Early Real-World Experience with Emicizumab and Concomitant Factor VIII Replacement Products in Adult, Male People with Hemophilia A without Inhibitors
Author(s)
Cafuir L1, Estrin A2, Chen E3, Hinds D4, Prince P2, Thorburn J2, Mead H4, Kempton C1
1Emory School of Medicine, Atlanta, GA, USA, 2Aetion Inc., New York, NY, USA, 3BioMarin Pharmaceutical Inc., Oakland, CA, USA, 4BioMarin Pharmaceutical Inc., Novato, CA, USA
Presentation Documents
Objectives: Real-world studies of emicizumab use tend to describe the overall hemophilia population, with limited data focusing on the adult population without inhibitors. With the advent of novel therapeutics, such as gene therapy, understanding healthcare resource utilization (HCRU) and cost of emicizumab use in specific populations is needed. Methods: Adult males with hemophilia A (PwHA) and without inhibitors who initiated emicizumab were identified using IBM MarketScan claims data after 10/4/2018 (approval date for emicizumab in PwHA without inhibitors). Patients were indexed on their first emicizumab claim; and were required to be enrolled ≥180 days prior to and ≥90 days after index date with ≥90 days of continuous use. Patients were followed until emicizumab discontinuation, health plan disenrollment, or end of data (12/31/2020). HCRU and costs were reported overall and among patients with prior factor VIII (FVIII) prophylaxis. All-cause HCRU, including emicizumab and FVIII use, and annualized costs were assessed over both the induction period (month 1) and maintenance period (months 2+). Results: 71 patients who initiated emicizumab (FVIII prophylaxis subgroup: 52) were identified; the mean age was 35 (subgroup: 34) years with follow-up of 12.0 (subgroup: 11.1) months. At baseline, the annualized all-cause total cost was $532,948 (subgroup: $645,727). 68% (subgroup: 73%) had at least one FVIII fill over the follow-up period, with a mean of 0.37 (subgroup: 0.46) fills per month during induction, and 0.17 (sub-group: 0.21) during maintenance. Most patients had ≥1 outpatient encounter per month, however total HCRU spend was mostly driven by treatment cost. The annualized mean cost was $50,491 (subgroup: $61,512) for FVIII treatment, $777,171 (subgroup: $793,168) for emicizumab and FVIII treatment, and $808,617 (subgroup: $814,592) for mean all-cause total cost. Conclusion: This study highlights the continued burden of treatment and healthcare cost for PwHA without inhibitors despite advances in treatment options.
Conference/Value in Health Info
2022-05, ISPOR 2022, Washington, DC, USA
Value in Health, Volume 25, Issue 6, S1 (June 2022)
Code
EE128
Topic
Economic Evaluation
Disease
Biologics and Biosimilars