OBJECTIVES: Hemophilia A is an inherited, life-long bleeding disorder caused by the lack of clotting factor VIII. Recombinant VIII products are the current standard of care and the treatment approach has shifted from on-demand treatment to prophylaxis in recent years. The objective of this study is to explore the cost-effectiveness of efmoroctocog alfa (a recombinant factor VIII Fc fusion protein, herein rFVIIIFc) prophylaxis treatment in previously treated severe hemophilia A patients in comparison to standard recombinant FVIII (rFVIII).

METHODS: A Markov model was developed with annual bleeding rates as the primary outcome. Effectiveness data for rFVIIIFc were obtained from A-LONG and Kids A-LONG clinical studies. An indirect comparison was made with rFVIII. The analysis was made from the Turkish healthcare payer perspective (SSI). Only drug acquisition costs were included in the cost component of the model. One-way and probabilistic sensitivity analysis were made. All outcomes and costs were discounted by 3%. The ICER was calculated for both life years and QALYs. rFVIIIFc and rFVIII are marketed with 250, 500, 1000,1500, 2000 IUs. The analyses were made for all doses. rFVIIIFc is the only recombinant factor with 3000 IU so the comparison was made with the average price of 2000 and 1000 IUs of rFVIII products.

RESULTS: The life years gained were 29.15 and 29.03 for rFVIIIFc and rFVIII respectively (incremental life years: 0.12). The QALYs gained were 24.92 and 24.40 (incremental QALY: 0.52) for rFVIIIFc and rFVIII respectively. In all comparisons, rFVIIIFc yielded lower annual costs compared to rFVIII and dominated the results for both life years and QALYs. The sensitivity analyses results showed that the findings were robust.

CONCLUSIONS: rFVIIIFc prophylaxis treatment in previously treated severe hemophilia A patients in comparison to rFVIII is a cost-effective option in Turkey.