Seizing opportunities for rare disease patients

As the end of the Parliamentary term approaches, it’s time to proactively push for improvements in the lives of people living with rare diseases

By Tilly Metz

Tilly Metz (LU, Greens/EFA) is a member of Parliament’s Environment, Public Health and Food Safety Committee

22 Feb 2024

As we approach the end of the European Parliament mandate, there lies a pivotal opportunity to champion the cause for rare disease patients.  

I see the urgent need to address the challenges faced by these individuals. Today, 95 per cent of rare diseases lack authorised treatments. For me, this is a clear sign of the inadequacy of the existing pharmaceutical model. My objective is to create a framework that ensures the availability and affordability of orphan medicines, thereby addressing a critical gap in patient care. 

The European Health Data Space emerges as a game-changer, empowering efficient research for precise diagnoses and treatments through secure and ethical health data sharing. I am empowered by the majority of patients' support for this initiative, and I remain determined to deliver tangible results.  

“95 per cent of rare diseases lack authorised treatments… a clear sign of the inadequacy of the existing pharmaceutical model”

Looking ahead, my ambition for the future is to make a European strategy on rare diseases a reality. This strategic initiative will help to integrate all European Union actions into the evolving European Health Union, maximising its potential. Let us squeeze out of it as much as we possibly can for the benefit of all patients.  

As we approach the end of this Parliamentary term, my call to all is clear – we must seize the moment to proactively advance improvements for people living with rare diseases. 

Read more about Rare Disease Day on our Rare Disease Hub or in our Rare Disease supplement.

Read the most recent articles written by Tilly Metz - Public demand is clear but political will could be a blocker for EU action in health


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