The Future of Rare Diseases in Europe and Czech Republic: What are the Solutions at EU and National Level?

20. Jun 2022 — Register Here

Between 6,000 and 8,000 rare diseases affect an estimated 30 million people in the EU. Nonetheless, rare diseases are diseases with a low prevalence. In the EU, any disease affecting fewer than 5 people in 10,000 is considered rare. The current climate poses both challenges for the healthcare systems and real opportunities for action both at the national and European levels.

In a little over twenty years, since 2000, the European Regulation on orphan medicinal products EC 141/2000 (the OMP Regulation) greatly contributed to the development of orphan medicines, with the approval of 200 drugs in at least one orphan indication, ultimately improving the lives of 6.3. million people living with rare diseases in Europe, their families, and carers. The Regulation is a positive example of how a regulatory framework can foster and drive innovation and investments to address unmet medical needs. However, there are still important steps to be taken to address the 95% of rare diseases without an authorised treatment. To address these challenges, a review of the OMP Regulation is expected by the end of the year.

The national dimension is also crucial to ensure orphan therapies reach patients. In Czech Republic a new orphan drug procedure to expedite access has been established, that builds on the current EU framework for orphan drugs. This new OMP way of evaluation is considering the social cost on top of health care payers perspective, and it allows to the representatives of patient organizations as well as the professional societies to be actively involved into the reimbursement process for orphan drugs.

JOIN US And get an insight into the ways the European framework on rare diseases paves the way forward and complements actions at national level. We will hold this debate with representatives of the Czech Republic that will hold the rotating presidency of
the Council from July 2022 to December 2022. Rare diseases are going to be on the Czech Presidency agenda and this meeting will give us the opportunity to discuss how the debate on fostering innovation and patient access to orphan drugs will advance in the coming six months.

Introductory remarks from EUCOPE.


  • Jakub Dvořáček Deputy Minister of Health, Czech Republic
  • Anna Arellanesova Chairperson, Rare Diseases Czech Republic
  • Prof. Pavla Doležalová Doctor, General University Hospital, Prague
  • Leona Perinova CEE Lead, Sobi

The debate will be followed by a Q&A with the audience.

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