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EHA at the Orphan Drug Conference in Amsterdam

Together Towards Fair Access for Rare Disease Therapies

President Astri Arnesen attended the Orphan Drug Conference in Amsterdam to discuss how the new regulations on EU Joint Clinical Assessments (JCAs) can be a tool to ensure faster and equitable access to new therapies for Rare Diseases (RD).  

The new regulations open up opportunities for more efficient evaluation of new therapies for the payers. When the evaluation is carried out on the European level, instead of each country carrying out a separate one, it helps ensure the best experts are involved, as not every country has experts in each RD.  It also opens up opportunities to reduce inequalities in the evaluations throughout Europe.

The reality today is that after a therapy receives market approval, it may be reimbursed and reach patients in some European countries, while in other countries, reimbursement is not approved, and the therapy is not made available. For the RD community, this situation is a heartbreaking disaster.  

To reach the objectives intended with the Joint Clinical Assessments, there are some hurdles we need to overcome:

  • Implement good infrastructure to ensure the best experts and patient representatives are involved in the evaluations.
  • Develop new, better, and sustainable pricing models to ensure equitable access in all European countries.
  • Ensure that all EU member states respect the joint evaluation and avoid second rounds of national evaluations, as these would only cause a longer waiting time from market approval to access than we have today.  

 

Only by joining forces and working collaboratively to overcome these hurdles can we ensure that every rare disease patient across Europe has access to the therapies they need and deserve. 

– Article written by Jarelys López Delgado

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