The long-term clinical benefits and economic costs associated with increased use of prophylaxis among patients with haemophilia A in China: Population-based predictions from 2018 to 2033

Aim To predict the long-term benefits and economic costs of the improvements in haemophilia care in China demonstrated by increasing use of prophylaxis, compared with the current status. Methods City-level predictions from 2018 to 2033 were conducted for five representative cities in China. The long-term clinical and economic outcomes in the scenario where haemophilia care has significantly improved and the existing scenario of haemophilia care were calculated and compared. The model input data were obtained from local records, expert interviews, published literature, and other sources. Outcome measures including number of bleeds and joint bleeds, number of target joints, disability rate, direct and indirect costs were calculated at the patient and population levels. Results The long-term predictions for 2033 demonstrated significantly improved bleed control and joint outcomes due to increased use of prophylaxis. The total number of averted bleed events per patient ranged from 3.9 in Shenyang to 16.1 in Zhengzhou in 2033, and the population-level averted bleed events ranged from 1963 in Xiamen to 14,868 in Zhengzhou. The treatment improvement also leads to significant economic costs driven by increase in clotting factor costs (more than 90%). At the population level, the additional total costs were highest in Zhengzhou (CNY 177.4 million) and lowest in Shenyang (CNY 45.4 million), due to their different population sizes and various existing treatment regimens. The outpatient and hospitalization costs decreased, while the factor costs increased. Conclusion The long-term prophylaxis is associated with avoided bleed events and disabilities. The improved treatment regimens are also associated with a significant economic burden, driven by factor costs.