Prophylactic treatment with recombinant factor VIII (rFVIII) replacement products has led to improvements in quality of life and outcomes for people living with hemophilia A (HA). However, the cost-effectiveness of these treatments hasn’t been determined. A study published in PharmacoEconomics found that prophylactic rFVIII Fc-fusion protein (rFVIIIFc) may be cost-effective for people with severe HA.
Approximately 56.6% of people living with HA in the United States have severe disease. rFVIII use makes up the majority of the lifetime costs for patients with HA. The primary goals of treatment are to reduce bleeding episodes and preserve joint health.
The study performed a cost-effectiveness analysis (CEA) from a US healthcare payer perspective of rFVIII-based treatments for adult patients with severe HA without inhibitors.
The study compared extended half-life (EHL) rFVIIIFc (Eloctate®, Sanofi), pegylated rFVIII (PEG-rFVIII; Adynovate®, Takeda), and standard half-life (SHL) rFVIII (Advate®, Takeda, used to represent a range of SHL rFVIII products).
Outcomes measured were total costs and quality-adjusted life-years (QALYs). The authors evaluated the effect of a drug based on frequency of breakthrough bleeding events and joint health. The analysis considered product acquisition costs only using the wholesale acquisition cost.
Treatment with rFVIIIFc provided 27.922 QALYs compared with 27.454 with PEG-rFVIII and 27.071 with SHL rFVIII. Treatment with rFVIIIFc had a lower cost at $18.235 million compared to the other 2 products. The cost of SHL rFVIII was $18.285 million and the cost was $20.198 million with PEG-rFVIII.
Overall, this CEA found that rFVIIIFc prophylaxis provides more QALYs at a lower cost than competitor products.
QALYs were sensitive to joint health measures, and the study is limited by a lack of available data on joint health. By using the wholesale acquisition cost, the CEA may be limited by differences in the true acquisition cost based on confidential pricing agreements.
Disclosure: This research was supported by Sanofi. Please see original reference for a full list of disclosures.
Bullement A, Knowles ES, DasMahapatra P, Ali T, Preblick R. Cost-effectiveness analysis of rFVIIIFc versus contemporary rFVIII treatments for patients with severe hemophilia A without inhibitors in the United States. Pharmacoecon Open. Published July 15, 2021. doi:10.1007/s41669-021-00283-6