OBJECTIVES: The standard therapy for patients with hemophilia A is the prophylactic treatment with replacement factor VIII (FVIII), with a consumption variability depending on the patient's weight and the therapy personalization. In 2018, emicizumab become available in Italy, becoming the first therapy to offer subcutaneous prophylaxis with less frequent administration (QW, Q2W or Q4W). The aim of this analysis was to compare the prophylactic treatment with FVIII (short and long acting) in terms of consumption and direct costs versus the emicizumab theoretical cost in patients with hemophilia A without FVIII inhibitors.

METHODS: A retrospective analysis was performed using administrative database from Local Health Units (~9 million inhabitants) including male patients with hemophilia A who started the treatment with emicizumab between January 2018 to September 2022. FVIII consumption was assessed over 12 months before the first prescription of emicizumab. The prophylaxis regimen was identified using a literature-validated algorithm.

RESULTS: A total of 72 patients treated with emicizumab were identified; 32 patients (mean age 28.2±21.2 years, N=12 <13 years, estimated mean weight 58.7±21.2 kg) were previously on prophylaxis with FVIII short-acting (N=21, 7 of which <13 years) or long-acting (N=11, 5 of which <13 years). In the year before starting emicizumab, patients had an average annual FVIII consumption of 382,398±339,979 IU with a total average annual cost of €262.678 (VAT included) per patient. In the same period, patients had a mean number of 5.5±7.4 prescriptions for drugs not related to hemophilia and an average number of 4.2±5.1 prescriptions for specialist tests/visits.

CONCLUSIONS: This preliminary analysis shows that the mean FVIII prophylaxis consumption (382,398IU) is higher than the previous mean consumption from Italian data. Considering the annual maintenance cost of emicizumab, estimated on an average patient of the same weight, the switch to emicizumab could result in a theoretical cost reduction up to approximately 20% per patient.