Medicamentos biológicos com indicações órfãs
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2021
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O mundo em constante mudança levou-nos e leva-nos cada vez mais a alcançar avanços científicos e tecnológicos importantes como foi o caso do desenvolvimento dos medicamentos biológicos. A chegada desta nova classe de medicamentos revolucionou a indústria farmacêutica e transformou a vida dos doentes em todo o mundo, com especial atenção para aqueles que vivem com doenças raras e para os quais o tratamento é por vezes negligenciado. A pertinência desta temática é abordada nesta tese, que tem como principal objetivo a análise observacional e retrospetiva da informação referente aos medicamentos biológicos de uso humano com indicações órfãs com autorização de introdução no mercado europeu e/ou americano. Os dados recolhidos para analise dos dados europeus foram acedidos através do site oficial da Agência Europeia do Medicamento no período de 1995 a dezembro de 2020 e referentes aos medicamentos para uso humano aprovados por procedimento centralizado de AIM, enquanto os dados referentes ao mercado Americano foram obtidos do site oficial da Food and Drug Administration (FDA), na secção dos medicamentos órfãos. Após análise dos dados, verificou-se que a aprovação de medicamentos biológicos com órfãos tem aumentado ao longo dos anos, com os EUA a apresentarem maior número de medicamentos biológicos órfãos aprovados (74 medicamentos) comparativamente com a União Europeia (48 medicamentos). Este facto pode dever-se ao aumento do conhecimento científico, molecular e genético da própria patofisiologia das doenças raras. Constatou-se ainda que as áreas da oncologia, hematologia e metabolismo e endocrinologia são as áreas com maior número de medicamentos biológicos indicados em doenças raras, representando mais de 70% do total de medicamentos biológicos órfãos com AIM na EU e nos EUA. Além disso, as terapias baseadas em proteínas recombinantes representam mais de 50% do total de biológicos órfãos aprovados nas duas regiões, sendo os anticorpos monoclonais o tipo de medicamento biotecnológico com maior relevo em ambos os mercados. Quanto aos medicamentos de terapia avançada, verificou-se que a União Europeia tinha até ao final de 2020 1 medicamento biológico órfão de terapia celular, 1 medicamento biológico órfão de engenharia de tecidos e 6 medicamentos biológicos órfãos de terapia génica com AIM aprovadas, enquanto que EUA tinham apenas 6 medicamentos biológicos órfãos de terapia génica. Esta classe de medicamentos tem cada vez mais importância para o tratamento de doenças raras e espera-se que com o aumento da inovação biotecnológica, estes ganhem cada vez mais percentagem de mercado no futuro. No entanto, o caminho para se alcançar o tratamento para todas as doenças raras existentes ainda é muito longo uma vez que existem medicamentos órfãos aprovados para apenas 5% dessas doenças, o que revela uma clara necessidade clínica não satisfeita (unmet medical need). Desta forma, a esperança e expetativas na procura de soluções de vanguarda que permitam proporcionar uma melhor qualidade de vida a estes doentes e solucionar este unmet medical need deve passar pela conjugação de esforços por parte dos governos dos principais países mundiais, em conjunto com empresas farmacêuticas e biotecnológicas no desenvolvimento de métodos de diagnostico e medicamentos órfãos, que permitam uma abordagem terapêutica inovadora, promissora e diferenciada, a custos acessíveis a todos os doentes.
The ever-changing world has led us to achieve significant scientific and technological advances, as was the case with the development of biological medicines. The arrival of this new class of drugs has revolutionized the pharmaceutical industry and transformed the lives of patients worldwide, with particular attention to those living with rare diseases for whom treatment is sometimes neglected. The relevance of this theme is addressed in this thesis, in which the main objective is the observational and retrospective analysis of information regarding biological medicines for human use with orphan indications with European and/or American marketing authorization. The European data collected were accessed through the official website of the European Medicines Agency for the period 1995 to December 2020 and pertaining to medicines for human use approved by centralized MA procedure, while the data related to the American market were obtained from the official website of the Food and Drug Administration (FDA), in the orphan drugs section. After analyzing the data, it was found that the approval of biological medicines with orphans has increased over the years, with the US having a higher number of approved biological orphan medicines (74 medicines) compared to the European Union (48 medicines). This may be due to increased scientific, molecular, and genetic understanding of the pathophysiology of rare diseases themselves. It was also found that oncology, hematology and metabolism, and endocrinology are the areas with the highest number of indicated biological medicines in rare diseases, accounting for more than 70% of the total number of orphan biological medicines with MAAs. Also, recombinant protein-based therapies account for more than 50% of the total biologics orphan approved in both regions, with monoclonal antibodies being the most prominent type of biotech medicines in both markets. As for advanced therapy medicinal products, it was found that by the end of 2020 the European Union had 1 biological orphan cell therapy, 1 biological orphan tissue engineering and 6 biological orphan gene therapy medicines with approved MAs, while the US had only 6 biological orphan gene therapy medicines. This class of medicines is essential for the treatment of rare diseases, and it is expected that with increased biotech innovation, they will gain more and more market share in the future. However, the road to achieving treatment for all existing rare diseases is still very long since the approved orphan medicines only treat 5% of such diseases, revealing a clear unmet medical need. Thus, the hope and expectations at the forefront of the search for solutions that will provide the best quality of life for those patients, and solve this unmet medical need, must involve the joining efforts of governments around the world and pharmaceuticals biotechnology companies. This will allow the development of diagnostic methods and medicines for innovative, promising, and differentiated therapeutic approaches at costs accessible to all patients.
The ever-changing world has led us to achieve significant scientific and technological advances, as was the case with the development of biological medicines. The arrival of this new class of drugs has revolutionized the pharmaceutical industry and transformed the lives of patients worldwide, with particular attention to those living with rare diseases for whom treatment is sometimes neglected. The relevance of this theme is addressed in this thesis, in which the main objective is the observational and retrospective analysis of information regarding biological medicines for human use with orphan indications with European and/or American marketing authorization. The European data collected were accessed through the official website of the European Medicines Agency for the period 1995 to December 2020 and pertaining to medicines for human use approved by centralized MA procedure, while the data related to the American market were obtained from the official website of the Food and Drug Administration (FDA), in the orphan drugs section. After analyzing the data, it was found that the approval of biological medicines with orphans has increased over the years, with the US having a higher number of approved biological orphan medicines (74 medicines) compared to the European Union (48 medicines). This may be due to increased scientific, molecular, and genetic understanding of the pathophysiology of rare diseases themselves. It was also found that oncology, hematology and metabolism, and endocrinology are the areas with the highest number of indicated biological medicines in rare diseases, accounting for more than 70% of the total number of orphan biological medicines with MAAs. Also, recombinant protein-based therapies account for more than 50% of the total biologics orphan approved in both regions, with monoclonal antibodies being the most prominent type of biotech medicines in both markets. As for advanced therapy medicinal products, it was found that by the end of 2020 the European Union had 1 biological orphan cell therapy, 1 biological orphan tissue engineering and 6 biological orphan gene therapy medicines with approved MAs, while the US had only 6 biological orphan gene therapy medicines. This class of medicines is essential for the treatment of rare diseases, and it is expected that with increased biotech innovation, they will gain more and more market share in the future. However, the road to achieving treatment for all existing rare diseases is still very long since the approved orphan medicines only treat 5% of such diseases, revealing a clear unmet medical need. Thus, the hope and expectations at the forefront of the search for solutions that will provide the best quality of life for those patients, and solve this unmet medical need, must involve the joining efforts of governments around the world and pharmaceuticals biotechnology companies. This will allow the development of diagnostic methods and medicines for innovative, promising, and differentiated therapeutic approaches at costs accessible to all patients.
Descrição
Orientação: Ana Sofia Fernandes
Palavras-chave
MESTRADO INTEGRADO EM CIÊNCIAS FARMACÊUTICAS, CIÊNCIAS FARMACÊUTICAS, UNIÃO EUROPEIA, EUROPEAN UNION, MEDICAMENTOS BIOLÓGICOS, DOENÇAS RARAS, PHARMACEUTICAL SCIENCES, BIOLOGICAL DRUGS, RARE DISEASES, ESTADOS UNIDOS DA AMÉRICA, UNITED STATES OF AMERICA, IMPLANTES LIPÍDICOS, MEDICAMENTOS ÓRFÃOS, LIPIDIC IMPLANTS, ORPHAN DRUGS, DMARD