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Methodologies on collection and use of patient experience data for medicines’ regulatory assessment
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A perspetiva dos doentes é considerada cada vez mais importante nas tomadas de decisão ao longo do ciclo de vida do medicamento. Deste modo, tem-se vindo a investigar as melhores abordagens para recolher dados da experiência de doentes para informar processos de tomada de decisão que sejam centrados nos mesmos.
O presente estudo tem como objetivo apresentar uma visão geral de duas metodologias usadas na recolha de dados da experiência dos doentes: resultados reportados pelos doentes e preferências dos doentes (das expressões originais na língua inglesa “patient-reported outcomes” e “patient preferences”, respetivamente). No contexto deste objetivo, este estudo propõe-se ainda a apresentar um estudo de caso sobre o uso destas metodologias no mieloma múltiplo com o intuito de discutir exemplos reais da sua contribuição para melhor informar os processos de tomada de decisão.
Uma pesquisa bibliográfica foi realizada na base de dados PubMed em dezembro de 2021 e posteriormente em agosto de 2022 para identificar artigos que abordassem o uso de dados da experiência dos doentes, resultados reportados pelos doentes e preferências dos doentes ao longo do ciclo de vida do medicamento. Adicionalmente, foi realizada uma pesquisa no motor de busca Google entre janeiro e agosto de 2022 para identificar literatura cinzenta relevante. Posteriormente, os documentos identificados foram selecionados com base nos critérios de seleção. Finalmente, a bibliografia dos artigos selecionados foi revista para identificar publicações adicionais.
Tomando como ponto de partida uma breve apresentação sobre dados da experiência dos doentes, este trabalho discute a utilização de dados reportados pelos doentes e de preferências dos doentes, descrevendo os seus potenciais papéis ao longo do ciclo de vida do medicamento, principais aspetos metodológicos, desafios, iniciativas recentes e recomendações, conforme descrito na literatura.
As referidas metodologias têm o potencial de informar várias etapas do ciclo de vida do medicamento. No entanto, existem ainda alguns desafios por ultrapassar. É impreterível fomentar a colaboração internacional e desenvolver orientações claras e harmonizadas. Só assim será possível incluir eficaz e sistematicamente a voz dos doentes no desenvolvimento de medicamentos e na prestação de cuidados de saúde que verdadeiramente valorizem as suas necessidades, prioridades e valores.
Patients’ perspective has become increasingly important in decision-making throughout the medical product life cycle. Therefore, multiple stakeholders are investigating the best approaches to collect patient experience data to inform patient-centric decision-making processes. The objective of the present study is to provide an overview of the landscape regarding two methodologies used for the collection of patient experience data: patient-reported outcomes and patient preferences. Drawing on the knowledge from this overview, an additional objective of this study is to present a case study on the use of patient-reported outcomes and patient preferences in multiple myeloma to discuss real examples of the contribution of these methods for better informing decision-making. A literature search was conducted on PubMed in December 2021 and August 2022 to identify articles addressing the use of patient experience data, patient-reported outcomes, and patient preferences in the context of decision-making throughout the medical product life cycle. Moreover, a Google search was conducted between January 2022 and August 2022 to identify grey literature relevant to the research objectives. Identified documents were screened in a two-step approach based on the selection criteria. Finally, references in selected articles were hand-searched to identify additional publications. Taking as a starting point a brief discussion of patient experience data, this work discusses PROs and patient preferences input over recent years by describing their potential roles throughout the medicines’ life cycle, main methodological aspects, challenges, ongoing efforts and initiatives to advance research and implementation, and key recommendations for future research, as reported by the literature. The increased focus on patient engagement has been proved by the growing literature and initiatives on this subject. In fact, patient-reported outcomes and patient preferences have the potential to inform multiple key steps of the medical product life cycle. However, these methodologies face some challenges that have yet to be addressed. It is vitally important to foster international multistakeholder collaboration and develop clear and harmonized guidance for the use of patient-reported outcomes and patient preferences. Once this is achieved, it is expected that systematic and effective collection, analyses, and sharing of patient input will maximise its utility and greatly improve patients’ lives.
Patients’ perspective has become increasingly important in decision-making throughout the medical product life cycle. Therefore, multiple stakeholders are investigating the best approaches to collect patient experience data to inform patient-centric decision-making processes. The objective of the present study is to provide an overview of the landscape regarding two methodologies used for the collection of patient experience data: patient-reported outcomes and patient preferences. Drawing on the knowledge from this overview, an additional objective of this study is to present a case study on the use of patient-reported outcomes and patient preferences in multiple myeloma to discuss real examples of the contribution of these methods for better informing decision-making. A literature search was conducted on PubMed in December 2021 and August 2022 to identify articles addressing the use of patient experience data, patient-reported outcomes, and patient preferences in the context of decision-making throughout the medical product life cycle. Moreover, a Google search was conducted between January 2022 and August 2022 to identify grey literature relevant to the research objectives. Identified documents were screened in a two-step approach based on the selection criteria. Finally, references in selected articles were hand-searched to identify additional publications. Taking as a starting point a brief discussion of patient experience data, this work discusses PROs and patient preferences input over recent years by describing their potential roles throughout the medicines’ life cycle, main methodological aspects, challenges, ongoing efforts and initiatives to advance research and implementation, and key recommendations for future research, as reported by the literature. The increased focus on patient engagement has been proved by the growing literature and initiatives on this subject. In fact, patient-reported outcomes and patient preferences have the potential to inform multiple key steps of the medical product life cycle. However, these methodologies face some challenges that have yet to be addressed. It is vitally important to foster international multistakeholder collaboration and develop clear and harmonized guidance for the use of patient-reported outcomes and patient preferences. Once this is achieved, it is expected that systematic and effective collection, analyses, and sharing of patient input will maximise its utility and greatly improve patients’ lives.
Descrição
Trabalho Final de Mestrado Integrado, Ciências Farmacêuticas, 2022, Universidade de Lisboa, Faculdade de Farmácia.
Palavras-chave
Patient experience data Patient-reported outcomes Patient preferences Medical product life cycle Multiple myeloma Mestrado integrado - 2022