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Abstract(s)
Drug repurposing consiste em encontrar novas indicações para medicamentos existentes fora das suas indicações iniciais. Esta estratégia, ligada ao conceito de polifarmacologia, tem como objetivo aumentar a eficácia terapêutica direcionada a vários tipos de doenças. A abordagem oferece vantagens significativas, como custos de desenvolvimento reduzidos e riscos de insucesso inferiores. Exemplos históricos, como a talidomida e o sildenafil, evidenciam o seu potencial. Além disso, a pandemia de COVID-19 sublinhou a sua importância, invertendo as tendências típicas a nível de apoios financeiros para ensaios clínicos. Apesar das suas vantagens, barreiras regulamentares, incluindo a falta de interesse das empresas farmacêuticas, o acesso limitado aos dados e quadros regulamentares insuficientes para os investigadores não pertencentes à indústria persistem. Instituições académicas e organizações de doentes, apesar do seu interesse para a saúde pública, carecem da infraestrutura e dos conhecimentos necessários para a conformidade regulamentar. Recomendações incluem a criação de orientações regulamentares, a melhoria da acessibilidade dos dados, o desenvolvimento de manuais regulamentares, o incentivo ao feedback regulamentar antecipado e o reforço do papel das agências regulamentares no apoio a novas indicações. Incentivos como a proteção de dados, vales de revisão prioritária e fundos de investigação também podem motivar o investimento da indústria na reorientação de medicamentos. A colaboração entre investigadores académicos e a indústria farmacêutica é crucial para fazer o desenvolvimento de iniciativas. Na Europa, o Regulamento 726/2004 oferece uma lei de exclusividade "8+2(+1)", enquanto os EUA oferecem uma proteção de três anos para novas indicações sem necessidade de provar um benefício clínico significativo. Ambas as regiões oferecem incentivos, como o sistema PRIME da UE e a via 505(b)(2) dos EUA, que facilitam a aprovação de medicamentos. Ainda assim, a escassez de incentivos impedem que as empresas farmacêuticas procurem novas indicações. Esforços para colaborações e melhorias nas vias regulamentares são cruciais para ultrapassar estas barreiras e fazer avançar o processo. Na Europa, o drug repurposing é facilitado através de várias vias regulamentares, incluindo a utilização off-label e on-label. DRUP, REMEDi4ALL e REPO4EU são iniciativas europeias com o objetivo de promover o drug repurposing através de colaborações, plataformas inovadoras e apoio regulamentar. Para além disso, a Associação Internacional de Sociedades de Benefício Mútuo defende modelos de fair price para melhorar a acessibilidade dos medicamentos.
Drug repurposing involves finding new uses for existing drugs beyond their original indications. This strategy, linked to the concept of polypharmacology, aims to enhance therapeutic efficacy by targeting multiple disease pathways. The approach offers significant benefits, including reduced development costs, shorter timelines, and lower failure risks. Historical examples, like thalidomide and sildenafil, highlight its potential. Furthermore, the COVID-19 pandemic underscored its importance, reversing typical clinical trial sponsorship trends. Despite its advantages, challenges such as regulatory barriers persist, including a lack of interest from pharmaceutical companies, limited access to data, and insufficient regulatory frameworks for non-industry researchers. Academic institutions and patient organizations, despite their public health interest, often lack the necessary infrastructure and expertise for regulatory compliance. Solutions include creating regulatory guidance, improving data accessibility, developing regulatory handbooks, encouraging early regulatory feedback, and strengthening regulatory agencies' roles in supporting new indications. Incentives such as extended data protection, priority review vouchers, and research funds could also motivate industry investment in drug repurposing. Collaboration between academic researchers and the pharmaceutical industry is crucial for advancing drug repurposing initiatives. In Europe, Regulation 726/2004 offers an "8+2(+1)" exclusivity formula, while the US provides a three- year protection for new indications without needing to prove significant clinical benefit. Both regions offer incentives like the EU's PRIME scheme and the US's 505(b)(2) pathway to facilitate drug approval. These frameworks aim to balance innovation and market protection, though enforcement issues and insufficient incentives often deter pharmaceutical companies from pursuing new indications. Collaborative efforts and improved regulatory pathways are crucial to overcoming these barriers and advancing drug repurposing. In Europe, drug repurposing is facilitated through various regulatory pathways, including off-label and on-label use. Key European initiatives include the DRUP study, REMEDi4ALL, and REPO4EU, which aim to advance drug repurposing through collaborative efforts, innovative platforms, and regulatory support. Additionally, the International Association of Mutual Benefit Societies (AIM) advocates for fair pricing models to enhance access and affordability for repurposed medicines.
Drug repurposing involves finding new uses for existing drugs beyond their original indications. This strategy, linked to the concept of polypharmacology, aims to enhance therapeutic efficacy by targeting multiple disease pathways. The approach offers significant benefits, including reduced development costs, shorter timelines, and lower failure risks. Historical examples, like thalidomide and sildenafil, highlight its potential. Furthermore, the COVID-19 pandemic underscored its importance, reversing typical clinical trial sponsorship trends. Despite its advantages, challenges such as regulatory barriers persist, including a lack of interest from pharmaceutical companies, limited access to data, and insufficient regulatory frameworks for non-industry researchers. Academic institutions and patient organizations, despite their public health interest, often lack the necessary infrastructure and expertise for regulatory compliance. Solutions include creating regulatory guidance, improving data accessibility, developing regulatory handbooks, encouraging early regulatory feedback, and strengthening regulatory agencies' roles in supporting new indications. Incentives such as extended data protection, priority review vouchers, and research funds could also motivate industry investment in drug repurposing. Collaboration between academic researchers and the pharmaceutical industry is crucial for advancing drug repurposing initiatives. In Europe, Regulation 726/2004 offers an "8+2(+1)" exclusivity formula, while the US provides a three- year protection for new indications without needing to prove significant clinical benefit. Both regions offer incentives like the EU's PRIME scheme and the US's 505(b)(2) pathway to facilitate drug approval. These frameworks aim to balance innovation and market protection, though enforcement issues and insufficient incentives often deter pharmaceutical companies from pursuing new indications. Collaborative efforts and improved regulatory pathways are crucial to overcoming these barriers and advancing drug repurposing. In Europe, drug repurposing is facilitated through various regulatory pathways, including off-label and on-label use. Key European initiatives include the DRUP study, REMEDi4ALL, and REPO4EU, which aim to advance drug repurposing through collaborative efforts, innovative platforms, and regulatory support. Additionally, the International Association of Mutual Benefit Societies (AIM) advocates for fair pricing models to enhance access and affordability for repurposed medicines.
Description
Trabalho Final de Mestrado Integrado, Ciências Farmacêuticas, 2024, Universidade de Lisboa, Faculdade de Farmácia
Keywords
Drug repurposing Regulatory framework Europe Mestrado Integrado - 2024