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Publication
Impacto regulamentar no desenvolvimento e acesso de medicamentos para a atrofia muscular espinhal
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Abstract(s)
A Atrofia Muscular Espinhal (AME) é uma doença neuromuscular rara de etiologia
genética, caraterizada pela perda progressiva da função muscular. Apesar dos avanços
científicos e esforços que se concretizaram ao nível da investigação e desenvolvimento
de novos medicamentos, no sentido de reverter, atrasar e/ou interromper o declínio
progressivo da função motora e da incapacidade, verificam-se, ainda, inúmeras
necessidades médicas insatisfeitas. Desta forma, considera-se impreterível a existência de
padrões regulamentares para avaliar a eficácia e a segurança de um tratamento prospetivo,
estabelecer a relação benefício-risco em grupos de doentes vulneráveis e acelerar o acesso
ao mercado de novos medicamentos promissores com indicações terapêuticas para a
doença. Neste âmbito, em 1983 e 2000, foi promulgada nos Estados Unidos da América
(EUA) e na União Europeia (UE), respetivamente, legislação destinada à regulação de
medicamentos órfãos, estabelecendo políticas de desenvolvimento e de investigação de
medicamentos órfãos através da criação de mecanismos de incentivos e ferramentas
regulamentares.
Não obstante, apesar do considerável progresso terapêutico observado, no que
respeita ao número de medicamentos disponíveis no mercado, é impostergável contínuos
investimentos nos processos de investigação e desenvolvimento e uma estreita
colaboração de intercâmbio de informação entre os diferentes intervenientes no processo,
particularmente entre indústria farmacêutica, agências reguladoras, doentes, comunidade
académica e científica.
Esta dissertação analisa o quadro regulamentar dos medicamentos órfãos na UE e
EUA, mecanismos de incentivos, ferramentas regulamentares e impacto regulamentar no
desenvolvimento e acesso de medicamentos para a AME. Por fim, será abordada a
avaliação da relação benefício-risco de dois medicamentos com indicações para a AME
e o seu impacto económico.
Spinal muscular atrophy (SMA) is a rare genetic neuromuscular disease, characterized by the progressive loss of muscular function. Despite scientific efforts done in research and development of new medicines, to slow down, interrupt and even revert the progressive decline of motor function, there are still several unmet medical needs. Thus, is considered indispensable the existence of regulatory standards to evaluate the effectiveness and safety during prospective treatments, to establish risk-benefit relations within groups of vulnerable patients and, to accelerate access to market of new promising medicines with therapeutic indication for SMA. In that context, in 1983 and 2000, it was enacted in the United States of America (USA) and European Union (EU), respectively, legislation with the purpose to regulate orphan medicines, establish politics to stimulate research and development by the creation of incentives and regulatory framework. Despite the considerable therapeutical efforts in terms of medicines available in the market, it is peremptory to continue investing in research and development, establish close partnership relations across the all participants in the process, such as scientific community, pharmaceutical industry, regulatory agencies and patients. This thesis analyses the regulatory framework for orphan medicines in the EU and USA, incentive mechanisms, regulatory tools and the impact of those policies for the development and access of medicines for SMA. Lastly, is made an evaluation of the benefit-risk relation for two medicines indicated for SMA and their economic impact.
Spinal muscular atrophy (SMA) is a rare genetic neuromuscular disease, characterized by the progressive loss of muscular function. Despite scientific efforts done in research and development of new medicines, to slow down, interrupt and even revert the progressive decline of motor function, there are still several unmet medical needs. Thus, is considered indispensable the existence of regulatory standards to evaluate the effectiveness and safety during prospective treatments, to establish risk-benefit relations within groups of vulnerable patients and, to accelerate access to market of new promising medicines with therapeutic indication for SMA. In that context, in 1983 and 2000, it was enacted in the United States of America (USA) and European Union (EU), respectively, legislation with the purpose to regulate orphan medicines, establish politics to stimulate research and development by the creation of incentives and regulatory framework. Despite the considerable therapeutical efforts in terms of medicines available in the market, it is peremptory to continue investing in research and development, establish close partnership relations across the all participants in the process, such as scientific community, pharmaceutical industry, regulatory agencies and patients. This thesis analyses the regulatory framework for orphan medicines in the EU and USA, incentive mechanisms, regulatory tools and the impact of those policies for the development and access of medicines for SMA. Lastly, is made an evaluation of the benefit-risk relation for two medicines indicated for SMA and their economic impact.
Description
Tese de mestrado, Regulação e Avaliação de Medicamentos e Produtos de Saúde, 2021, Universidade de Lisboa, Faculdade de Farmácia.
Keywords
Atrofia muscular espinhal Medicamentos órfãos Assuntos regulamentares Nusinersen and onasemnogene abeparvovec Teses de mestrado - 2021