OBJECTIVES: Emicizumab is a new therapeutic option for the routine prophylactic treatment of patients with Hemophilia A with inhibitors. Activated Prothrombin Complex Concentrate (aPCC) is currently the only bypassing agent approved in Portugal to be used in prophylaxis. The present study aims to compare the costs and clinical consequences of emicizumab vs. aPCC as prophylactic treatment of adults and pediatric patients from the National Health System (NHS) and societal perspectives.

METHODS: A Markov model was developed to project the expected annual bleeding rates and its impact in patient’s QoL and overall costs through a lifetime horizon. The relevant clinical outcomes of the weekly subcutaneous treatment with emicizumab were obtained from the HAVEN 1 trial. Indirect comparison based on literature review provided the clinical data for aPCC. Both treatments were assumed to equally decrease case severity, consequently reducing mortality. Direct costs were collected from official public data and consisted of medication acquisition, hospitalizations, adverse events and transportation. Indirect costs due to patient and caregiver absenteeism were included in the societal perspective. Population characterization was validated by Portuguese experts. A discount rate of 5% was applied to both costs and consequences and sensitivity analyses (deterministic and probabilistic) were performed.

RESULTS: Despite assuming no incremental gain on life years when compared to aPCC, the results show an increase of 3.85 QALYs with emicizumab. Considering 100% of adherence and life-long treatment in both arms, emicizumab results in a decrease of € 6.6 million over lifetime, which represents more than 50% of cost reduction. Emicizumab is cost-saving in 100% of tested scenarios.

CONCLUSIONS: Emicizumab demonstrated to be a dominant alternative in the prophylactic treatment of hemophilia A with factor VIII inhibitors comparatively to aPCC, which means it demonstrated to be more effective at a lower cost to NHS and society than current treatment in Portugal.