0077/2021 - Transformando incertezas em regulamentação legitimadora? As decisões das agências NICE e CONITEC para doenças raras. Transforming uncertainties into legitimate regulation? The decisions of agencies NICE and CONITEC for rare diseases.
A avaliação de tecnologias em saúde (ATS), enquanto prática científica e tecnológica é, ao mesmo tempo, um desafio, a fim de determinar o valor das tecnologias a serem incorporadas. Este estudo teve como objetivo explorar e comparar os resultados e elementos técnicos das avaliações emitidas para doenças raras, entre a agência inglesa (NICE) e a brasileira (CONITEC). A primeira etapa do estudo envolveu a busca sistemática das avaliações no período de 2013 a 2019. Na segunda etapa, os relatórios foram analisados com base em: (i) revisão narrativa descritiva; e (ii) cálculo da frequência absoluta e relativa de acordo com cada domínio e componente (elemento) aplicado do modelo da rede Europeia de ATS. O total de 24 medicamentos foram distintamente avaliados no período do estudo. Por meio de 126 questões (elementos) distribuídas entre nove domínios, a análise revelou que 67 (53,2%) e 44 (35,0%) estavam descritas nos relatórios, 42 (33,3%) e 59 (47,0%) foram consideradas apenas parcialmente e 17 (13,5%) e 23 (18,0%) não foram consideradas nos relatórios do NICE e da CONITEC, respectivamente. Foi constatado uma concordância relativamente baixa da agência brasileira em relação a inglesa nos relatórios emitidos para doenças raras. Permanece indeterminado se as agências são capazes de capturar os diversos valores desses medicamentos, bem como gerenciar as incertezas nas avaliações.
Palavras-chave:
Avaliação de Tecnologias em Saúde, Doenças Raras, Regulamentação Governamental, Incerteza.
Abstract:
The evaluation of health technologies (HTA), as a scientific and technological practice, is, at the same time, a challenge in order to determine the value of the technologies to be incorporated. This study aimed to explore and compare the results and technical elements of the evaluations issued for rare diseases, between the English (NICE) and the Brazilian agency’s (CONITEC). The first part of the study involved the systematic search for evaluations2013 to 2019. In the second stage, the reports were analyzed based on: (i) descriptive narrative review; and (ii) calculation of the absolute and relative frequency according to each domain and component (element) applied in the European HTA network model. A total of 24 medicines were distinctly assessed during the study period. Through 126 questions (elements) distributed among nine domains, the analysis revealed that 67 (53.2%) and 44 (35.0%) were described in the reports, 42 (33.3%) and 59 (47.0 %) were only considered partially and 17 (13.5%) and 23 (18.0%) were not considered in the NICE and CONITEC reports, respectively. The Brazilian agency\'s relatively low agreement with the English agency was found in reports issued for rare diseases. It remains to be seen whether the agencies are able to capture the various values of these medicines, as well as manage uncertainties in the evaluations.
Keywords:
Health Technology Assessment, Rare Diseases, Government Regulation, Uncertainty.
Transforming uncertainties into legitimate regulation? The decisions of agencies NICE and CONITEC for rare diseases.
Resumo (abstract):
The evaluation of health technologies (HTA), as a scientific and technological practice, is, at the same time, a challenge in order to determine the value of the technologies to be incorporated. This study aimed to explore and compare the results and technical elements of the evaluations issued for rare diseases, between the English (NICE) and the Brazilian agency’s (CONITEC). The first part of the study involved the systematic search for evaluations2013 to 2019. In the second stage, the reports were analyzed based on: (i) descriptive narrative review; and (ii) calculation of the absolute and relative frequency according to each domain and component (element) applied in the European HTA network model. A total of 24 medicines were distinctly assessed during the study period. Through 126 questions (elements) distributed among nine domains, the analysis revealed that 67 (53.2%) and 44 (35.0%) were described in the reports, 42 (33.3%) and 59 (47.0 %) were only considered partially and 17 (13.5%) and 23 (18.0%) were not considered in the NICE and CONITEC reports, respectively. The Brazilian agency\'s relatively low agreement with the English agency was found in reports issued for rare diseases. It remains to be seen whether the agencies are able to capture the various values of these medicines, as well as manage uncertainties in the evaluations.
Palavras-chave (keywords):
Health Technology Assessment, Rare Diseases, Government Regulation, Uncertainty.
Vicente, G., Cunico, C., Leite, S. N.. Transformando incertezas em regulamentação legitimadora? As decisões das agências NICE e CONITEC para doenças raras.. Cien Saude Colet [periódico na internet] (2021/Mar). [Citado em 18/09/2024].
Está disponível em: http://cienciaesaudecoletiva.com.br/artigos/transformando-incertezas-em-regulamentacao-legitimadora-as-decisoes-das-agencias-nice-e-conitec-para-doencas-raras/17976?id=17976
