OBJECTIVES: To assess the cost-effectiveness of different recombinant factor VIII (FVIII) for prophylactic treatment of previously untreated patients (PUPs) with severe hemophilia A from the perspective of Chinese healthcare system.

METHODS: A cost-effectiveness model was constructed using a Markov model to simulate the risk of developing FVIII inhibitors, the risk of bleeding, annualized bleeding rate (ABR), and the risk of developing bleeding related complications in PUPs with severe hemophilia A who were under lifetime prophylactic treatment with antihemophilic factor (recombinant) plasma/albumin-free method (rAHF-PFM) or BAY 81-8973. Data used in this model were from systematic literature review, clinical expert survey and public sources. The model was used to perform base case analysis, one-way sensitivity analysis and probabilistic sensitivity analysis (PSA) with discounting for both simulated health benefits and direct medical costs at 5% per year.

RESULTS: Base case analysis estimated that prophylactic treatment with rAHF-PFM could extend total survival time by 0.055 years (17.965 years vs. 17.910 years), increased quality-adjusted life years (QALYs) by 0.779 QALY (9.290 QALY vs. 8.511 QALY), and saved lifetime direct medical costs by CNY4,578,403 (CNY12,060,222 vs. CNY16,638,625) compared to BAY 81-8973 in PUPs. One-way sensitivity analysis suggested that the result was mainly driven by ABR of prophylactic treatment with recombinant FVIII and the utility of patients without bleeding. PSA with 5,000 Monte Carlo simulations under the overall uncertainty of model inputs with 95% confidence interval estimated that rAHF-PFM had a 97.5% chance to be a superior treatment (less costly and more QALYs than BAY 81-8973) in PUPs with severe hemophilia A.

CONCLUSIONS: Based on the best available evidence, rAHF-PFM is likely to be economically superior compared to BAY 81-8973 in PUPs with severe hemophilia A for lifetime prophylactic treatment in China.