OBJECTIVES: The development of an inhibitor is one of the most serious problem in patients with Hemophilia A. Promising therapies are becoming available to reduce bleeds rate and improve quality of life for patients.. Recently the European Medicines Agency (EMA) has granted a marketing authorization for emicizumab (Hemlibra^®), but no reimbursed price is still available. Based on consistent clinical benefit produced, this analysis aimed to estimate the potential cost-effectiveness of emicizumab in patients with Hemophilia A and inhibitor, under the Italian healthcare system, assuming different hypothetical price scenarios.

METHODS: A decision analytic Markov model was developed. The model considers three health states: Hemophilia with inhibitor, bleed and death and it used a lifetime horizon. Emicizumab was compared with bypassing agents prophylaxis (aPCC or rFVII). Clinical efficacy of evaluated therapies was extracted from clinical studies. Health utility values and cost data were retrieved from literature. Costs were expressed in €, 2018. The cost of emicizumab prophylaxis was assumed equal to aPCC prophylaxis, in the basecase scenario. An extensive sensitivity analysis was performed to assess the possible impact of emicizumab price range on the costs-effectiveness estimates.

RESULTS: In the basecase, emicizumab prophylaxis was more effective and cost-saving compared with both aPCC and rFVII prophylaxis, reporting 0.944 QALYs gain and a cost reduction of €1,207,450/patient. In the sensitivity analyses emicizumab prophylaxis was cost-effective (ICER=€100,000/QALY) up to a price of 219.7€/mg (+5% of basecase). While a 5% price reduction of emicizumab (199.6€/mg) reported a lifetime cost saving of €2,644,952/patient. Similar results was obtained when emicizumab was compared with rFVII.

CONCLUSIONS: Small change in emicizumab price has a high impact in cost-effectiveness results. A ±5% price variation can change the results from a cost-effective ICER=€100,000/QALY to a dominant scenario, with a cost reduction of €2 mln per patient.