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

Paolo Angelo Cortesi; Giancarlo Castaman; Gianluca Trifirò; Simona Serao Creazzola; Giovanni Improta; Giampiero Mazzaglia; Angelo Claudio Molinari; Lorenzo Giovanni Mantovani

doi:10.1055/s-0039-3401822

Thrombosis and Haemostasis, Table of Contents

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

¹Research Centre on Public Health (CESP), University of Milano-Bicocca, Monza, Italy

,

Giancarlo Castaman

²Center for Bleeding Disorders and Coagulation, Careggi University Hospital, Florence, Italy

,

Gianluca Trifirò

³Department of Biomedical and Dental Sciences and Morpho-functional Imaging, University of Messina, Messina, Italy

,

Simona Serao Creazzola

⁴UOC Farmaceutica Convenzionata e Territoriale, ASL Napoli 1 Centro, Naples, Italy

,

Giovanni Improta

⁵Department of Public Health, University of Naples “Federico II,” Naples, Italy

,

Giampiero Mazzaglia

¹Research Centre on Public Health (CESP), University of Milano-Bicocca, Monza, Italy

,

Angelo Claudio Molinari

⁶Regional Reference Center for Hemorrhagic Diseases, Giannina Gaslini Institute, Genova, Italy

,

Lorenzo Giovanni Mantovani

¹Research Centre on Public Health (CESP), University of Milano-Bicocca, Monza, Italy

› Author Affiliations

Abstract

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.

Keywords

haemophilia A - inhibitors - prophylaxis - cost-effectiveness - budget impact

Full Text

References

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Supplementary Material