Vellopoulou K, Georgilis A, Byrne A, Gray E, De Meyer N, Kourlaba G. Value in Health. 2019;22:S844.

Objectives: To estimate the impact on the Greek public payer budget from the use of damoctocog alfa pegol (DaPeg), an extended half-life recombinant factor VIII, for prophylactic and on-demand (OD)-treatment of patients ≥12 years with severe haemophilia A (HemA) having received previous treatment in Greece.

Methods: A 5-year budget impact model (BIM) was adapted with local data. The BIM compares a market scenario where a proportion of HemA patients receives DaPeg to an alternative market scenario where DaPeg is not available and patients are treated with available comparators, namely efmoroctocog-alfa, turoctocog-alfa, moroctocog-alfa and octocog-alfa (rAHF-PFM, BAY 81-8973). Epidemiologic data were derived by combining data from three Greek hemophilia centers and the World Hemophilia Federation (2017). Assuming equivalent efficacy (bleeding rates) across all comparators, clinical outcomes were not modelled. Current and future market shares for prophylaxis and OD-treatment were provided by the manufacturer. Only treatment costs were considered for prophylaxis and OD-treatment (€, 2019) with the doses obtained from the SPC and clinical trials, respectively. The difference between the total costs in each market scenario was the net budget impact.

Results: For a total of 231 patients with severe HemA, the introduction of DaPeg with a market share ranging from 5% to 27% in prophylaxis and 3% to 9% in OD-treatment from year 1 to 5, respectively, results in cost savings compared to the world without DaPeg. The budget saving magnitude increases with each successive year of the model horizon, driven by the lower annual per-patient costs of DaPeg. The cost savings increase from €99,510 in year 1 to €833,567 in year 5. The cumulative cost-savings over the modelled period exceed €2.5million.

Conclusions: The introduction of DaPeg into the Greek market of severe HemA results in a negative budget impact, generating cost savings for the public payer in the first 5 years of its use.