Opening Remarks by Commissioner Stella Kyriakides at the European Conference on Rare Diseases 2022

(Source: European Commission)

“Check against delivery”

Distinguished Guests, Ladies and Gentlemen,

I am delighted to join you at the 11th European Conference on Rare Diseases and Orphan Products.

This is an area where the European Union needs to make a clear commitment to deliver action for rare disease patients.

The European response spans across different policy areas and provides a comprehensive approach to addressing rare diseases.


It combines six key elements:

  • funding, including through our ambitious EU4Health Programme.
  • speeding up diagnosis and improving standards of care through our European Reference Networks;
  • supporting the definition, codification and registration of rare diseases;
  • promoting the development of national rare diseases plans and strategies;
  • building and broadening the rare diseases knowledge base, also through research; and
  • empowering patient organisations.

These actions bring greater recognition and visibility for rare diseases.

We also have EU legislation in place.

Over the last 20 years our rules provided an environment that encourages the development of medicines for patients with rare diseases, and safeguards their quality, safety and efficacy.

Yet 95% of rare diseases still have no treatment.

Access to orphan medicines, once authorised, differs greatly across Member States.

So clearly there is work for us to do here.

That is why, the Pharmaceutical Strategy for Europe will address unmet medical needs through a reform of the EU rules on medicines for rare diseases.

Building on what works and changing what doesn’t: that’s how we make progress.

Making progress on caring for patients with rare diseases happens when we pool resources. Research into rare diseases is clearly an area where it has paid off for us to work together at EU level, along with public funders, researchers, clinicians, patients and industry.

We’ve come a long way since launching E-Rare-1 with 10 partners from 8 countries in 2006. We now have the European Joint Programme on Rare Diseases – a large co-funded activity under Horizon 2020 with around 140 partners from 35 countries.

We’ve also been involved in putting rare disease research on the global health agenda.

I’m glad to say patients are deeply involved in shaping this research ecosystem, for example with EURORDIS involved in European and international research projects.

Meanwhile, ERN research activities receive funding to further their unique research opportunities for members and their patients.

Looking ahead, the multi-stakeholder European Partnership on Rare Diseases will be launched in 2024 under the Horizon Europe programme. It will give a boost to the rare disease research benefitting rare disease patients.

Dear Friends,

Access to knowledge, diagnosis and treatment for rare disease patients will remain central to EU policy.

Today, our main focus is on full implementation of current EU actions on rare diseases and the upcoming reform of EU rules. I am convinced that by working together we will continue to deliver tangible benefits to patients across the EU.

Alone we are rare, together we are strong.

The exemplary support you’ve shown to people with rare diseases fleeing Ukraine is clear proof that your community’s motto, is more than just words.

I wish you very productive discussions today.

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