2030 that proposed policy recommendations that would improve policy and a better future for people living with rare diseases in Europe. This brought a consensus on what rare disease policies in Europe should be for the next decade, including novel recommendations for a renewed European framework for rare diseases to bridge the gap between policy areas and member states.

EU Council Presidency

The move towards policy reform around rare diseases took another huge step once the trio EU Council Presidency of France, the Czech Republic and Sweden included rare diseases in the programme of their priorities. In reality, this translates to a series of events, opening with a High-Level Conference organised by France’s Ministry of Health and Solidarity on the occasion of Rare Disease Day, which will bring together key policymakers to set out recommendations for change to European legislation on rare diseases.

This is no small feat, and should not be seen for any less than what it is: the European Union's roadmap to collectively prepare for the adoption of a new European plan for rare diseases, which should include a comprehensive framework connecting the dots between different national and EU initiatives and policy areas.

A decisive moment in time

It is great news that the realisation has finally come that it is time for decisive change to improve the precarious and uncertain situation for patients with rare diseases. Of the estimated 30 million EU citizens that have been diagnosed with some 6000 to 8000 different diseases, only 6% of these diseases can be treated with effective medical therapies. To improve this shocking statistic we need an EU wide framework that stops patients from wandering and missing opportunities.

This will lead to international communication between health institutions and alleviate internal structures that currently often hold back multidisciplinary discussions. On top of that, this framework would lead to improving the access patients have to optimal therapies and medicines. We can all agree that healthcare and the world’s perception of health have drastically changed since 2009. Hopefully, this realisation will push forth the process of realizing these necessary changes. The EU needs to lead the way in a forming a digitalized, inclusive, and purposeful framework for rare diseases.

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Want to know more?

For more information please contact Guus de Serière