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Publicação
Impacto da legislação pediátrica no desenvolvimento de novos medicamentos: comparação entre Europa, Estados Unidos da América e Japão
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Orientador(es)
Resumo(s)
O mercado de medicamentos pediátricos é pequeno e o desenvolvimento destes é
dispendioso, sendo maioritariamente financiado pela mesma indústria que desenvolve
fármacos para adultos. Historicamente, o desenvolvimento e a adaptação de
medicamentos para crianças nem sempre foi feita com base em evidências e estudos
rigorosos. Ainda hoje se evidencia uma proporção significativa desta população tratada
com medicamentos que não são indicados ou aprovados para uso pediátrico, sendo a
utilização off-label comprovada por vários estudos.
Os Estados Unidos da América (EUA), a União Europeia (UE) e o Japão
desenvolveram várias estratégias na tentativa de melhorar o desenvolvimento de
tratamentos pediátricos eficazes e acessíveis. Algumas abordagens passaram por
promulgar legislação pediátrica específica e adotar diretrizes para promover a realização
de ensaios clínicos. Relativamente à asma, uma das doenças mais prevalentes
mundialmente entre crianças e adultos, há a necessidade de investigar de forma mais
aprofundada a própria fisiopatologia da doença de forma a desenvolver medicamentos
mais eficazes.
Uma vez que, atualmente, a maioria dos novos fármacos para adultos tem origem na
UE, EUA e Japão, o presente trabalho procurou a avaliar a influência da legislação no
desenvolvimento de medicamentos pediátricos nessas regiões. Verificou-se que as ações
regulatórias nos EUA e na UE permitiram promover a realização de ensaios clínicos e o
desenvolvimento de novos medicamentos pediátricos, combinando o poder regulatório e
recompensas para a indústria farmacêutica. No geral, a importância da regulamentação
pediátrica e desenvolvimento específico de medicamentos pediátricos parece ganhar
relevância nas 3 regiões. Seria de esperar uma vantagem mais evidente dos EUA a todos os níveis, uma vez que a sua legislação pediátrica surgiu primeiro, mas tal não se
verificou. Relativamente à asma o Japão aparenta possuir maior preocupação em
envolver crianças nos estudos, demonstrando a importância dada ao desenvolvimento
pediátrico de medicamentos, mesmo num país onde não existem leis específicas para tal.
The market for pediatric medicines is small and the development of these drugs is expensive and mostly funded by the same industry that develops adult drugs. Historically, the development and adaptation of medicines for children has not always been based on rigorous evidence and studies. Even today there is a significant proportion of this population treated with medicines that are not indicated or approved for pediatric use, and the off-label use in children is proven by several studies. The United States of America (USA), the European Union (EU) and Japan have developed various strategies to improve the pediatric treatment and access to medicines. Some of the approaches were about enacting specific pediatric legislation and to adopt guidelines to promote clinical trials. Regarding asthma, one of the most prevalent diseases worldwide among children and adults, there is a need to further investigate the pathophysiology of the disease in order to develop more effective drugs. Since most of the new adult drugs currently come from the EU, USA and Japan, the present work has sought to assess the influence of legislation on the development of pediatric medicines in these regions. Regulatory action in the USA and EU has been shown to promote clinical trials and the development of new pediatric medicines by combining regulatory power and rewards for the pharmaceutical industry. From the general point of view, the importance of pediatric regulation and specific development of pediatric medicines seems to be gaining relevance in the 3 regions. It would be expected a more obvious advantage of the USA at all levels, since its pediatric legislation was enacted first, but this was not the case. Regarding asthma, Japan was apparently more aware of pediatric involvement in clinical studies, demonstrating the importance given to pediatric drug development even in a country where there are no specific laws for that.
The market for pediatric medicines is small and the development of these drugs is expensive and mostly funded by the same industry that develops adult drugs. Historically, the development and adaptation of medicines for children has not always been based on rigorous evidence and studies. Even today there is a significant proportion of this population treated with medicines that are not indicated or approved for pediatric use, and the off-label use in children is proven by several studies. The United States of America (USA), the European Union (EU) and Japan have developed various strategies to improve the pediatric treatment and access to medicines. Some of the approaches were about enacting specific pediatric legislation and to adopt guidelines to promote clinical trials. Regarding asthma, one of the most prevalent diseases worldwide among children and adults, there is a need to further investigate the pathophysiology of the disease in order to develop more effective drugs. Since most of the new adult drugs currently come from the EU, USA and Japan, the present work has sought to assess the influence of legislation on the development of pediatric medicines in these regions. Regulatory action in the USA and EU has been shown to promote clinical trials and the development of new pediatric medicines by combining regulatory power and rewards for the pharmaceutical industry. From the general point of view, the importance of pediatric regulation and specific development of pediatric medicines seems to be gaining relevance in the 3 regions. It would be expected a more obvious advantage of the USA at all levels, since its pediatric legislation was enacted first, but this was not the case. Regarding asthma, Japan was apparently more aware of pediatric involvement in clinical studies, demonstrating the importance given to pediatric drug development even in a country where there are no specific laws for that.
Descrição
Tese de mestrado, Regulação e Avaliação do Medicamento e Produtos de Saúde, 2019, Universidade de Lisboa, Faculdade de Farmácia.
Palavras-chave
Legislação pediátrica Desenvolvimento de medicamentos Utilização off label Asma Teses de mestrado - 2019