Valor terapêutico versus valor monetário: análise das inovações farmacêuticas e a precificação de medicamentos no Brasil.

Abstract

INTRODUCTION: Pharmaceutical innovation plays a key role in advancing healthcare, contributing to increased life expectancy and quality of life, as well as reducing the global burden of disease. However, the growing number of innovative and high-cost medicines has placed increasing pressure on public and private budgets. In this context, defining the value of these innovations is a multidimensional challenge that involves criteria such as clinical efficacy, safety, economic impact, and social benefits. Value-Based Pricing (VBP) has emerged as a promising strategy to align drug prices with the actual therapeutic benefits offered by new technologies. Therefore, it is essential to develop objective, transparent, and reproducible criteria for measuring pharmaceutical innovativeness, in order to support clinical, regulatory, and economic decision-making. OBJECTIVES: A) To propose a transparent and reproducible method for evaluating the innovativeness of new medicines; B) To discuss its applicability in Brazil’s economic regulation model, in light of VBP strategies; C) To test the feasibility of adapting the tool across different therapeutic contexts. METHODS: A) The Pharmaceutical Innovativeness Index (PII) was developed based on the Health Technology Assessment (HTA) framework and a critical review of international methodologies. The PII was initially applied to oncology drugs approved by the FDA between 2011 and 2021, followed by validation rounds with experts, practical testing, and methodological refinements. B) To assess the applicability of the PII in Brazil’s economic regulation context, a focus group was conducted with experts in health economics and drug pricing, who evaluated the index domains, their relevance, and practical use. Fictional case studies were used to test the tool’s discriminative capacity. Finally, a proposal was developed for incorporating the PII into Brazil’s pricing process for pharmaceutical innovations. C) The PII was then adapted to the context of innovations for type 2 diabetes mellitus, allowing its application to prevalent chronic conditions. RESULTS: A) The PII includes four domains: two related to clinical and social dimensions – Unmet Therapeutic Need (UTN) and Added Therapeutic Value (ATV) – and two related to methodological features – Study Design and Methodological Quality (risk of bias). The combined scores assigned to each domain determine the degree of pharmaceutical innovativeness. The analysis of oncology drugs showed wide variation in innovativeness levels, reflecting differences in added therapeutic value and evidence quality. B) The focus group indicated that the proposed domains were appropriate and that the PII is a useful tool to support economic regulation decisions. The regulatory proposal developed demonstrates the feasibility of incorporating the index as an additional criterion in CMED’s pricing processes, promoting greater alignment with international VBP experiences. C) The application of the PII to type 2 diabetes mellitus innovations highlighted the tool’s adaptability to different clinical settings, including those characterized by high prevalence, low lethality, and multiple therapeutic options. CONCLUSION: The PII proved to be a flexible and reproducible tool for evaluating pharmaceutical innovativeness across diverse therapeutic areas, by offering objective and consistent criteria to estimate the added value of new technologies. The proposed economic regulation model for medicines in Brazil, incorporating the PII as an additional pricing criterion, aims to align the monetary value of drugs with their therapeutic value. This strategy seeks to promote greater coherence and transparency in pricing decisions and to encourage the development of technologies that provide meaningful clinical benefits to the population.