Evaluation of the Societal Impact of Emicizumab in France Since Its Market Authorization: Budget Impact From 2018 to 2024
Author(s)
Henri Leleu, PhD, MD1, Laurie Fraticelli, PhD2, Gwladys Touvron, Pharm.D.3, Roula Ajjouri, MBA4, Valérie Chamouard, Pharm.D.5.
1public health expertise, Paris, France, 2Health, Systemic, Process, UR 4129 Research Unit, University Claude Bernard Lyon 1, University of Lyon, Lyon, France, 3Roche France, Boulogne billancourt, France, 4ROCHE, Boulogne billancourt, France, 5Haemophilia Treatment Centre and French Reference Centre on Haemophilia, Louis Pradel Hospital, Hospices Civils de Lyon, Bron, France.
1public health expertise, Paris, France, 2Health, Systemic, Process, UR 4129 Research Unit, University Claude Bernard Lyon 1, University of Lyon, Lyon, France, 3Roche France, Boulogne billancourt, France, 4ROCHE, Boulogne billancourt, France, 5Haemophilia Treatment Centre and French Reference Centre on Haemophilia, Louis Pradel Hospital, Hospices Civils de Lyon, Bron, France.
OBJECTIVES: Emicizumab is a prophylactic treatment indicated for the prevention of bleeding in hemophilia A patients with severe or moderate disease with a severe bleeding phenotype. The HAVEN clinical studies have demonstrated that emicizumab significantly reduces bleeding episodes compared to no prophylaxis. Available in France since 2018, the societal impact of emicizumab, beyond its clinical benefits, remains underexplored. This study evaluates the societal impact of emicizumab's introduction in France from 2018 to 2024.
METHODS: A retrospective budget impact model was constructed based on bleeding data from the HAVEN 1 and HAVEN 3 trials, combined with real-world data on bleeding management and the share of patients treated with emicizumab based on National Health Insurance data. The current scenario with emicizumab was compared to a theoretical scenario without it. Outcomes included reductions in hospitalizations associated with bleeding and complications, including factor VIII use, savings in direct costs, and indirect costs. The analysis was conducted from a French societal perspective.
RESULTS: The model estimates that between 2018 and 2024, the use of emicizumab would have avoided approximately 10,000 hospitalization days, generating an estimated saving of 6 million euros in hospital expenditures. Including treatment costs related to bleeding, total savings for the French Health Insurance are estimated at 1.0 billion euros. For patients and caregivers, the reduction in hospital visits and the burden associated with the disease would have avoided the loss of approximately 245,000 workdays, representing 35 million euros in productivity loss for society.
CONCLUSIONS: The introduction of emicizumab in France has likely generated a significant positive societal impact, including substantial savings, freeing up hospital resources, and reducing the amount of time associated with disease management for patients and caregivers. These results underscore the importance of adopting a societal perspective in evaluating innovative treatments for chronic diseases such as hemophilia A.
METHODS: A retrospective budget impact model was constructed based on bleeding data from the HAVEN 1 and HAVEN 3 trials, combined with real-world data on bleeding management and the share of patients treated with emicizumab based on National Health Insurance data. The current scenario with emicizumab was compared to a theoretical scenario without it. Outcomes included reductions in hospitalizations associated with bleeding and complications, including factor VIII use, savings in direct costs, and indirect costs. The analysis was conducted from a French societal perspective.
RESULTS: The model estimates that between 2018 and 2024, the use of emicizumab would have avoided approximately 10,000 hospitalization days, generating an estimated saving of 6 million euros in hospital expenditures. Including treatment costs related to bleeding, total savings for the French Health Insurance are estimated at 1.0 billion euros. For patients and caregivers, the reduction in hospital visits and the burden associated with the disease would have avoided the loss of approximately 245,000 workdays, representing 35 million euros in productivity loss for society.
CONCLUSIONS: The introduction of emicizumab in France has likely generated a significant positive societal impact, including substantial savings, freeing up hospital resources, and reducing the amount of time associated with disease management for patients and caregivers. These results underscore the importance of adopting a societal perspective in evaluating innovative treatments for chronic diseases such as hemophilia A.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE448
Topic
Economic Evaluation, Epidemiology & Public Health
Topic Subcategory
Budget Impact Analysis
Disease
Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)