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Fostering therapeutic innovation: the role of PRIority MEdicines (PRIME) scheme, breakthrough therapy and sakigake designation in innovative drug development
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A crescente complexidade da inovação farmacêutica e a urgência em dar resposta a desafios de saúde pública conduziram à implementação de vias regulamentares expeditas destinadas a acelerar a aprovação de medicamentos essenciais. Este estudo centra-se particularmente no PRIiority Medicines (PRIME) scheme da European Medicines Agency (EMA), na Breakthought Therapy Designation (BTD) da Food and Drug Administration (FDA) e no Sakigake Designation System da Pharmaceuticals and Medical Devices Agency (PMDA). O estudo avalia cada via regulamentar acelerada em termos das suas caraterísticas, objetivos, critérios de elegibilidade e resultados. Além disso, é feita uma comparação entre estes medicamentos aprovados por via rápida e os aprovados através de outros mecanismos regulamentares em cada região, excluindo medicamentos genéricos, para determinar se atingem com êxito os objetivos pretendidos. Adicionalmente, este estudo procurou comparar o desempenho destas vias rápidas em diferentes regiões. Foi utilizada uma abordagem de métodos mistos, combinando a análise de dados quantitativos das taxas de aprovação, áreas terapêuticas e tipos de medicamentos (tais como medicamentos biológicos e medicamentos órfãos) recolhidos do sítio Web e dos relatórios de cada agência reguladora, com conhecimentos qualitativos retirados de revisões da literatura e estudos de caso. Considerando as datas de implementação dos programas em estudo, os horizontes temporais diferem – de 2016 a 2023 para o PRIME Scheme, de 2015 a 2023 para o Breakthrough Therapy Designation e de 2014 a 2023 para o Sakigake Designation System. A comparação entre estes programas é ajustada a um período sobreposto de 2016 a 2023. Os resultados indicam que estes quadros regulamentares expeditos promovem a inovação, sendo a oncologia a área terapêutica dominante em todas as regiões, e os medicamentos biológicos e órfãos constituem uma proporção significativa das aprovações. A análise revela que estas vias expeditas reduzem com êxito os prazos de avaliação. A importância desta investigação reside no seu contributo para o debate em curso sobre o equilíbrio entre a necessidade de inovação rápida e a supervisão regulamentar.
The increasing complexity of pharmaceutical innovation and the urgency to address public health challenges have driven the establishment of expedited regulatory pathways aimed at accelerating the approval of life-saving medicines. This study focuses particularly on the European Medicines Agency's (EMA) PRIiority Medicines (PRIME) scheme, the Food and Drug Administration's (FDA) Breakthrough Therapy Designation (BTD) and the Pharmaceuticals and Medical Devices Agency’s (PMDA) Sakigake Designation System. It aims to assess each expedited regulatory pathway in terms of their features, objectives, eligibility criteria, and outcomes. Moreover, a comparison is made between these expedited-approved medicines with ones approved through other regulatory mechanisms in each region, excluding generic medicinal products, to determine if they successfully achieve their intended objectives. Additionally, this study sought to compare the performance of these expedited pathways across different regions. A mixed-methods approach was employed, combining quantitative data analysis of approval rates, therapeutic areas, and drug types (such as biologic and orphan medicinal products) collected from each regulatory agency’s website and reports, with qualitative insights drawn from literature reviews and case studies. Considering the implementation dates of the programs under study, the time horizons differ – from 2016 to 2023 for the PRIME Scheme, from 2015 to 2023 for the Breakthrough Therapy Designation and from 2014 to 2023 for the Sakigake Designation System. The comparison between these programs is adjusted to an overlapping timeframe from 2016 to 2023. The results indicate that these expedited regulatory frameworks foster innovation, with oncology being the dominant therapeutic area across all regions, and biologics and orphan drugs constituting a significant proportion of approvals. The analysis reveals that these expedited pathways successfully reduce evaluation timelines. The significance of this research lies in its contribution to the ongoing discussion about balancing the need for rapid innovation with regulatory oversight.
The increasing complexity of pharmaceutical innovation and the urgency to address public health challenges have driven the establishment of expedited regulatory pathways aimed at accelerating the approval of life-saving medicines. This study focuses particularly on the European Medicines Agency's (EMA) PRIiority Medicines (PRIME) scheme, the Food and Drug Administration's (FDA) Breakthrough Therapy Designation (BTD) and the Pharmaceuticals and Medical Devices Agency’s (PMDA) Sakigake Designation System. It aims to assess each expedited regulatory pathway in terms of their features, objectives, eligibility criteria, and outcomes. Moreover, a comparison is made between these expedited-approved medicines with ones approved through other regulatory mechanisms in each region, excluding generic medicinal products, to determine if they successfully achieve their intended objectives. Additionally, this study sought to compare the performance of these expedited pathways across different regions. A mixed-methods approach was employed, combining quantitative data analysis of approval rates, therapeutic areas, and drug types (such as biologic and orphan medicinal products) collected from each regulatory agency’s website and reports, with qualitative insights drawn from literature reviews and case studies. Considering the implementation dates of the programs under study, the time horizons differ – from 2016 to 2023 for the PRIME Scheme, from 2015 to 2023 for the Breakthrough Therapy Designation and from 2014 to 2023 for the Sakigake Designation System. The comparison between these programs is adjusted to an overlapping timeframe from 2016 to 2023. The results indicate that these expedited regulatory frameworks foster innovation, with oncology being the dominant therapeutic area across all regions, and biologics and orphan drugs constituting a significant proportion of approvals. The analysis reveals that these expedited pathways successfully reduce evaluation timelines. The significance of this research lies in its contribution to the ongoing discussion about balancing the need for rapid innovation with regulatory oversight.
Descrição
Tese de mestrado, Regulação e Avaliação do Medicamento e Produtos de Saúde, 2025, Universidade de Lisboa, Faculdade de Farmácia.
Palavras-chave
Expedited regulatory pathways Innovative medicines PRIME scheme Breakthrough therapy designation Sakigake designation Teses de mestrado - 2025