OBJECTIVES: Inherited retinal diseases (IRDs) are characterized by functional vision loss and genetic mutations. Voretigene neparvovec (VN) is the first gene therapy approved for the treatment of leber congenital amaurosis, a severe progressive IRD, and is indicated for vision loss caused by RPE65 gene mutations. This study aimed to assess the cost effectiveness and budgetary impact of applying VN for treating IRDs in the Kingdom of Saudi Arabia (KSA).

METHODS: Cost effectiveness analysis (CEA) and business impact analysis (BIA) models were constructed to compare treatment with VN for IRD with and without managed entry agreement (MEA). Input data included clinical, utility, cost, population, and market share data obtained from publications, key opinion leaders (KOLs), and Ministry of Health (MOH) sources. For CEA, the outcomes included cost per quality-adjusted life-year (QALY) gained and additional outcomes reported. For BIA, the model outcomes included total budget impact and total incremental cost over five years.

RESULTS: CEA results showed that for a WTP threshold of SAR 75,000, the incremental cost of VN use was SAR -644,425 and 5.6 QALYs (blindness-free years: 14.9 with VN vs. 9.6 with best supportive care (BSC)) (incremental cost effectiveness ratio (ICER) is dominant). In the base case, in the societal perspective, the total cost is lower with VN (SAR 5,571,527) compared to BSC (SAR 6,215,952). BIA results showed that the total 5-year cost of managing IRDs with BSC will be SAR 30 Million, and the total incremental cost of introducing VN will be SAR 69 Million without MEA and SAR 64 Million with MEA.

CONCLUSIONS: In the KSA, introducing VN will result in significantly improved clinical outcomes over a period of five years, and a lower budget increase can be achieved with MEA. The utility value parameters have a high impact on the cost-effectiveness of VN use.