OBJECTIVES

Weekly prophylaxis with subcutaneous bispecific antibody (emicizumab) has showed higher efficacy in patients affected by haemophilia A with inhibitor (HAI), compared to patients treated on demand or on prophylaxis with bypassing agents (BPAs). However, no economic evaluations on emicizumab prophylaxis are available in Europe. This study assessed cost-effectiveness of emicizumab prophylaxis compared to BPAs prophylaxis and its possible budget impact.

METHODS

A decision analytic Markov model and a budget impact model were developed in Excel® to assess cost-effectiveness and budget impact of emicizumab prophylaxis in HAI patients. The models were populated using treatment efficacy from clinical trials and key clinical, cost and epidemiological data retrieved through an extensive literature review. The cost-effectiveness model estimated costs (€) and Quality-Adjusted LifeYears (QALY) using lifetime horizon; while, the budget impact model estimated costs (€) using 3 years time horizon. Both models used Italian National Health System (NHS) perspective. Results were presented as incremental cost-effectiveness ratios (ICERs) per QALY gained and as 3 years budget impact.

RESULTS

Emicizumab prophylaxis was found dominant compared to BPAs prophylaxis, with an improvement of 0.94 Quality adjusted life-years and a cost saving of €21.9 million per patient lifetime. Emicizumab prophylaxis reported 100% probability of being cost-effective at any threshold tested in the sensitivity analyses (€0-€250,000 per QALY gained). Furthermore, the availability of emicizumab prophylaxis in the next three years was associated to an overall budget reduction of more than €40 million. The sensitivity analysis confirmed the emicizumab positive impact on budget with a cost saving between 25 and 58 € millions in the next 3 years.

CONCLUSIONS

The clinically effective emicizumab prophylaxis can be considered a cost-saving treatment for HAI patients, with a significant positive impact on the healthcare budget. Based on our study results, emicizumab prophylaxis is a sustainable and convenient healthcare option for the Italian NHS.