Applying the Efficiency Frontier to Inform Value-Based Pricing for Gaucher Disease Therapies in Brazil

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Background: Gaucher Disease (GD) is a rare genetic disorder characterized by visceral and hematological clinical manifestations. Various therapeutic options are currently available, all associated with high costs to the healthcare system, requiring rigorous economic evaluations to support decisions on the incorporation and pricing of these technologies in the Brazilian Unified Health System (SUS). Objectives: To assess the applicability of the Efficiency Frontier (EF), an economic methodology that correlates annual costs with specific clinical benefits, for the rational pricing of available therapies for GD in the Brazilian context. Methods: A comparative economic analysis was conducted between imiglucerase, velaglucerase alfa, taliglucerase alfa (Enzyme Replacement Therapy - ERT), and miglustat (Substrate Synthesis Inhibition - SSI) from the perspective of the SUS. Clinical efficacy data, assessed by the average increases in hemoglobin levels and platelet counts, were extracted from a previously published systematic review and meta-analysis. Annual costs were obtained from official sources: CMED (with an 18% tax), Maximum Price of Sale to the Government (PMVG), and the Health Price Database (BPS). The EF was constructed by identifying and excluding dominated therapies (those with higher costs and equal or lower efficacy), allowing for the derivation of linear equations that relate maximum acceptable costs to the clinical benefits achieved. Results: Imiglucerase was consistently dominated in all analyzed scenarios, while taliglucerase alfa, velaglucerase alfa, and miglustat constituted the EF. Conclusion: The results indicated a discrepancy between currently practiced prices and the clinical benefits obtained, suggesting that the EF methodology can be a useful complementary tool to traditional analyses, contributing to the rationalization of health resources, particularly for rare diseases

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