Presentation (CTI)

OBJECTIVES: The rapid emergence of advanced therapies and medicines for rare diseases has exposed important limitations in the current Brazilian pharmaceutical pricing model. Reliance on International Reference Pricing has shown limited ability to recognize therapeutic value, address uncertainty, or support sustainable access to highly innovative technologies. In parallel, Brazil has initiated discussions on modernization of pharmaceutical economic regulation involving multiple stakeholders. Within this context, value-based pricing (VBP) models have gained relevance as alternatives for aligning prices with therapeutic benefit and health system sustainability. The objective ir to present a conceptual framework for VBP of innovative medicines in Brazil.
METHODS: Methodological research on pharmaceutical innovativeness assessment, including review of international models and conceptual adaptation to the Brazilian regulatory environment. The proposal incorporates the Pharmaceutical Innovativeness Index (PII), composed of four domains: Unmet Therapeutic Need, Added Therapeutic Value, Study Design, and Methodological Quality. The framework was discussed with specialists in health technology assessment, pharmaceutical pricing and public policy through expert consultations, focus groups, and technical workshops. Validation discussions included practical exercises using fictional advanced therapy case studies to explore innovativeness classification, efficiency frontier approaches, provisional pricing, post-marketing reassessment, and real-world evidence generation.
RESULTS: The framework proposes a structured VBP approach aligned with therapeutic value, innovativeness, and evidence robustness. The efficiency frontier is proposed as an economic anchor to maintain proportionality between incremental innovativeness and price. Technologies with higher PII scores may receive differentiated pricing pathways, while products with greater uncertainty would require conditional pricing and reassessment. Incremental innovations may receive conditional premiums linked to gains in safety, adherence, or convenience. The model seeks to improve transparency, predictability, and consistency in pricing decisions while maintaining incentives for meaningful innovation.
CONCLUSIONS: The proposed framework may contribute to ongoing discussions regarding modernization of pharmaceutical economic regulation in Brazil, particularly for advanced therapies and other high-cost innovative technologies.