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

Using a Pareto Efficiency Frontier to Guide Value-Based Pricing of Gaucher Disease Therapies in Brazil

By
Marcus Carvalho Borin
Ludmila Peres Gargano
Francisco de Assis Acurcio
Juliana Álvares-Teodoro
Augusto Afonso Guerra
October 9, 2026

Pharmacoepidemiology And Drug Safety

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1

The study explored a Pareto-based price–benefit analysis of Gaucher disease therapies available through Brazil’s SUS in January 2023.
2

The analysis included imiglucerase, velaglucerase alfa, taliglucerase alfa, and miglustat; eliglustat was excluded because SUS did not provide it.
3

The study related annual drug acquisition costs to selected hematologic benefits, particularly hemoglobin and platelet outcomes.
4

The analysis was not a full economic evaluation and excluded utilities, QALYs, long-term survival modeling, budget impact, and probabilistic uncertainty analysis.
5

The Pareto-based frontier was intended as a descriptive price–benefit benchmark, not an optimization rule or independent pricing criterion.

Original Source(s)

Pharmacoepidemiology And Drug Safety

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

by Marcus Carvalho Borin, Ludmila Peres Gargano, Francisco de Assis Acurcio, Juliana Álvares-Teodoro, Augusto Afonso Guerra
October 9, 2026

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