A voluntary collaboration of EU member states, patients and industry plans to present recommendations this week for a framework to assess the value of Orphan drugs that could be used as the basis for pricing negotiations at the national level.
In principle, the plan seeks to reduce the health technology assessment burden on smaller EU member states and make the reimbursement process more predictable for companies, in turn increasing patient access to Orphan drugs.
While the concept of collaboration on health technology assessments (HTAs) may be attractive in theory, putting such a system into practice may prove difficult. The initiative's success depends on the continued participation of European member states, starting with a planned pilot trial to test the feasibility of the collaborative assessment process.
Regardless of the assessment framework's success, other recommendations from the EC working group would be worth implementing to improve access to Orphan drugs in Europe, according to industry representatives who spoke to BioCentury.
The collaboration, known as the