OPERATIONALISING VALUE-BASED PRICING FOR ADVANCED THERAPY MEDICINAL PRODUCTS IN BRAZIL: A MULTI-STAKEHOLDER WORKSHOP INFORMING THE REVISION OF THE NATIONAL DRUG PRICING FRAMEWORK
Author(s)
Marcus C. Borin, PhD1, Ludmila Peres Gargano, PhD1, Mariana M. Barbosa, PhD1, Alex Brunno Nascimento Martins, MSc1, Marina Nascimento Silva, BSPharm1, Livia Silva Nassif, BSPharm1, Wallace Mateus Prata, PhD2, Mateus Amâncio, MSc3, Nicole Freitas de Mello, PhD4, Viviane Del Lama Cardoso Salas, BSPharm4, Luciene Bonan, PhD4, Juliana Alvares, PhD5, Augusto Afonso Guerra Junior, PhD6.
1CCATES/UFMG, Belo Horizonte, Brazil, 2FUNED; CCATES/UFMG, Belo Horizonte, Brazil, 3CMED - ANVISA, Brasília, Brazil, 4DGITIS/SCTIE/MS, Brasilia, Brazil, 5Federal University of Minas Gerais; CCATES/UFMG, Belo Horizonte, Brazil, 6Mohamed bin Zayed University of Artificial Intelligence, Abu Dhabi, UAE; CCATES/UFMG, Belo Horizonte, Brazil.
1CCATES/UFMG, Belo Horizonte, Brazil, 2FUNED; CCATES/UFMG, Belo Horizonte, Brazil, 3CMED - ANVISA, Brasília, Brazil, 4DGITIS/SCTIE/MS, Brasilia, Brazil, 5Federal University of Minas Gerais; CCATES/UFMG, Belo Horizonte, Brazil, 6Mohamed bin Zayed University of Artificial Intelligence, Abu Dhabi, UAE; CCATES/UFMG, Belo Horizonte, Brazil.
OBJECTIVES: To evaluate the feasibility of a value-based pricing framework combining the Pharmaceutical Innovativeness Index (PII) and the Efficiency Frontier (EF) as an alternative to International Reference Pricing (IRP) for advanced therapy medicinal products (ATMPs) in Brazil, and to generate consensus recommendations to inform the modernisation of the national pharmaceutical pricing framework.
METHODS: A three-day deliberative workshop was convened in Brasília, Brazil (March 2026) by CCATES/UFMG in partnership with DGITS/SECTICS and CMED/Anvisa, gathering researchers, regulators, payers, the regulated sector, civil society and international experts. Plenary sessions examined pricing experiences from France, Germany and Colombia. Six working groups applied the PII and the EF to two fictitious cases: a single-dose gene therapy for an ultra-rare paediatric neurodegenerative disease and a bispecific CAR-T for an aggressive haematological malignancy. Each group assessed pricing under the current IRP-based framework and under a PII+EF model. Group outputs were consolidated through structured plenary deliberation.
RESULTS: The PII+EF framework provided an objective structure to reduce decision-making subjectivity, increase transparency and align maximum regulated prices with incremental innovativeness and clinical utility. Consensus emerged on reducing the centrality of IRP, retaining it solely as an upper boundary, and on adopting dynamic pricing informed by real-world evidence, outcomes-based risk-sharing agreements and adaptive assessment criteria for orphan drugs. Calibration of the PII was identified as a methodological priority to avoid the inadvertent penalisation of therapies for rare diseases, where evidence limitations are expected. A recommendation was issued for CMED, in partnership with CCATES/UFMG, Anvisa and Conitec, to initiate a Regulatory Impact Analysis to update current resolutions.
CONCLUSIONS: The combined PII+EF approach is feasible as a structured decision-support tool for pricing ATMPs and provides a basis for regulatory modernisation in Brazil, supporting integration across pricing and HTA bodies while balancing system sustainability with equitable patient access.
METHODS: A three-day deliberative workshop was convened in Brasília, Brazil (March 2026) by CCATES/UFMG in partnership with DGITS/SECTICS and CMED/Anvisa, gathering researchers, regulators, payers, the regulated sector, civil society and international experts. Plenary sessions examined pricing experiences from France, Germany and Colombia. Six working groups applied the PII and the EF to two fictitious cases: a single-dose gene therapy for an ultra-rare paediatric neurodegenerative disease and a bispecific CAR-T for an aggressive haematological malignancy. Each group assessed pricing under the current IRP-based framework and under a PII+EF model. Group outputs were consolidated through structured plenary deliberation.
RESULTS: The PII+EF framework provided an objective structure to reduce decision-making subjectivity, increase transparency and align maximum regulated prices with incremental innovativeness and clinical utility. Consensus emerged on reducing the centrality of IRP, retaining it solely as an upper boundary, and on adopting dynamic pricing informed by real-world evidence, outcomes-based risk-sharing agreements and adaptive assessment criteria for orphan drugs. Calibration of the PII was identified as a methodological priority to avoid the inadvertent penalisation of therapies for rare diseases, where evidence limitations are expected. A recommendation was issued for CMED, in partnership with CCATES/UFMG, Anvisa and Conitec, to initiate a Regulatory Impact Analysis to update current resolutions.
CONCLUSIONS: The combined PII+EF approach is feasible as a structured decision-support tool for pricing ATMPs and provides a basis for regulatory modernisation in Brazil, supporting integration across pricing and HTA bodies while balancing system sustainability with equitable patient access.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HPR230
Topic
Economic Evaluation, Health Policy & Regulatory, Methodological & Statistical Research
Topic Subcategory
Pricing Policy & Schemes