Abstract
BACKGROUND: Treatment for haemophilia A has expanded from plasma-derived factor VIII (pdFVIII) and standard half-life (SHL) recombinant FVIII (rFVIII) to extended half-life (EHL) rFVIII and non-factor products that mimic FVIII activity. OBJECTIVE: To determine amounts of clotting factor concentrates (CFCs) and emicizumab dispensed and associated healthcare expenditures in Japanese patients with haemophilia A. METHODS: This retrospective, non-interventional, observational study analysed data from 2016 to 2020 from a large-scale, hospital-based administrative database. Patients had haemophilia A without inhibitors and ≥ 2 prescriptions of the same CFC or emicizumab. RESULTS: In total, 974 patients with haemophilia A (median age, 30.0 years; median follow-up, 3.7 years) were included. Outpatient use of EHL rFVIII and emicizumab increased, although pdFVIII/SHL rFVIII were still used over the study. Median annual total healthcare expenditures/patient increased from ¥9,200,230 in 2016 to ¥19,748,221 in 2020. Overall, the median annual drug expenditure/patient increased from ¥8,723,120 in 2016 to ¥18,051,689 in 2020. Drug expenditure was highest with emicizumab, with an increase in median annual expenditure/patient from 2018 (n = 4, ¥26,030,206) to 2020 (n = 107, ¥45,430,408). Overall, 233 patients (23.9%) switched from an SHL to an EHL product. Although amounts of FVIII prescribed increased in the 3 months after switching, overall, there was no noticeable difference before and after switching. Median total healthcare and FVIII product expenditures increased following switching. CONCLUSIONS: Prescribing of EHL products increased over the study and healthcare expenditures increased for patients who switched from SHL to EHL rFVIII products.