Economic evaluation in the context of rare diseases: is it possible?

• Published in: Cadernos de saúde pública Vol. 31; no. 3; pp. 496 - 506
• Main Authors: Silva, Everton Nunes da, Sousa, Tanara Rosângela Vieira
• Format: Journal Article
• Language: English
• Published: Brazil Escola Nacional de Saúde Pública Sergio Arouca, Fundação Oswaldo Cruz 01-03-2015; Escola Nacional de Saúde Pública, Fundação Oswaldo Cruz
• Subjects: Cost-Benefit Analysis - economics; Decision Making; Delivery of Health Care - economics; Economía de la Salud; Enfermedades Raras; Ethics, Research; Evaluación de Costo-Efectividad; Humans; Public Policy - economics; PUBLIC, ENVIRONMENTAL & OCCUPATIONAL HEALTH; Rare Diseases - economics; Technology Assessment, Biomedical - economics; Health Economics; Doenças Raras; Evaluación de Costo-Efectividad; Cost-Effectiveness Evaluation; Economia da Saúde; Rare Diseases; Avaliação de Custo-Efetividade; Economía de la Salud; Enfermedades Raras
• ISSN: 0102-311X; 1678-4464; 1678-4464; 0102-311X
• DOI: 10.1590/0102-311X00213813

This study analyzes the available evidence on the adequacy of economic evaluation for decision-making on the incorporation or exclusion of technologies for rare diseases. The authors conducted a structured literature review in MEDLINE via PubMed, CRD, LILACS, SciELO, and Google Scholar (gray literature). Economic evaluation studies had their origins in Welfare Economics, in which individuals maximize their utilities based on allocative efficiency. There is no widely accepted criterion in the literature to weigh the expected utilities, in the sense of assigning more weight to individuals with greater health needs. Thus, economic evaluation studies do not usually weigh utilities asymmetrically (that is, everyone is treated equally, which in Brazil is also a Constitutional principle). Healthcare systems have ratified the use of economic evaluation as the main tool to assist decision-making. However, this approach does not rule out the use of other methodologies to complement cost-effectiveness studies, such as Person Trade-Off and Rule of Rescue.