The OD Expert Group launches the Report on an EU HTA for Rare Disease

Today, rare disease patients across EU member states experience delayed or lack of access to approved Orphan Medicinal Products (OMPs). On June 29, during our event, the OD Expert Group officially launched its reports on HTA. Through a series of workshops, consultations, and as a result of a collaborative effort, the OD Expert Group has developed through the years its proposals for a European HTA fit for Rare Diseases, capable of addressing the key challenges linked to medicinal products for rare and ultra-rare conditions:

  1.  Part of the access issue for OMPs resides in the fact that HTA processes across EU member states are broadly not adapted to handle the uncertainty around the clinical effectiveness of OMPs that can be demonstrated based on the available clinical evidence at the point in time of the assessment. The Expert Group worked on developing a framework to overcome present barriers and improve access to treatments for rare conditions, focusing on clinical evidence in joint clinical assessments.
  2. To make well-informed decisions in joint clinical assessments, HTA bodies rely on input from experts from outside their own organizations with subject matter expertise or experience. In rare diseases in general, and increasingly so as we turn to even less prevalent rare diseases, relevant experts are likely to have engagements that constitute an interest in the assessment. Stakeholders involvement is key in the process but specificities rise in such a small population when dealing with rare disease. We recommend that interests and even conflicts of interests are handled through transparency and declaration of  interests rather than exclusion when assessing OMP.

Read our Report: AN EU HTA FIT FOR RARE DISEASES – Part 1: Clinical evidence in joint clinical assessments

Read our Report: AN EU HTA FIT FOR RARE DISEASES – Part 2: Stakeholder involvement in joint clinical assessments


Posted on 10. July 2023