Economic evaluation in the context of rare diseases: is it possible?
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 literat...
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| Published in: | Cadernos de saúde pública Vol. 31; no. 3; pp. 496 - 506 |
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| Format: | Journal Article |
| Language: | English |
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Brazil
Escola Nacional de Saúde Pública Sergio Arouca, Fundação Oswaldo Cruz
01-03-2015
Escola Nacional de Saúde Pública, Fundação Oswaldo Cruz |
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| Abstract | 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. |
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| AbstractList | 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. 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. El objetivo fue sistematizar las evidencias disponibles sobre la pertinencia de utilizar la evaluación económica para la incorporación/exclusión de tecnología en enfermedades raras. Se realizó una revisión sistemática de la literatura en MEDLINE vía PubMed, CRD, LILACS, SciELO y Google Académico (literatura gris). Los estudios de evaluación económica se originan de la Economía del Bienestar, en la que los individuos maximizan sus utilidades, basándose en la eficiencia de asignación. No existe un criterio ampliamente aceptado para examinar las utilidades, a fin de dar más peso a los individuos con mayores necesidades. Generalmente, los estudios no equilibran asimétricamente las utilidades, todas son consideradas iguales, lo que en Brasil es también un principio constitucional. Los sistemas de salud han ratificado el uso de la evaluación económica como la principal herramienta para ayudar en la toma de decisiones. Sin embargo, este abordaje no excluye el uso de otras metodologías complementarias a los estudios de coste-efectividad, como la técnica de compensación personal o la regla del rescate. O objetivo deste estudo foi analisar as evidências disponíveis sobre a adequação do uso de avaliação econômica sobre incorporação/exclusão de tecnologias para doenças raras. Foi realizada uma revisão estruturada da literatura, nas bases MEDLINE, via PubMed, CRD, LILACS, SciELO e Google Acadêmico (literatura cinzenta). Os estudos de avaliação econômica têm origem na Economia do Bem-Estar, na qual os indivíduos maximizam suas utilidades, fundamentando-se na eficiência alocativa. Não há um critério amplamente aceito para ponderar as utilidades esperadas, no sentido de dar mais peso aos indivíduos com maiores necessidades em saúde. Geralmente não se ponderam assimetricamente as utilidades; todas são tratadas de forma igualitária, que, no caso brasileiro, também é um princípio constitucional. Os sistemas de saúde têm ratificado o uso de avaliação econômica como principal instrumento para auxiliar na tomada de decisão. No entanto, essa postura não exclui o uso de outras metodologias complementares aos estudos de custo-efetividade, como Person Trade-Off e regra de resgate. |
| Author | Silva, Everton Nunes da Sousa, Tanara Rosângela Vieira |
| AuthorAffiliation | Universidade Federal do Rio Grande do Sul Universidade de Brasília |
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| Cites_doi | 10.1111/j.1471-8847.2008.00233.x 10.1093/qjmed/hci128 10.1503/cmaj.081429 10.1002/(SICI)1099-1050(199701)6:1<71::AID-HEC239>3.0.CO;2-Z 10.1016/S0277-9536(02)00244-7 10.1002/(SICI)1099-1050(200003)9:2<127::AID-HEC500>3.0.CO;2-Y 10.1017/S0266462307051550 10.1007/s10389-010-0377-z 10.1002/hast.3 10.1002/(SICI)1099-1050(199902)8:1<25::AID-HEC398>3.0.CO;2-H 10.1590/S0104-42302011000600021 10.1590/S0102-311X2012000300008 10.1136/bmj.331.7523.1016 10.1590/S1413-81232010000900019 |
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| Keywords | 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 |
| Language | English |
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Altera a Lei nº 8.080, de 19 de setembro de 1990, para dispor sobre a assistência terapêutica e a incorporação de tecnologia em saúde no âmbito do Sistema Único de Saúde - SUS publication-title: Diário Oficial da União – volume: 43 start-page: 3 year: 2012 end-page: 4 article-title: How should we model rare disease allocation decisions? publication-title: Hastings Cent Rep – publication-title: Priority medicines for Europe and the world contributor: fullname: Kaplan, W; Laing, R – year: 2005 publication-title: Methods for the economic evaluation of health care programmes contributor: fullname: Drummond, MF; Sculpher, MJ; Torrance, GW; O'Brien, BJ; Stoddart, GL – volume: 19 start-page: 103 year: 2011 end-page: 111 article-title: Development of a weighted scale to assess the quality of cost-effectiveness studies and an application to the economic evaluations of tetravalent HPV vaccine publication-title: J Public Health – volume: 15 Suppl 3 start-page: 3443 year: 2010 end-page: 3454 article-title: Medicamentos de alto custo para doenças raras no Brasil: o exemplo das doenças lisossômicas publication-title: Ciênc Saúde Coletiva – volume: 28 start-page: 479 year: 2012 end-page: 489 article-title: Consequências da judicialização das políticas de saúde: custos de medicamentos para as mucopolissacaridores publication-title: Cad Saúde Pública – volume: 9 start-page: 127 year: 2000 end-page: 136 article-title: Societal value, the person trade-off, and the dilemma of whose values to measure for cost-effectiveness analysis publication-title: Health Econ – volume: 53 start-page: 941 year: 1963 end-page: 973 article-title: Uncertainty and the welfare economics of medical care publication-title: Am Econ Rev – start-page: 123 year: 2002 end-page: 140 publication-title: Economia da saúde: conceitos e contribuição para a gestão da saúde contributor: fullname: Pôrto, SM; Piola, SF; Vianna, SM – publication-title: 2012 report on the state of the art of rare disease activities in Europe - Part I: overview of rare disease activities in Europe – start-page: 1 year: 2001 end-page: 21 publication-title: Economic evaluation in health care: merging theory with practice contributor: fullname: McGuire, A; Drummond, M; McGuire, A – volume: 14 start-page: 16 year: 2008 end-page: 17 article-title: Challenges in the economic evaluation of orphan drugs publication-title: Eurohealth – publication-title: Orphan Drug Act: background and proposed legislation in the 107th congress contributor: fullname: Villarreal, MA – publication-title: Rare diseases and related terms – volume: 8 start-page: 25 year: 1999 end-page: 39 article-title: Incorporating societal concerns for fairness in numerical valuations of health programs publication-title: Health Econ – publication-title: Policies for orphan diseases and orphan drugs contributor: fullname: Denis, A; Simoens, S; Fostier, C; Mergaert, L; Cleemput, I – publication-title: Rare diseases: understanding this public health priority – volume: 182 start-page: E787 year: 2010 end-page: E792 article-title: Policy alternatives for treatments for rare diseases publication-title: CMAJ |
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| SubjectTerms | 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 |
| Title | Economic evaluation in the context of rare diseases: is it possible? |
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