Abstract
Introduction: Health Technology Assessment (HTA) stands out as a valuable tool to inform decision-making involving pricing, reimbursement, investment, and the development of therapeutic guidelines, capable of determining the quality and value of pharmaceutical innovations. However, defining innovation, or even judging the value of a new drug, can be a challenge, and there is still no consensus among the stakeholders involved. The Italian Medicines Agency (AIFA) has established an approach to assess innovation based on therapeutic need, added therapeutic value, and quality of evidence. Like this, other methodologies propose to assess innovations, but with a high degree of subjectivity and lack of transparency regarding the criteria used. Understanding innovativeness as the added therapeutic value considering society's preferences and needs, this study proposes a transparent tool, with well-defined criteria and roadmap, to determine pharmaceutical innovativeness with clinical and methodological parameters, based on the social relevance and therapeutic value of new drugs. Methods: The study was developed by adapting two HTA-based methods identified in the literature through a comprehensive search (AIFA methodology and EVITA instrument). The instruments were applied to oncology drugs approved by the FDA between 2011 and 2021 by a group of three researchers. After each round of evaluation, the researchers discussed and aligned the parameters, criteria, and domains in consensus meetings. For the domains identified as relevant, objective criteria were established for classification into levels, and each level received a score. Finally, an algorithm was proposed considering the scores received, adjusted for the relevance of the domain, i.e., weights assigned to the domains. Results: The Innovativeness Index (II) was proposed, with an evaluation script, domains, criteria, and an algorithm. The evaluation should begin with the definition of the clinical indication, outcomes of interest, identification of therapeutic alternatives, temporal perspective, and data sources for analysis. Four domains were considered: 1) Therapeutic Need, which assesses the existence and benefits of alternatives; and 2) Added Therapeutic Value, which refers to the incremental clinical benefit when compared to alternatives; these domains were graded into five levels ranging from absent to maximum; 3) Study Design; and 4) Methodological Quality, both classified into three levels. The criteria for the drug to be classified into each level can be adapted according to the indication and previously defined outcomes. The weights of the domains were assigned by the researchers considering the social perspective, but will be validated in the next steps of the study. Discussion and conclusions: The proposed methodology considers clinical and social value weighted by the methodological limitation of the available evidence to determine the degree of innovation of pharmaceutical products. The II stands out as a transparent, adaptable and reproducible tool, and aims to reduce the subjectivity of the analyses and assist decision-making. The II has the potential to assist healthcare payers, pricing agencies (e.g. CMED in Brazil), investors in the pharmaceutical market, researchers, physicians and governments.
