The Pharmaceutical Innovativeness Index: Supporting Value-Based Economic Regulation of Innovative Medicines

The regulation of new medicine prices must balance financial sustainability with equitable access to innovation. Value-Based Pricing (VBP) strategies seek to align drug prices with their clinical and societal impact. The Pharmaceutical Innovativeness Index (PII) is a transparent and reproducible tool proposed to assess the degree of innovativeness of new medicines, with potential to support pricing decisions within economic regulation frameworks. An exploratory qualitative study was conducted through a focus group study was conducted with experts in health economics and pharmaceutical regulation to evaluate the applicability of the PII and to discuss key domains relevant to the assessment of pharmaceutical innovation. Responses were collected anonymously using an interactive digital platform and analyzed through inductive thematic content analysis. Based on these findings, the research team developed a conceptual pricing model integrating the PII with additional value-based criteria. Two hypothetical case studies were created to explore its practical feasibility. Participants identified Added Therapeutic Value (ATV) and Unmet Therapeutic Need (UTN) as the most relevant domains, while Methodological Quality (MQ) and Study Design (SD) were also recognized as essential to ensure rigor and reduce bias. The PII scores showed strong alignment with expert judgment in the illustrative case studies. The proposed model incorporates international best practices—such as the efficiency frontier approach—and additional dimensions including safety and incremental innovation. Overall, the PII demonstrated potential to enhance transparency, consistency, and regulatory efficiency in drug pricing decisions in Brazil. However, it should currently be regarded as an exploratory framework requiring further empirical validation and regulatory adaptation before implementation.