OBJECTIVES: Lipodystrophy (LD) comprises a heterogeneous group of disorders characterized by generalized (GL) or partial (PL) lack of adipose tissue. The global prevalence of LD is estimated to range between 1.3 and 4.7 cases per million. However, the prevalence and economic burden of LD in Saudi Arabia remain unknown. Metreleptin, a leptin analog, is used as an adjunct to diet and other medications for disease-associated complications in patients with congenital or acquired LD. The aim of this study is to estimate the budgetary impact of using Metreleptin on payers financial resources to inform decision-makers in Saudi Arabia.

METHODS: A budget impact analysis was conducted from the perspective of public payers, considering a 3-year time horizon and focusing on Metreleptin different strengths (11.3mg, 5.8mg, and 3mg). The analysis utilized direct medical costs, and LD-eligible patients were identified based on expert input from physicians treating LD patients in Saudi Arabia. Market share assumptions were set at 50% in year 1, 75% in year 2, and 100% in year 3. To assess the robustness of the results, one-way sensitivity analysis (OWSA) was performed.

RESULTS: Over the 3-year period, the cumulative budget impact for GL patients was USD 25,297,375, and for PL patients, it was USD 12,276,945. The total costs for Metreleptin strengths (3mg, 5.8mg, and 11.3mg) were USD 2,218,702, USD 11,546,121, and USD 23,809,497, respectively, for the included patient population. After performing scenario analyses, the total budget impact decreased by 20.42% of the overall estimated Metreleptin cumulative budget. The OWSA indicated that the analysis is sensitive to the price of Metreleptin and disease prevalence.

CONCLUSIONS: Given the uncertain assumptions regarding the total number of eligible patients and subgroups (GL and PL) for Metreleptin each year, small variations in patient numbers could have a significant effect on the overall budget impact.