OBJECTIVES

: Hemophilia A (HA) is a bleeding disorder caused by a lack of blood clotting factor VIII. Development of inhibitors is one of most serious complications of the disease. Emicizumab offers advantage of reduced bleeding rates and lower injection frequency over currently available treatments (prophylactic and on-demand activated prothrombin complex concentrate (APCC) or recombinant activated FVII (rFVIIa) and immune therapy induction (ITI)). To estimate budgetary impact of Emicizumab on the Saudi MOH budget once adopted as a prophylactic therapy in pediatric HA patients with inhibitors.

METHODS

: A budget impact model was developed in Microsoft Excel^® over a 5-year time horizon from 2018-2022. The model assumes that all patients will be gradually switched from prophylactic and on-demand rFVIIa, prophylactic aPCC or ITI therapies to prophylactic Emicizumab. The impact was evaluated using two scenarios – ‘With Emicizumab’ and ‘Without Emicizumab’. The total costs included drug acquisition costs, drug preparation and administration costs, hemorrhages’ management costs and travel costs. The budget impact was estimated by calculating the difference in costs between the two scenarios.

RESULTS

: Switching 100% of patients from prophylactic and on-demand rFVIIa, and aPCC therapies or ITI to prophylactic Emicizumab resulted in ~21% savings in total and average patient costs. The total costs over 5-year for ‘with Emicizumab’ and ‘without Emicizumab’ scenarios were SAR 146.3 million and SAR 186.0 million; respectively ($1 USD= 3.75). The main drivers of these savings were lower bleeding rates and lower administration costs for Emicizumab compared with other treatments. Drug acquisition cost was the primary component of the total costs contributing over 75% of the total costs. Number of hospitalizations and surgeries related to hemorrhages were significantly lower in ‘with Emicizumab’ compared with ‘without Emicizumab’.

CONCLUSIONS

: The budget impact analysis suggested that switching HA pediatric patients with inhibitors to prophylactic Emicizumab therapy was associated with significant cost savings.