OBJECTIVES : Italy has recently approved a bispecific antibody (emicizumab) administered subcutaneously once a week, every two weeks or monthly, for the treatment of severe Haemophilia A (HA) without inhibitors in Italy. The treatment efficacy and low treatment burden could allow clinicians to improve patient’s management and outcome, however economic assessment of emicizumab in non-inhibitors patients are still lacking. Aim of the study was assessing the cost and consequences of emicizumab prophylaxis compare to extended half-life (HLF) and standard half-life (SHL) rFVIII prophylaxis in severe HA patients without inhibitors.

METHODS : A markov model was developed to assess the cost-consequence of four treatments: emicizumab, efmoroctocog-alfa (EHL), lonoctocog-alfa (SHL), and octocog-alfa (SHL) prophylaxis. The model estimated the costs and consequence (e.g. bleeding and number of infusion) of 1 years old patient’s lifetime treatment using Italian National health system perspective. The model was populated using treatment efficacy from clinical trials and key clinical and costs retrieved through an extensive literature review.

RESULTS : A life-time prophylaxis reported an overall cost per patient of 6.686€ mln using emicizumab, 6.207€ mln efmoroctocog-alfa, 5.317€ mln lonoctocog-alfa and 5.480€ mln octocog-alfa. All three rFVIII prophylaxis reported a lower overall costs with a lifetime costs difference between 0.480€ mln (efmoroctocog-alfa) and 1.370€ mln (lonoctocog-alfa). However, emicizumab prophylaxis was associated to a lower number of bleeding (-265 bleeds) and arthroplasty (-1.78 surgeries) per patient compare to all three rFVIII prophylaxis. Further, emicizumab prophylaxis required -4,701, -7,896 and -9,180 infusions per patient lifetime compare to efmoroctocog-alfa, lonoctocog-alfa, and octocog-alfa, respectively.

CONCLUSIONS : Emicizumab prophylaxis for severe Haemophilia A patients without inhibitors is associated to a lifetime increase costs between 7-20%, bleeding reduction of 65%, and number of infusions reduction between 63-77%. This data could help clinicians and budget holder to better understand the impact of emicizumab prophylaxis in haemophilia A management.