COST-EFFECTIVENESS OF FITUSIRAN PROPHYLAXIS VERSUS FACTOR VIII ON-DEMAND OR PROPHYLACTIC THERAPY FOR HEMOPHILIA A PATIENTS WITHOUT INHIBITORS
Author(s)
Mohammad T. Alashqar, PharmD1, Shoroq M. Altawalbeh, PharmD, PhD2, Milap C. Nahata, MS, PharmD3;
1The Ohio State University, Institute of Therapeutic Innovations and Outcomes, College of Pharmacy, Columbus, OH, OH, USA, 2Jordan University of Science and Technology, Irbid, Jordan, 3The Ohio State University, Institute of Therapeutic Innovations and Outcomes, College of Pharmacy, Columbus, OH, USA
1The Ohio State University, Institute of Therapeutic Innovations and Outcomes, College of Pharmacy, Columbus, OH, OH, USA, 2Jordan University of Science and Technology, Irbid, Jordan, 3The Ohio State University, Institute of Therapeutic Innovations and Outcomes, College of Pharmacy, Columbus, OH, USA
OBJECTIVES: To evaluate the cost-effectiveness of fitusiran prophylaxis compared with FVIII prophylaxis and on-demand FVIII therapy in patients with severe hemophilia A without inhibitors from a US healthcare payer perspective.
METHODS: A lifetime state-transition (Markov) model was developed to simulate outcomes for males with severe hemophilia A initiating treatment at age 12 years. The model included three health states: no bleed, bleed, and death, with a 2-week cycle length to accurately capture bleeding dynamics. Clinical efficacy inputs for fitusiran and comparator strategies were derived from the ATLAS clinical trial program and published literature. Bleeding probabilities were based on treated annualized bleeding rates. Costs reflected drug acquisition, administration, and bleed-related medical care (2025 US $). Health utilities were sourced from EQ-5D data in hemophilia populations. Outcomes included lifetime costs, quality-adjusted life years (QALYs), incremental cost-effectiveness ratios (ICERs), and number of bleeds avoided. Deterministic and probabilistic sensitivity analyses were performed.
RESULTS: In the base-case cost-effectiveness analysis, fitusiran 50 mg every 2 months was the least costly strategy (nearly US $28 million) and the most effective, yielding 23.3 QALYs, and therefore dominated both on-demand FVIII therapy and FVIII prophylaxis, which were associated with higher costs and fewer QALYs. Over a lifetime horizon, fitusiran prophylaxis prevented 746 and 144 bleeding events compared with on-demand FVIII therapy and FVIII prophylaxis, respectively. In one-way sensitivity analyses, fitusiran prophylaxis remained cost-effective across all tested parameters, with loss of cost-effectiveness observed only when the annual fitusiran dose exceeded 366 mg. Probabilistic sensitivity analysis further showed that fitusiran was the preferred strategy in more than 80% of simulations across willingness-to-pay threshold of up to US $200,000 per QALY.
CONCLUSIONS: From a US payer perspective, fitusiran prophylaxis was a cost-effective and cost-saving strategy for hemophilia A without inhibitors, providing superior health outcomes at lower overall cost compared with on-demand and prophylactic FVIII therapy.
METHODS: A lifetime state-transition (Markov) model was developed to simulate outcomes for males with severe hemophilia A initiating treatment at age 12 years. The model included three health states: no bleed, bleed, and death, with a 2-week cycle length to accurately capture bleeding dynamics. Clinical efficacy inputs for fitusiran and comparator strategies were derived from the ATLAS clinical trial program and published literature. Bleeding probabilities were based on treated annualized bleeding rates. Costs reflected drug acquisition, administration, and bleed-related medical care (2025 US $). Health utilities were sourced from EQ-5D data in hemophilia populations. Outcomes included lifetime costs, quality-adjusted life years (QALYs), incremental cost-effectiveness ratios (ICERs), and number of bleeds avoided. Deterministic and probabilistic sensitivity analyses were performed.
RESULTS: In the base-case cost-effectiveness analysis, fitusiran 50 mg every 2 months was the least costly strategy (nearly US $28 million) and the most effective, yielding 23.3 QALYs, and therefore dominated both on-demand FVIII therapy and FVIII prophylaxis, which were associated with higher costs and fewer QALYs. Over a lifetime horizon, fitusiran prophylaxis prevented 746 and 144 bleeding events compared with on-demand FVIII therapy and FVIII prophylaxis, respectively. In one-way sensitivity analyses, fitusiran prophylaxis remained cost-effective across all tested parameters, with loss of cost-effectiveness observed only when the annual fitusiran dose exceeded 366 mg. Probabilistic sensitivity analysis further showed that fitusiran was the preferred strategy in more than 80% of simulations across willingness-to-pay threshold of up to US $200,000 per QALY.
CONCLUSIONS: From a US payer perspective, fitusiran prophylaxis was a cost-effective and cost-saving strategy for hemophilia A without inhibitors, providing superior health outcomes at lower overall cost compared with on-demand and prophylactic FVIII therapy.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
EE490
Topic
Economic Evaluation
Disease
SDC: Rare & Orphan Diseases, SDC: Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)