Pharmacoeconomic Analysis of rFVIIa Versus PCC in the Treatment of Minor-to-Moderate Bleeds in Hemophilia Patients with Inhibitors in China
Author(s)
Liu J1, Zhen R2, Feng J3, Li S3
1Centre for Health Management and Policy Research, School of Public Health, Cheeloo College of Medicine, Shandong University, Jinan, 37, China, 2Institute of Chinese Medical Sciences, University of Macau, Beijing, 11, China, 3Centre for Health Management and Policy Research, School of Public Health, Cheeloo College of Medicine, Shandong University, Jinan, China
Presentation Documents
OBJECTIVES : In China, treatment options for bleeding episodes in hemophilia patients with inhibitors most often involve recombinant factor VIIa (rFⅦa) or prothrombin-complex concentrate (PCC).The aim of this study was to compare the cost and effectiveness of two most commonly used treatment regimens : rFⅦa followed by PCC (rFⅦa - PCC) and PCC followed by rFⅦa (PCC - rFⅦa) for the treatment of minor-to-moderate bleeds in hemophilia patients with inhibitors. METHODS : An observational study was conducted in China from September 2020 to January 2021. We applied the online questionnaire collecting patients’ treatment information over the past year. The cost included direct medical cost, direct nonmedical cost and indirect cost. Clinical outcomes included the hemostatic efficiency and re-bleeding rate within 12 hours. RESULTS : Overall, 32 bleeds occurred in 26 hemophilia patients with inhibitors (12 adults; mean age=20.4±13.2 years; 25 patients with high response inhibitors; 21 patients with severe hemophilia, 4 patients with moderate hemophilia) were included. There were 13 bleeds episodes treated with rFⅦa - PCC and 19 bleeding episodes with PCC - rFⅦa. Median cost of bleed treated with rFⅦa - PCC and PCC - rFⅦa were CNY 38,935(direct medical cost, direct nonmedical cost and indirect cost were CNY 37620, 115 and 1200, respectively) and CNY 47,280 (direct medical cost, direct nonmedical cost and indirect cost were CNY 45620, 660 and 1000, respectively). The hemostasis efficiency of rFⅦa - PCC was 77%, and PCC - rFⅦa was 63%. The re-bleeding rate of rFⅦa - PCC (8%) was lower than PCC - rFⅦa (32%). CONCLUSIONS : The rFⅦa - PCC treatment regimen was cheaper than PCC - rFⅦa with higher hemostatic efficiency and lower re-bleeding rate within 12 hours. Therefore, rFⅦa - PCC was found to be dominant (increasing clinical outcome and decreasing costs) compared with PCC - rFⅦa.
Conference/Value in Health Info
2021-11, ISPOR Europe 2021, Copenhagen, Denmark
Value in Health, Volume 24, Issue 12, S2 (December 2021)
Code
POSC395
Topic
Clinical Outcomes, Economic Evaluation
Topic Subcategory
Clinician Reported Outcomes, Cost-comparison, Effectiveness, Utility, Benefit Analysis
Disease
Rare and Orphan Diseases