Background and Importance Haemophilia type A is a hereditary bleeding disorder linked to a deficiency in FVIII, treated by intravenous administration of FVIII. Emicizumab represents an alternative to FVIII concentrates, without anti-FVIII inhibitor, administered in a single subcutaneous dose.¹

Aim and Objectives The aim is to evaluate the use of emicizumab compared to the three biosimilars used for prophylaxis in haemophilia type A, in order to ensure good care with a good quality of life.

Material and Methods The analysis of the cost of using emicizumab compared to the three biosimilars we have in the hospital, namely plasma and recombinant FVIII (Moroctocog-alfa and Octocog) by calculating the direct, indirect and intangible costs, to assess the advantages and consequences of emicizumab use.

Results According to the cost minimisation analysis, we found a total annual cost for FVIII €125,293.7, Octocog €252,183.7, Moroctocog-alfa €2,753,70.6 with a significant intangible cost because the frequent trip to the hospital makes the patient tired and increases the non-medical cost and the indirect cost, with the possibility in 30% of patients of developing anti-FVIII inhibitors and therefore the administration of high dose plasma FVIII of 6000-9000 IU three times a week with an annual cost of €366,565.8.

On the other hand, emicizumab is indicated even for patients with an anti-FVIII inhibitor whose annual cost is €233,402.9, with a gain of €136,484.23, in addition to a good quality of life. We deduce that plasma FVIII is useful for patients without an inhibitor and emicizumab should be reserved for haemophiliacs with an anti-FVIII inhibitor.

Conclusion and Relevance Our cost evaluation study is a tool for decision support and reduction of uncertainty between four drugs, which makes it possible to adapt purchases according to the needs expressed for an optimal allocation of resources following the evolution of health expenditure.

References

1. Joel L. Moake , MD, Baylor College of Medicine, le manuel MSD 2022.

Conflict of Interest No conflict of interest