Rare disease patients cannot be forgotten: a coordinated approach is needed

This HAE Day, Hilde Vautmans MEP makes the case for a European Action Plan for Rare Diseases to support Europe's rare disease patients

By Hilde Vautmans

Hilde Vautmans (Renew Europe, Belgium) is a signatory of the 2023 MEP call for a rare disease action plan.

22 May 2024

Up to 36 million people in the EU live with a rare disease. Counting their loved ones countless more are affected. Through my involvement with Michiel and Amber, siblings afflicted with progeria, an exceptionally rare and progressive genetic disorder, I've gained insight into the difficulties rare disease patients and their families face. This has underscored the pressing need for cohesive policy measures to address the unmet needs of rare disease patients. The European Union has made significant strides in this regard, such as setting up European Reference Networks to connect doctors, patient representatives, and researchers virtually across borders, but there is still much work to be done.

The 16th of May is a key milestone to raise awareness of hereditary angioedema (HAE), a rare genetic disease characterised by sporadic and unforeseeable swelling episodes affecting different parts of the body, which is debilitating and can be life-threatening without an accurate diagnosis and adequate treatment. On this HAE Day, I would like to highlight the importance of taking action at the EU level to meet the unmet needs of people living with HAE and other rare diseases.

The adoption of a European Action Plan for Rare Diseases would signify a significant stride forward in our collective efforts to combat these rare conditions, such as HAE. It would provide vital guidance to Member States in crafting national strategies, promote the sharing of best practices and expertise across borders, and reaffirming the European Union's commitment to prioritising the well-being of rare disease patients. Importantly, it would also send a clear message that rare disease patients are not forgotten and that their well-being is a priority for the European Union.

Our liberal group Renew Europe has been at the forefront of advocating for a European Action Plan for Rare Diseases, recognising the necessity of a structured framework to confront the unique challenges posed by these conditions. Such a plan would not only elevate the voices of rare disease patients at the European level but also foster collaboration among Member States and stakeholders to enhance diagnosis, treatment, and support services.

At the heart of any well-worked plan there must be a commitment to ensuring equal access to innovative therapies for all rare disease patients. Too often, European Citizens living with rare diseases face barriers to accessing the treatments they need due to factors such as limited funding for rare diseases novel treatments, limited availability, and lack of awareness among healthcare providers. By addressing these challenges head-on, we can ensure that every rare disease patient has the opportunity to receive the care and support they deserve.

In addition to improving access to treatments, a comprehensive European Action Plan for Rare Diseases must also focus on enhancing research and innovation in this field, an area where efforts have been lacking. Enhancing collaboration among European member states is essential to fill this gap and drive progress. By pooling the knowledge and resources of all 27 member states, we can achieve more significant advancements and ultimately drive progress towards better outcomes for rare disease patients and their families.

In conclusion, addressing the unmet needs of rare disease patients requires a coordinated and comprehensive approach at the European level. We can make meaningful progress towards improving the lives of individuals living with rare diseases and their families by prioritising access to innovative therapies and adopting a European Action Plan for Rare Diseases. As a member of European Parliament for Renew, I am committed to advocating for policies that ensure that no one is left behind, regardless of the rarity of their condition.

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