Translational Pharmacoeconomics in Rare Diseases: Integrating Clinical, Economic, and Social Value in Health Decision-Making

Rare diseases pose a growing challenge for health systems due to the combination of high clinical need, limited evidence, and elevated costs. This article analyzes the intersection between translational medicine and pharmacoeconomics, identifying a structural gap between the clinical value of innovations and traditional economic evaluation frameworks. Drawing on a narrative review of the literature—in which 240 scientific articles were identified and the 30 most methodologically and thematically relevant were selected through systematic screening by title, abstract, and full-text relevance—and a comparative analysis of frameworks in Europe, the United States, and Latin America, we propose the concept of translational pharmacoeconomics as an integrative framework that explicitly incorporates clinical, economic, and social dimensions of value into health decision-making. This approach has direct implications for the design of more equitable, adaptive, and sustainable health policies, particularly in contexts of evidence scarcity and unmet clinical need.