PF331 COST‐EFFECTIVENESS OF EMICIZUMAB VERSUS BY‐PASSING AGENTS IN PATIENTS WITH HAEMOPHILIA A AND FVIII INHIBITORS IN FRANCE

Background: The development of an anti‐FVIII inhibitor is the most serious complication of haemophilia A. On February 23, 2018, Emicizumab obtained a European Marketing Authorization for the prevention of bleeding episodes in patients with haemophilia A who developed an anti‐FVIII inhibitor. In France, emicizumab is reimbursed by National Insurance since February 7 th 2019. As part of its inscription on the list of products reimbursed by French National Health Insurance, emicizumab has been evaluated by health authorities (HAS): The Transparency Committee granted a major added medical benefit (ASMR II), and a medico‐economic analysis was evaluated by the Health Economic and Public Health Committee. Aims: The purpose of this analysis is to evaluate the Incremental Cost‐Effectiveness Ratio (ICER) of emicizumab compared to the current management by Bypassing Agents (BPA) taking into account the final price that has just being published. Methods: The analysis is based on a Markov model with two health states (alive, dead) over a 5‐year time horizon. Emicizumab is compared with current BPA management (aPCC and rFVIIa) in prophylaxis or on‐demand, according to their real‐life use in the FranceCoag Network cohort (RFC). The ICER is expressed in euros per QALY (Quality‐Adjusted Life Year). Bleeding rates are derived from the HAVEN 1 clinical trial for emicizumab and BPA on‐demand, and an indirect comparison for BPA prophylaxis. Quality of life data are derived from the HAVEN 1 trial. The costs of treatment acquisition, adverse events and hospitalizations were taken into account. Deterministic and probabilistic sensitivity analyzes were performed to evaluate the uncertainty around the result. Results: Emicizumab prophylaxis is a dominant strategy, in children and adult patients: it is more effective and less costly than the current BPA strategy. Over 5 years, the mean total cost per patient was 2,3 M€ in patients treated with emicizumab, versus 2,7 M€ with BPA, associated to 3,3 and 2,4 QALYs respectively. Thus, emicizumab brings 0,9 additional QALYs per patients for a saving of almost 426 000€. Exploration of uncertainty does not alter these results. Summary/Conclusion: The health economic analysis shows the cost‐effectiveness of emicizumab versus BPA in France. The methodology used was accepted by the CEESP in its Opinion of October 9, 2018. The limitations of this analysis are mainly based on the lack of data to model the consequences of the reduction of bleeding on the long‐term evolution of arthropathy and disability.