ESPEROCT (turoctocog alfa pégol)
Reason for request
Unfavourable opinion for reimbursement in the treatment and prophylaxis of bleeding in patients 12 years and above with haemophilia A (congenital factor VIII deficiency).
Role in the care pathway?
The objective of haemophilia A treatment is to achieve early control of and/or to prevent bleeding episodes and their short- and long-term complications, particularly haemophilic arthropathy.
Treatments may be managed using two treatment methods: treatment of bleeding episodes on demand, or continuous or intermittent prophylaxis and/or before a situation promoting bleeding, such as a surgical procedure.
Long-term prophylaxis is the standard treatment in children with severe haemophilia. There is no consensus as to the role of prophylaxis in adults.
Until recently, the only first-line treatment for haemophilia A in the absence of inhibitors was replacement therapy based on the intravenous administration of recombinant or plasma-derived clotting factor VIII concentrates. For the long-term prophylaxis of patients with severe haemophilia A only, there is now an alternative therapy: emicizumab (HEMLIBRA), a bispecific humanised monoclonal antibody that imitates the clotting function of FVIII and which is administered subcutaneously.
Role of the medicinal product in the care pathway
- the demonstration of the efficacy of ESPEROCT (turoctocog alfa pegol) in the treatment and prevention of bleeding in cases of severe haemophilia A in a non-comparative study,
- the lack of data demonstrating a benefit, particularly in terms of efficacy or quality of life, compared to the available alternatives,
- the current state of knowledge, which cannot rule out a risk of the onset of detrimental clinical effects associated with potential PEG accumulation, particularly in the choroid plexus, after numerous years of treatment, a potential risk mentioned in the RMP, in a context in which available alternatives do not present this risk,
- and in view of the numerous therapeutic alternatives available, both for the treatment of bleeding and for long-term prophylaxis, which contribute to coverage of the medical need,
the Committee considers that ESPEROCT (turoctocog alfa pegol) has no role in the treatment and prophylaxis of bleeding in patients 12 years and above with haemophilia A.
The Committee deems that the clinical benefit of ESPEROCT (turoctocog alfa pegol) is insufficient to justify public funding cover in the MA indication in view of the available alternatives.
Clinical Added Value