Events & Meetings / News - June 24, 2022

Event Recap: ‘The Future of Rare Diseases in Europe & Czech Republic’ Webinar

The European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) hosted a virtual roundtable on the future of rare diseases in Europe and the Czech Republic on Monday, 20 June 2022.

With the Czech Republic holding the rotating presidency of the Council of the European Union from July 2022 to December 2022, the roundtable was a unique occasion to share insights on how the European framework on rare diseases complements actions being undertaken at the national level.

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 the 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 healthcare payers´ perspective, and it allows the representatives of patient organizations as well as the professional societies to be actively involved in the reimbursement process for orphan drugs.


  • Jakub Dvořáček, Deputy Minister of Health, Czech Republic
  • Anna Arellanesova, Chairperson, Rare Diseases Czech Republic
  • Pavla Doležalová, Doctor, General University Hospital, Prague
  • Leona Perinova, CEE Lead, Swedish Orphan Biovitrum
  • Oliver Sude, Deputy Secretary General, EUCOPE
  • Vittoria Carraro, Associate Director, Government Affairs, EUCOPE


  • Antoine Mialhe, Senior Managing Director, FTI Consulting

Re-watch the event. 

Review our Event Report – The Future of Rare Diseases in Europe and Czech Republic (20 June)