Background: The pricing regulation of new medicines must balance financial sustainability with access to innovation. Value-Based Pricing (VBP) strategies aim to align drug prices with their clinical and social impact. The Pharmaceutical Innovativeness Index (PII) is a proposed tool to assess the degree of innovativeness of medicines with the potential to support pricing decisions within economic regulation frameworks. Methods: An exploratory study was conducted using a focus group with experts in health economics and pharmaceutical regulation. Participants evaluated the applicability of the PII in drug pricing and discussed key domains relevant to the pricing of pharmaceutical innovations. Based on the focus group results, a pricing model was developed to integrate the PII and other critical criteria into the decision-making process. Two case studies were used to assess the feasibility of implementing the proposed model. Results: Participants rated Added Therapeutic Value (ATV) and Unmet Therapeutic Need (UTN) as the most relevant domains in determining innovativeness. Methodological Quality (MQ) and Study Design (SD) were also considered important to reduce bias in the evaluation process. The PII scores showed good alignment with expert perceptions in the case studies. The proposed model incorporates international practices and includes additional criteria such as safety and incremental innovation. Conclusion: The proposed pricing model provides a structured approach to assess and reward innovative technologies. The PII demonstrated potential to enhance transparency, consistency, and regulatory efficiency in drug pricing decisions in Brazil, though regulatory adjustments would be required for implementation.

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