Thromb Haemost 2020; 120(02): 216-228
DOI: 10.1055/s-0039-3401822
Coagulation and Fibrinolysis
Georg Thieme Verlag KG Stuttgart · New York

Cost-Effectiveness and Budget Impact of Emicizumab Prophylaxis in Haemophilia A Patients with Inhibitors

Paolo Angelo Cortesi
1   Research Centre on Public Health (CESP), University of Milano-Bicocca, Monza, Italy
Giancarlo Castaman
2   Center for Bleeding Disorders and Coagulation, Careggi University Hospital, Florence, Italy
Gianluca Trifirò
3   Department of Biomedical and Dental Sciences and Morpho-functional Imaging, University of Messina, Messina, Italy
Simona Serao Creazzola
4   UOC Farmaceutica Convenzionata e Territoriale, ASL Napoli 1 Centro, Naples, Italy
Giovanni Improta
5   Department of Public Health, University of Naples “Federico II,” Naples, Italy
Giampiero Mazzaglia
1   Research Centre on Public Health (CESP), University of Milano-Bicocca, Monza, Italy
Angelo Claudio Molinari
6   Regional Reference Center for Hemorrhagic Diseases, Giannina Gaslini Institute, Genova, Italy
Lorenzo Giovanni Mantovani
1   Research Centre on Public Health (CESP), University of Milano-Bicocca, Monza, Italy
› Author Affiliations
Funding This analysis was funded by an unrestricted grant of Roche Italy spa.
Further Information

Publication History

28 May 2019

22 October 2019

Publication Date:
30 December 2019 (online)


Recent evidence demonstrated that weekly prophylaxis with subcutaneous bispecific antibody (emicizumab) has shown higher efficacy in adolescent and adults patients affected by haemophilia A (HA) with inhibitor, compared with patients treated on demand or on prophylaxis with bypassing agents (BPAs). However, no economic evaluations assessing the value and sustainability of emicizumab prophylaxis have been performed in Europe. This study assessed the cost-effectiveness of emicizumab prophylaxis compared with BPA prophylaxis and its possible budget impact from the Italian National Health Service (NHS) perspective. A Markov model and a budget impact model were developed to estimate the cost-effectiveness and budget impact of emicizumab prophylaxis in HA patients with inhibitors. The model was populated using treatment efficacy from clinical trials and key clinical, cost and epidemiological data retrieved through an extensive literature review. Compared with BPAs prophylaxis, emicizumab prophylaxis was found to be more effective (0.94 quality adjusted life-years) and cost saving (–€19.4/–€24.4 million per patient lifetime) in a cohort of 4-year-old patients with HA and inhibitors who failed immune tolerance induction. In the probabilistic sensitivity analysis, emicizumab prophylaxis had always 100% probability of being cost-effective at any threshold. Further, the use of emicizumab prophylaxis was associated to an overall budget reduction of €45.4 million in the next 3 years. In conclusion, the clinically effective emicizumab prophylaxis can be considered a cost-saving treatment for HA with inhibitor patients. Furthermore, emicizumab treatment is also associated to a significant reduction of the health care budget, making this new treatment a sustainable and convenient health care option for Italian NHS.


No Roche employees had sight of, contributed to or commented upon the manuscript draft ahead of submission. All authors contributed to the concept and objectives of the analysis.

Authors' Contributions

P.A.C. designed and developed the economic model and ran the analyses. The economic implications of the results were interpreted by P.A.C., G.T., S.C.S., I.G., M.G. and L.G.M., while the implications for clinical practice were interpreted by A.C.M. and G.C. The draft manuscript was prepared by P.A.C. and was reviewed, edited and approved by all authors.

Supplementary Material