A atuação da Agência Nacional de Vigilância Sanitária na avaliação crítica de ensaios clínicos apresentados para registro de medicamentos biológicos: estudo de caso com filgrastim

• Ano de defesa: 2011
• Autor(a) principal: Thees, Maria Fernanda Reis e Silva [UNIFESP]
• Tipo de documento: Dissertação
• Tipo de acesso: Acesso aberto
• Idioma: por
• Instituição de defesa: Universidade Federal de São Paulo (UNIFESP)
• Palavras-chave em Português: Agência Nacional de Vigilância Sanitária - Anvisa; ensaio clínico; produtos biológicos; Qualidade; Fator Estimulador de Colônias de Granulócitos; Controle de Qualidade; Controle de Medicamentos e Entorpecentes; Ensaios Clínicos como Assunto; Quality; clinical trial; biological products; Granulocyte Colony-Stimulating Factor; Quality Control; Drug and Narcotic Control; Clinical Trials as Topic; Produtos Biológicos/normas; Biological Products/standards
• Link de acesso: http://repositorio.unifesp.br/handle/11600/8807

Objectives: this study aimed to determine whether Anvisa performed a critical assessment of the quality of the clinical trials submitted at the market authorization of biological products, and to develop a guideline for the quality assessment of clinical trials for Anvisa’s staff to use in order to improve and standardize the evaluation of the registration process and to contain unnecessary risks to the population. Methods: it was chosen as a reference for carrying out the analysis the product filgrastim (GCSF). The clinical trials submitted to Anvisa were collected for the ten G-CSF found at Anvisa’s system. The quality assessment was performed using the Cochrane Method for those studies that aimed to evaluate the efficacy and safety of filgrastim. In parallel, a systematic search was carried out in electronic databases to locate published clinical trials that had evaluated the safety and efficacy of filgrastim for comparison of their outcomes with the ones reported in the studies presented for Anvisa, in order to detect any publication bias in the later. Results: of the 20 studies included for quality assessment, only three had low risk of bias, according to the Cochrane Method. By comparison of the outcomes between studies found in Anvisa and those found in the systematic search, a publication bias was detected in the first, with a higher percentage of "hard" outcomes, that is, real clinical efficacy outcomes, reported in the studies of the systematic search than in the trials submitted to the registry. By the problems detected, it was possible to develop a guideline and a checklist for assessing the quality of clinical trials to be used internally by the staff of Anvisa. Conclusion: it was shown that Anvisa’s specialist staff must be better prepared to assess the methodological quality of clinical trials of biological products and that the Agency seeks to improve its analysis, with this research being one of the first steps towards technical excellence.