OBJECTIVES: Hemophilia A is a condition associated with increased risk of bleeding due to an inherited deficiency of factor VIII (FVIII). There are prophylactic non-inhibitor treatment, recombinant or plasma FVIII, and inhibitor treatment options, including FVIII inhibitor bypass activity (FEIBA). Emicizumab is approved for hemophilia A patients with and without inhibitors, although clinical trials included mainly inhibitor patients. The aim of this study was to understand cost of current and upcoming treatment options (including emicizumab) for patients with hemophilia A in Turkey.

METHODS: A Markov-based cost-consequences model was utilized to calculate cost of recombinant or plasma FVIII, inhibitor agents, and emicizumab for patients in Turkey. The model inputs were derived from published literature. The prophylaxis dosage was estimated using a combination of literature, product leaflet, and expert opinion. Base year for treatment cost of recombinant or plasma FVIII and healthcare service costs was 2020. Given the reimbursement price of emicizumab was not yet set by payers, an official mandatory reimbursement discount rate of 41% was used for analyses.

RESULTS: The annual per-patient treatment cost of hemophilia A including therapy, management of bleeding, inpatient and outpatient care, was estimated at 382,379 Turkish Lira (TL) and 2,109,730 TL, for non-inhibitor patients and inhibitor patients, respectively. The average annual treatment cost (non-inhibitor and inhibitor patients) was 459,937 TL. The annual per-patient cost of emicizumab treatment was 887,490 TL. If all inhibitor patients and 20% of non-inhibitor patients received emicizumab, the annual per-patient cost increased to 501,545 TL. The extent of cost increase was directly related to the proportion of non-inhibitor patients receiving emicizumab.

CONCLUSIONS: FVIII remains cheaper treatment option for non-inhibitor, hemophilia A patients in Turkey. Emicizumab can only yield reduced budget impact for inhibitor patients. If emicizumab receives broader reimbursement in Turkey, additional treatment costs for hemophilia A patients will be realized.