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Advisor(s)
Abstract(s)
Os medicamentos biológicos são medicamentos obtidos ou derivados de organismos vivos
sendo que, nas últimas duas décadas, a sua utilização clínica tem demonstrado uma enorme e
importante relevância no tratamento de doenças graves e crónicas. Estes medicamentos diferem
de medicamentos de síntese química devido à sua maior complexidade molecular e,
inerentemente, ao maior potencial imunogénico que apresentam. Apesar destes medicamentos
trazerem uma melhor qualidade de vida aos doentes, o acesso a este tipo de medicamentos
tornou-se limitado e desafiador, explicado principalmente pelo seu elevado custo.
O início da queda de várias patentes de medicamentos biológicos permitiu a chegada dos
medicamentos biossimilares, que vieram trazer aos sistemas de saúde uma poupança financeira,
e às indústrias biofarmacêuticas o desafio da comprovação da biossimilaridade com o
medicamento biológico de referência.
Com a entrada dos medicamentos biossimilares no panorama biofarmacêutico, outros
medicamentos começaram a surgir, nomeadamente os medicamentos biossuperiores, os
medicamentos biológicos não-originais e ainda os medicamentos stand-alone. Apesar destes
medicamentos colmatarem necessidades do mercado, tornou-se bastante claro que a base
regulamentar e a terminologia do mundo dos medicamentos biológicos se tornou complexa e
inexacta a nível global. A entrada no mercado de medicamentos com perfis de segurança,
qualidade e eficácia duvidosos torna-se assim uma realidade e um risco que podiam ser
impedidos através de uma harmonização regulamentar entre agências reguladoras. O
surgimento de conceitos como a permutabilidade, troca, substituição automática e ainda a
extrapolação de dados são de extrema relevância já que as suas definições não são consensuais
entre agências reguladoras, levando a divergências regulamentares em várias regiões do
planeta.
O mercado biofarmacêutico continuará a crescer e a desenvolver-se no futuro, tornando-se
imperativo uma confluência de esforços para uma maior coesão tanto a nível regulamentar
como de terminologia.
Biologic medicines are obtained or derived from living organisms and, in the last two decades, their clinical use has demonstrated an enormous and important relevance in the treatment of serious and chronic diseases. These medicines differ from synthetic drugs due to their greater molecular complexity and, inherently, the greater immunogenic potential they tend to present. Although biologic medicines bring a better quality of life to patients, the access to this medicines has become limited and challenging, mainly explained by their high cost. The lapsing of several patents for biological medicines allowed the arrival of biosimilar medicines, which came to bring financial savings to health systems, and to biopharmaceutical industries the challenge of proving biosimilarity with the reference biological medicine. With the introduction of biosimilar medicines in the biopharmaceutical panorama, other medicines started to appear, such as biobetter medicines, non-original biologics medicines and stand-alone medicines. Although these medicines address market needs, it has become clear that the regulatory base and terminology in the world of biologic medicines has become complex and globally inaccurate. The entry of medicines with uncertain safety, quality and efficacy profiles in the biopharmaceutical market may become a reality and a risk that could be prevented through regulatory harmonization between regulatory agencies. The arise of concepts such as interchangeability, switch, automatic substitution and data extrapolation are extremely relevant since their definitions are not consensual among regulatory agencies, leading to regulatory divergences in various regions of the planet. In the future, the biopharmaceutical market will continue to develop and grow, meaning that a confluence of efforts for greater cohesion at a regulatory and terminology level is imperative.
Biologic medicines are obtained or derived from living organisms and, in the last two decades, their clinical use has demonstrated an enormous and important relevance in the treatment of serious and chronic diseases. These medicines differ from synthetic drugs due to their greater molecular complexity and, inherently, the greater immunogenic potential they tend to present. Although biologic medicines bring a better quality of life to patients, the access to this medicines has become limited and challenging, mainly explained by their high cost. The lapsing of several patents for biological medicines allowed the arrival of biosimilar medicines, which came to bring financial savings to health systems, and to biopharmaceutical industries the challenge of proving biosimilarity with the reference biological medicine. With the introduction of biosimilar medicines in the biopharmaceutical panorama, other medicines started to appear, such as biobetter medicines, non-original biologics medicines and stand-alone medicines. Although these medicines address market needs, it has become clear that the regulatory base and terminology in the world of biologic medicines has become complex and globally inaccurate. The entry of medicines with uncertain safety, quality and efficacy profiles in the biopharmaceutical market may become a reality and a risk that could be prevented through regulatory harmonization between regulatory agencies. The arise of concepts such as interchangeability, switch, automatic substitution and data extrapolation are extremely relevant since their definitions are not consensual among regulatory agencies, leading to regulatory divergences in various regions of the planet. In the future, the biopharmaceutical market will continue to develop and grow, meaning that a confluence of efforts for greater cohesion at a regulatory and terminology level is imperative.
Description
Trabalho Final de Mestrado Integrado, Ciências Farmacêuticas, 2020, Universidade de Lisboa, Faculdade de Farmácia.
Keywords
Medicamentos biológicos Medicamentos biossimilares Medicamentos biossuperiores Regulamentação Harmonização Mestrado integrado - 2020