Applying a Pareto-Based Efficiency Frontier to Inform Value-Based Pricing for Gaucher Disease Therapies in Brazil.

purposeGaucher disease (GD) requires lifelong high-cost treatment within the Brazilian Unified Health System (SUS). In Brazil, treatment eligibility and monitoring are defined by Clinical Protocol and Therapeutic Guidelines (PCDT), while pharmaceutical prices are regulated by the Drug Market Regulation Chamber (CMED). Exploratory price-value approaches may help make explicit the relationship between drug prices and measurable clinical benefit. We explored the applicability of a Pareto-based Efficiency Frontier (EF) as a descriptive hematologic price-value framework for GD therapies in Brazil.

methodsAn exploratory Pareto-based EF analysis compared annual drug acquisition prices with standardized hematologic benefits, defined as hemoglobin and platelet improvements, derived from a published systematic review. Annual prices were obtained from CMED, Maximum Price of Sale to the Government (PMVG), and median public procurement prices from the Health Price Database (BPS). Prices are presented in Brazilian reais and USD adjusted by purchasing power parity. Directly dominated technologies were excluded, and all remaining directly non-dominated therapies were connected by linear interpolation to form a Pareto-based 12-month reference EF. This construction retains alternatives that would be excluded by extended dominance under a conventional convex EF. The analysis was restricted to treatment-naïve patients.

resultsAnnual costs ranged from approximately USD-PPP 94900 to 763 200. Under the primary 12-month analysis, imiglucerase was directly dominated across all three pricing scenarios, whereas taliglucerase alfa, velaglucerase alfa, and miglustat defined the Pareto-based frontier. In the BPS hemoglobin analysis and in all three platelet analyses, taliglucerase alfa would be subject to extended dominance under a conventional convex EF but was retained as a directly non-dominated therapy. This classification was conditional on indirect comparisons from heterogeneous evidence and the selected 12-month hematologic point estimates. In the descriptive scenario analysis, direct dominance of imiglucerase was less stable when maximum effects across longer follow-up durations were considered.

conclusionThis exploratory application of a Pareto-based Efficiency Frontier describes the relationship between current price scenarios and comparative hematologic benefit for GD therapies. Findings should not be interpreted as definitive comparative value evidence, an ICER-based optimization rule, or a stand-alone pricing instrument. The approach may complement broader value-based assessment alongside QALY-based modeling, long-term outcomes, formal uncertainty analysis, and non-hematologic clinical dimensions.