OBJECTIVES: The objective of this study is to explore the budget impact of efmoroctocog alfa (a recombinant factor VIII Fc fusion protein, herein rFVIIIFc) prophylaxis treatment in severe haemophilia A patients in comparison to standard recombinant FVIII (rFVIII) in Turkey.

METHODS: A budget impact model based on the clinical effectiveness data for rFVIIIFc and rFVIII was designed. Number of patients with severe haemophilia A, their age distribution and number of patients in prophylaxis treatment were estimated from published epidemiological data. Annual bleeding rates and number of units needed to treat a bleeding episode were taken from literature. The analysis was made from the Turkish healthcare payer perspective (SSI). Only reimbursement prices of products were used in the analysis.

RESULTS: Number of severe haemophilia A patients was estimated as 3,557 for 2022. This population was divided into <18 and >18 age groups and then was further divided into patients under prophylaxis and on-demand therapy. There were 1,110 patients in the <18 prophylaxis group and 1,627 patients in the >18 prophylaxis group. Annualized bleeding rates for each group were taken from the literature. Accordingly, there were 2,175 and 4,440 annual bleedings in the <18 age group for rFVIIIFc and rFVIII calculated respectively. The number of bleedings for >18 was 4,720 (rFVIIIFc) and 5,289 (rFVIII). Required dose for treatment of a bleeding for adults and children were taken from the literature (for adults 41.75 IU/kg, for children 57.99 IU/kg). Average weight for adults was taken as 74.1 for adults and 37.9 for children from a recent survey in Turkey. Based on this data, total annual cost saving by using rFVIIIFc was estimated as 16,296,201 TRY for <18 and 7,857,920 TRY for >18.

CONCLUSIONS: Use of rFVIIIFc in prophylaxis treatment of severe haemophilia A is a budget saving option in Turkey.