BACKGROUND: Hemophilia A is a bleeding disorder characterized by a defi ciencyin factor VIII (FVIII). Routine prophylaxis with FVIII products or emicizumab is therecommended approach to management for people with hemophilia A (PwHA).The burden of lifelong prophylaxis treatments to prevent bleeding events forPwHA is substantial, impacting both the patients and the healthcare system.The objective of this study is to estimate the relative cost-effectiveness of theseprophylactic therapies for PwHA, including the newly approved efanesoctocogalfa.
METHODS: A Markov model was developed with four FVIII-based health states(normal clotting function, mild hemophilia, moderate hemophilia, severehemophilia) and death to compare emicizumab, efanesoctocog alfa, standardhalf-life (SHL) and other extended half-life (EHL) FVIII products as prophylaxisfor PwHA in the United States (US). Advate and Eloctate data were used torepresent SHL and EHL, respectively. The model considered a cohort of 38 yearsold hemophilia A patients with average weight of 94.4 kg treated withprophylaxis. Probability of experiencing each health state was based on eachproduct’s pharmacokinetic data, where FVIII activity levels were used to determine disease severity. Other clinical inputs, including annualized bleedrates and adverse event risks, were estimated from clinical trial data. Wholesaleacquisition cost (WAC) of drugs and healthcare costs of bleed management andadverse events were included and were estimated from published literature.Utilities by disease severity were derived from published real-world health-related quality of life studies for hemophilia patients. Disutility of infusion wasobtained from the published literature. Costs (in 2023 US dollars), quality-adjusted life-year (QALY), and total bleeds were estimated over a lifetimehorizon. Cost-effectiveness was estimated as incremental cost per QALY gained.One-way and probabilistic sensitivity analyses were conducted.
RESULTS: Over a lifetime, emicizumab was estimated to be less costly (total costat $17.0 million for emicizumab vs $24.0 million, $19.3 million, and $18.2 millionfor efanesoctocog alfa, SHL, and EHL) and more effective (18.61 vs 18.58,16.91, and 17.33 QALY) compared with efanesoctocog alfa, SHL, and EHL FVIII,respectively. Although efanesoctocog led to fewer bleeds (26.58 vs 59.91 bleedsover a lifetime), PwHA on emicizumab were expected to incur fewer bleeds(59.91 vs 198.45, and 108.58 bleeds) compared with EHL and SHL. Results wererobust to one-way and probabilistic sensitivity analyses.
CONCLUSIONS: Findings in this cost-effectiveness analysis suggest thatemicizumab is less costly and more effective (i.e., dominant) over a lifetimecompared with available FVIII prophylaxis in pediatrics and adult PwHA in theUS.
