PB1235 - Budget impact analysis of gene therapy for hemophilia A, and hemophilia B for the Saudi Arabian public health sector

Background: Gene therapy emerged as a transformative treatment option for hemophilia, potentially offering long-term effects with a single administration. This novel approach, though associated with high upfront costs, could significantly alter the economic landscape of hemophilia care, especially in Saudi Arabia.

Aims: To assess the budget impact associated with introducing gene therapy as a treatment option for all eligible patients, on the public healthcare sector in Saudi Arabia.

Methods: This analysis was conducted for a 5-years’ time horizon 2024-2029, from a societal perspective. The target population was adult hemophilia A and B patients, without FVIII/FIX inhibitors, respectively. Costing data were collected from the Saudi Food and Drug Authority's database, literature, and surveys. We assumed a 20% market share of hemophilia A gene therapy in the eligible cohort (n=159) at the price of $1,545,000/patient, and a 30% market share of the hemophilia B gene therapy in the eligible cohort (n=45) and the price of $3,500,000/patient. A series of sensitivity analyses were conducted to identify the factors which impacted budget the most.

Results: The administration of gene therapy to eligible Saudi Arabian patients would result in a marginal rise in the five-year cumulative costs from $217,928,984 to $220,572,455 (1.2%) for hemophilia A (Figure 1) and from $53,793,817 to $80,312,402 (49.3 %) for hemophilia B (Figure 2). Sensitivity analysis suggested that the model is sensitive to gene therapy acquisition cost, and market share.

Conclusion(s): Adoption of gene therapy for eligible patients in Saudi Arabia had a limited impact on healthcare budget for hemophilia A and a significant impact for hemophilia B. However, these considerable upfront expenses, may lead to cost savings over time, contingent on the sustained effectiveness of FVIII or FIX expression. This is based on the premise that the longevity of therapeutic expression can offset the initial investment, thereby presenting an economically viable treatment option.