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Medicamentos de terapia avançada: desenvolvimento pré-clínico e clínico
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| 5.4 MB | Adobe PDF |
Advisor(s)
Abstract(s)
Avanços recentes na biomedicina têm permitido desenvolver novas abordagens
terapêuticas, culminando na criação de uma nova classe de medicamentos baseados em
células, tecidos ou genes – os medicamentos de terapia avançada (ATMPs, do inglês
Advanced Therapy Medicinal Products). Esta classe de medicamentos apresenta elevado
potencial para transformar a vida dos doentes e dos sistemas de saúde, proporcionando
novas opções terapêuticas para doenças para as quais o tratamento disponível é limitado ou
inexistente.
Devido às suas características únicas, os ATMPs, apresentam vários desafios durante
o processo de desenvolvimento e produção, distinguindo-os dos restantes produtos
farmacêuticos. Deste modo, as agências que regulam e supervisionam os produtos
farmacêuticos têm feitos esforços para disponibilizar as orientações necessárias para o
desenvolvimento e comercialização destes novos medicamentos inovadores. Na Europa, a
Agência Europeia do Medicamento (EMA, do inglês European Medicines Agency) publicou
várias guidelines acerca da qualidade, desenvolvimento pré-clínico e clínico dos ATMPs,
potenciando e regulando a entrada destes produtos no mercado.
Com base nas considerações anteriores, na presente Dissertação pretende-se
descrever: i) as diferentes categorias dos ATMPs, ii) as guidelines aplicáveis a estes
medicamentos, nomeadamente a avaliação do risco, processo de produção, avaliação préclínica
e clínica, iii) importância e aplicações destas terapias e iv) quais os ATMPs autorizados
atualmente pela EMA.
Na última década, mais de 15 ATMPs receberam autorização de introdução no
mercado (AIM) na Europa, no entanto algumas AIM não foram renovadas ou o medicamento
foi retirado do mercado por questões de segurança e/ou falta de eficácia ou viabilidade
económica. Assim, é necessário continuar o desenvolvimento de novos ATMPs assim como
do seu enquadramento legal, acelerando a aprovação destas terapias inovadoras e
mantendo os elevados padrões de qualidade, segurança e eficácia.
Recent advances in biomedicine have empowered the development of new therapeutic approaches, culminating in the creation of a new class of medicines based on cells, tissues, or genes – the advanced therapy medicines products (ATMPs). This class of medicines has high potential to transform the lives of patients and healthcare systems, providing new therapeutic options for diseases for which available treatment is limited or non-existent. Due to their unique characteristics, ATMPs present several challenges during the development and manufacturing process, differentiating them from other pharmaceutical products. Therefore, agencies that regulate and supervise pharmaceutical products have made efforts to provide the necessary guidance for the development and commercialization of these new innovative medicines. In Europe, the European Medicines Agency published several guidelines about quality, non-clinical and clinical development of ATMPs, promoting and regulating the entrance of these products into the market. Based on the previous considerations, this Dissertation aims to describe: i) the different categories of ATMPs, ii) the guidelines applicable to these medicines, namely risk assessment, manufacturing process, pre-clinical and clinical evaluation, iii) the importance and applications of these therapies, and iv) which ATMPs are currently authorized by EMA. In the last decade, more than 15 ATMPs have received marketing authorization (MA) in Europe, however some MAs weren’t renewed, or the medicine was withdrawn from the market due to safety concerns and/or lack of efficacy or economic viability. Therefore, it’s necessary to continue the development of new ATMPs as well as their legal framework, accelerating the approval of these innovative therapies and maintaining high standard of quality, safety and efficacy.
Recent advances in biomedicine have empowered the development of new therapeutic approaches, culminating in the creation of a new class of medicines based on cells, tissues, or genes – the advanced therapy medicines products (ATMPs). This class of medicines has high potential to transform the lives of patients and healthcare systems, providing new therapeutic options for diseases for which available treatment is limited or non-existent. Due to their unique characteristics, ATMPs present several challenges during the development and manufacturing process, differentiating them from other pharmaceutical products. Therefore, agencies that regulate and supervise pharmaceutical products have made efforts to provide the necessary guidance for the development and commercialization of these new innovative medicines. In Europe, the European Medicines Agency published several guidelines about quality, non-clinical and clinical development of ATMPs, promoting and regulating the entrance of these products into the market. Based on the previous considerations, this Dissertation aims to describe: i) the different categories of ATMPs, ii) the guidelines applicable to these medicines, namely risk assessment, manufacturing process, pre-clinical and clinical evaluation, iii) the importance and applications of these therapies, and iv) which ATMPs are currently authorized by EMA. In the last decade, more than 15 ATMPs have received marketing authorization (MA) in Europe, however some MAs weren’t renewed, or the medicine was withdrawn from the market due to safety concerns and/or lack of efficacy or economic viability. Therefore, it’s necessary to continue the development of new ATMPs as well as their legal framework, accelerating the approval of these innovative therapies and maintaining high standard of quality, safety and efficacy.
Description
Keywords
Medicamentos de terapia avançada Legislação eu Padrões de qualidade e segurança Desenvolvimento pré-clínico