Efmoroctocog alfa (1) – Elocta®

Haemophilia patients requiring factor VIII replacement

• For the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital Factor VIII deficiency), the additional benefit compared with the appropriate comparator therapy is not proven.
• The additional benefit compared with the appropriate comparator therapy is therefore not proven.
• In the dossier, the pharmaceutical manufacturer identifies recombinant and human plasma-derived preparations as the appropriate comparator therapy.
• However, to demonstrate the additional benefit of Efmoroctocog alfa, only an unadjusted historical control for prophylactic treatment against recombinant blood coagulation factor VIII preparations is presented.
• No direct comparative studies against the appropriate comparator therapy are available.
• For the indirect historical control, a pool of eight studies in total has been identified.
• The pharmaceutical manufacturer has not provided any relevant data for the assessment of the additional benefit of Efmoroctocog alfa.
• The G-BA considers the results presented, taken as a whole, to be insufficient to allow the derivation of patient-relevant effects regarding the additional benefit of Efmoroctocog alfa compared with the appropriate comparator therapy.
• morbidity
  • In principle, all available results from the endpoint categories of mortality, morbidity, health-related quality of life and adverse events must be taken into account for the benefit assessment.

Courtesy translation only, please refer to the German original.