PB0199 - Budget impact of increasing rVIII-SingleChain use in patients in Italy with moderate and severe hemophilia A

Background: rVIII-SingleChain is a recombinant factor VIII (rFVIII), the safety and efficacy of which has been demonstrated in clinical trials and real-world studies in patients with hemophilia A.

Aims: To estimate the potential budget impact of increasing rVIII-SingleChain usage over a 3-year period in Italy.

Methods: Patients with moderate and severe hemophilia A receiving prophylaxis or episodic treatment were included. Two analyses were conducted. First, comparing two scenarios where rVIII-SingleChain share is constant (basecase) or increases over time by taking from other rFVIII products (alternative), considering the list prices of rFVIII products in Italy. Assumptions included the anticipated patient share estimates of rVIII-SingleChain and competitor products, while current list prices were kept constant (over 3-year period). For each scenario, the total cost of rFVIII products used for prophylaxis and in the treatment of bleeds was calculated. Mean product consumption and mean annual bleeding rate for rVIII-SingleChain, rFVIIIFc, octocog alfa and BAY 81-8973 were based on pooled real-world data from Italy and Germany. Consumption and bleeding rates for other products were derived from the product labels. Second, a sensitivity analysis considered the current regional product acquisition costs in the basecase and alternative scenarios, while all the other parameters remained the same.

Results: If the patient share of rVIII-SingleChain increases from 9% (basecase, constant over the three years) in first year to 30% in the third year in the alternative scenario, there is expected to be a decrease in total expenditure for rFVIII products of €2.9 million in year 1, €7.2 million in year 2 and €11.3 million in year 3. These findings remained consistent when regional acquisition costs were used instead of list prices.

Conclusion(s): Increasing utilization of rVIII-SingleChain in the treatment of patients with hemophilia A may lead to cost-savings as a result of reduced FVIII consumption.