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Febbraio 2021

EU Fund on Rare Disease Reflection Paper EURORDIS 


In today’s world, science and technology offer an unprecedented chance to address the unmet medical needs of people living with a rare disease. This potential is currently not translated into actual health benefits for the large majority of people living with a rare disease due to issues concerning availability, accessibility and affordability of treatments.  

Coordinated, more strategic policies are required to address these shortcomings and inequalities and ultimately improve the health outcomes and quality of life of people living with a rare disease, where the added value of European action has never been in doubt, but that now needs a transition from incremental improvement to success by design.  

This reflection paper was developed in the context of the French Presidency of the EU Council to consider what kind of flagship initiatives could be developed to steer and support Member States in addressing inequities in access across countries and in maintaining the sustainability of their healthcare system. 

An EU-Fund is proposed to help finance access to the transformative and potentially curative gene and cell therapies for very rare diseases and co-finance the generation of post-marketing authorisation evidence across Member States during the years initially following approval, in order to reduce impact of uncertainties whilst at the same time allowing for timely access to life saving therapies.