OBJECTIVES: To compare clinical outcomes and economic burden of prophylactic treatment with on-demand treatment in adult patients with moderate-to-severe haemophilia A in China.

METHODS: The analysis was conducted from a societal perspective over a one-year period. A disease burden model was developed to evaluate clinical outcomes and costs of prophylactic treatment versus on-demand treatment with recombinant factor VIII in 5 selected centres in China. Total bleeding events, hospitalizations for major surgeries, outpatient visits and productivity loss due to haemophilia were modelled and compared. Treatment patterns, treatment effectiveness, healthcare resource utilization, and direct medical costs were derived from a survey conducted in 5 centres across the country. Opinions from patient organization experts were used for inputs regarding indirect and societal costs. Scenario analyses were undertaken to assess the impact of an increased proportion of adults receiving prophylactic treatment.

RESULTS: The mean annual number of total bleeds was lower in patients receiving prophylaxis than those receiving on-demand treatment (4.4 vs. 27.0). The base-case analysis favoured prophylaxis over on-demand as it is associated with lower total costs per patient in one-year (￥430,652 CNY vs. ￥446,413 CNY). Prophylactic treatment saved ￥42,952 CNY in non-drug costs compared to on-demand treatment due to decreased bleeding and productivity loss, which offset more expensive prophylactic drug costs. When extrapolated to national level, the societal savings would be 531 million CNY per year. Compared with the base-case, assuming that the proportion of adult prophylaxis is increased by 10%, 20% and 30%, the projected incremental cost saving would be ￥12, ￥24 and ￥36 million CNY each year, respectively.

CONCLUSIONS: Prophylactic treatment for adult patients with haemophilia A was a cost-saving strategy compared with on-demand treatment in China from societal perspective and should be chosen as a preferred approach in clinical practice.