Cost-Effectiveness of Eftrenonacog Alfa in Treatment of Severe Haemophilia B Patients in Turkey

OBJECTIVES: Haemophilia B is a rare genetic disease caused by a deficiency of clotting factor IX (FIX). Recombinant factor IX is the standard of care and prophylactic treatment for severe bleeding phenotypes is recommended in recent years. Eftrenonacog alfa is an extended half-life recombinant factor IX product that uses Fc fusion technology (rFIXFc) for half-life extension. The objective of this study is to assess the cost-effectiveness of rFIXFc in prophylactic treatment of severe haemophilia B patients in comparison to recombinant FIX (rFIX) treatment from the Turkish healthcare payer perspective.

METHODS: A cost-effectiveness model was developed to capture the long-term costs and outcomes of treatment of severe haemophilia B with rFIXFc and rFIX. Effectiveness data for rFIXFc were obtained from the B-LONG, Kids B-LONG and B-YOND clinical trials. An indirect comparison was made to compare the effectiveness data of rFIXFc and rFIX. Pettersson Score was used as the primary outcome. The cost components in the model were factor acquisition costs and cost of resolving bleeding episodes. The time horizon of the model was 70 years and all outcomes and costs were discounted by 3%.

RESULTS: The final Pettersson score was 15.50 for rFIXFc and 18.50 for rFIX indicating better outcomes for rFIXFc. rFIXFc yielded 27.14 QALYs whereas the QALY for rFIX was 25.90 (incremental QALY: 1.25). The total cost of prophylaxis treatment was 62,189,198 TRY for rFIXFc and 100,442,102 TRY for rFIX. The cost-effectiveness analysis results have shown the dominance of rFIXFc over rFIX in treatment of severe haemophilia B patients. In other words, rFIXFc achieved better outcomes with lower costs. The sensitivity analysis results have indicated that the findings were robust.

CONCLUSIONS: rFIXFc is a cost-effective option in prophylaxis treatment of severe haemophilia B patients in Turkey.