Economic Costs and Concomitant FVIII Consumption in Hemophilia Patients Treated with Emicizumab in the 1st Year − A US Experience

Analyzed Data

Data from men with hemophilia A without inhibitors who were switched to emicizumab were identified using IBM MarketScan for the analysis. To be included in the analysis, individuals had to have continuous health insurance coverage for more than 180 days before switching to emicizumab and at least 90 days after the switch, with records of more than 90 days of continuous emicizumab administration.

Participants were monitored until treatment interruption (temporary or permanent) or loss of data recording. Data were tracked on the use of emicizumab, concomitant FVIII administration, healthcare utilization, and treatment costs (HCRU). Each piece of information was separately evaluated in different periods − during emicizumab induction therapy, during maintenance therapy, and annually.

A total of 71 men treated with emicizumab were included, with an average age of 35 years and an average follow-up period of 12 months. The subgroup of patients previously receiving FVIII prophylaxis included 52 men with an average age of 34 years and an average follow-up period of 11.1 months.

Findings

At the start of emicizumab treatment, average total treatment costs were 532,949 US dollars (USD), and 645,727 USD in the subgroup with prior FVIII prophylaxis. After starting emicizumab treatment, HCRU per patient per month was higher during the emicizumab induction therapy period compared to the maintenance therapy period, with observed higher monthly FVIII prescriptions (pharmacy data) and related administrative activities in outpatient care (0.37 vs. 0.17), as well as higher visits without FVIII prescriptions (2.23 vs. 1.55) and emergency visits (0.06 vs. 0.03). Similar trends in monitored HCRU values were also seen in the subgroup with prior FVIII prophylaxis.

Drug costs for hemophilia represented 95% of all treatment-related costs. The annual average cost was 50,491 USD for concomitant FVIII treatment (61,512 USD in the subgroup with prior FVIII prophylaxis), and total costs for emicizumab and concomitantly administered FVIII in the 1st year of emicizumab treatment reached 777,171 USD (793,168 USD in the subgroup with prior FVIII prophylaxis).

Conclusion

This data illustrates the early experience shortly after the introduction of emicizumab in the treatment of patients with hemophilia A without inhibitors. The cited analysis may not be representative of all patients with hemophilia A without inhibitors treated with emicizumab. However, it highlights the ongoing therapeutic and financial burden in this patient group.

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Source: Cufuir L., Estrin A., Chen E. et al. Early real-world experience with emicizumab and concomitant factor VIII replacement products in adult males with hemophilia A without inhibitors. J Med Econ 2022 Jan-Dec; 25 (1): 984−992, doi: 10.1080/13696998.2022.2102779.