Cost and effectiveness of treatments for mild‐to‐moderate bleeding episodes in haemophilia patients with inhibitors in Korea

Abstract

First-line treatment for mild-to-moderate bleeding episodes in patients with haemophilia and inhibitors in Korea is currently activated prothrombin complex concentrate (aPCC) with recombinant activated factor VII (rFVIIa) as second-line therapy or as a last resort. The aim of this study was to estimate the cost and effectiveness of aPCC vs. rFVIIa for treating mild-to-moderate bleeds in inhibitor patients from the Korean reimbursement authorities' perspective. Clinical outcomes and resource utilization data (number of doses, average dose, number of outpatient visits, inpatient stays, ambulance transport and concomitant medications) were collected from an observational study involving four Korean paediatric haemophilia centres. Cost-effectiveness was modelled using a decision analysis approach and sensitivity analyses undertaken. rFVIIa was a more effective haemostatic therapy (87.1% efficacy in bleed resolution) than aPCC (64.0%). rFVIIa effected more rapid haemostasis, resolving bleeding in a mean of 6.6 h vs. 25.2 h for aPCC. Fewer rFVIIa doses were required per bleed vs. aPCC (means 1.7 and 2.3). Mean total direct medical costs from bleed initiation to cessation were estimated at Korean Won (KRW)12 460 thousand (US$12 311) for rFVIIa given as first-line therapy and KRW18 304 thousand (US$18 085) for aPCC given as first-line therapy. Sensitivity analyses confirmed the cost-effectiveness of rFVIIa vs. aPCC given as first-line therapy. In Korea, use of rFVIIa as first-line therapy for treatment of mild-to-moderate bleeding episodes in inhibitor patients is both clinically effective and cost-effective compared with initial aPCC treatment. rFVIIa should be considered as the first-line treatment for mild-to-moderate bleeding episodes in patients with haemophilia and inhibitors in Korea.