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Orientador(es)
Resumo(s)
Ainda subsiste muita incerteza sobre a melhor maneira de utilizar novas terapias
imunomoduladoras em doentes oncológicos, especialmente em doentes com cancro do pulmão.
A prevalência global desta doença, a mortalidade e o mau prognóstico a ela associados
justificam a necessidade de um rápido desenvolvimento e aplicação de novas terapias, capazes
de mudar o atual paradigma complicado da doença. Dado que as imunoterapias, nomeadamente
os inibidores de pontos de controlo imunitário (ICI), têm produzido resultados melhorados em
doentes com cancro do pulmão, mas exigem despesas elevadas para as suas aquisições, este
estudo procura sistematizar os dados atualmente publicados sobre ICIs para doentes com cancro
do pulmão, de modo a ajudar a clarificar os métodos atuais mais custo-efetivo para as aplicar e
a evidência que ainda falta ser gerada. As combinações com estas terapias possuem o potencial
de melhorar a eficácia dos regimes, através do uso de mecanismos de ação sinérgicos, mas
também representam riscos de eventos adversos agravados e custos elevados, sendo o foco do
presente estudo.
Foi construída uma revisão sistemática de estudos disponíveis que analisavam a eficácia,
a relação custo-eficácia ou o custo-utilidade dos ICI no cancro do pulmão. Foram analisados
175 estudos através do respetivo resumo, tendo sido selecionados 85 para análise integral e, dos
quais, 43 foram selecionados para inclusão no presente trabalho.
Os estudos incluídos analisaram regimes variados com ICIs em monoterapia ou em
combinação, quer entre ICIs quer com outras opções como a quimioterapia ou o bevacizumab.
Embora a maioria dos regimes tenha apresentado resultados significativamente melhores em
comparação com a quimioterapia clássica, apenas o Pembrolizumab e o Atezolizumab em
monoterapia foram considerados custo-efetivos, em grupos de doentes selecionados por
biomarcadores. Os preços elevados com que estes medicamentos entram no mercado foram
considerados como a maior barreira à utilização generalizada dos ICI. Este estudo sugere a
investigação de uma melhor utilização dos biomarcadores atuais ou estratégias de partilha de
custos e riscos para contrariar esta dificuldade, permitindo, potencialmente, que os sistemas de
cuidados de saúde possam adquirir de forma sustentável estas novas opções terapêuticas e
utilizá-las nos subgrupos de doentes oncológicos que mais beneficiam com as mesmas, ao
mesmo tempo que apoiam e incentivam a inovação.
Much uncertainty remains on how to best use novel immunomodulatory therapies in oncologic patients, especially lung cancer patients. This disease’s global prevalence, mortality and poor prognosis warrant fast development and application of novel therapies, capable of changing the current disease’s grave paradigm. As immunotherapies, namely immune checkpoint inhibitors (ICIs), have yielded improved outcomes for lung cancer patients, but demand high expenditures for their purchases, this study looks to systemize currently published data on ICIs for lung cancer patients, to help clarify the evidence that’s still lacking and the current most cost-effective methods to apply such therapies. As combinations with these options hold the potential of improving effectiveness, through the enhanced benefits of synergistic mechanisms of action, but also aggravated adverse events and high costs, these regimens were the focus of the present study. A systematic literature review of studies analysing effectiveness, cost-effectiveness or cost-utility of ICIs in lung cancer was constructed. 175 studies were abstract screened, with 85 being selected for integral analysis and, from those, 43 being selected for inclusion in the present review. Varied regimens with ICIs in monotherapy or in combination, either between ICIs or with other options like chemotherapy or bevacizumab, were analysed in the included studies. Although most regimens presented significantly improved outcomes in comparison to classical chemotherapy, only Pembrolizumab and Atezolizumab in monotherapy were found to be costeffective, in biomarker-selected groups of patients. The high prices at which these medicines enter the market were found to be the biggest barrier to ICI’s generalized use in lung cancer patients. Investigation of new ways to use current biomarkers for ICIs or cost-sharing and risksharing strategies were suggested to balance this issue, potentially allowing healthcare systems to sustainably imburse the use of these novel options in oncologic patients’ subgroups that most benefit from them, while still supporting and incentivising innovation.
Much uncertainty remains on how to best use novel immunomodulatory therapies in oncologic patients, especially lung cancer patients. This disease’s global prevalence, mortality and poor prognosis warrant fast development and application of novel therapies, capable of changing the current disease’s grave paradigm. As immunotherapies, namely immune checkpoint inhibitors (ICIs), have yielded improved outcomes for lung cancer patients, but demand high expenditures for their purchases, this study looks to systemize currently published data on ICIs for lung cancer patients, to help clarify the evidence that’s still lacking and the current most cost-effective methods to apply such therapies. As combinations with these options hold the potential of improving effectiveness, through the enhanced benefits of synergistic mechanisms of action, but also aggravated adverse events and high costs, these regimens were the focus of the present study. A systematic literature review of studies analysing effectiveness, cost-effectiveness or cost-utility of ICIs in lung cancer was constructed. 175 studies were abstract screened, with 85 being selected for integral analysis and, from those, 43 being selected for inclusion in the present review. Varied regimens with ICIs in monotherapy or in combination, either between ICIs or with other options like chemotherapy or bevacizumab, were analysed in the included studies. Although most regimens presented significantly improved outcomes in comparison to classical chemotherapy, only Pembrolizumab and Atezolizumab in monotherapy were found to be costeffective, in biomarker-selected groups of patients. The high prices at which these medicines enter the market were found to be the biggest barrier to ICI’s generalized use in lung cancer patients. Investigation of new ways to use current biomarkers for ICIs or cost-sharing and risksharing strategies were suggested to balance this issue, potentially allowing healthcare systems to sustainably imburse the use of these novel options in oncologic patients’ subgroups that most benefit from them, while still supporting and incentivising innovation.
Descrição
Trabalho Final de Mestrado Integrado, Ciências Farmacêuticas, 2021, Universidade de Lisboa, Faculdade de Farmácia.
Palavras-chave
Lung Cancer Immunotherapy Immunomodulatory Cost-effectiveness Mestrado integrado - 2021
