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Acesso a medicamentos em Portugal através de programas de acesso precoce e autorização de utilização excecional
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Abstract(s)
Introdução: O acesso a medicamentos representa um desafio significativo de saúde pública, particularmente no contexto de doenças graves e/ou crónicas em que não existem alternativas terapêuticas. Em Portugal, os Programas de Acesso Precoce (PAP) e as Autorizações de Utilização Excecional (AUE) constituem mecanismos que possibilitam o acesso a medicamentos antes de uma decisão de financiamento público,
desde que seja demonstrada a inexistência de alternativa terapêutica em que o doente corra o risco imediato de vida ou de sofrer complicações graves.
Objetivos: O objetivo principal deste estudo é caracterizar o acesso a medicamentos em Portugal através de PAPs, entre 1 de janeiro de 2015 e 20 de julho de 2024. Adicionalmente, o estudo pretende descrever os procedimentos e a legislação atualmente em vigor dos PAPs e AUEs, realizar uma análise qualitativa e quantitativa dos medicamentos aprovados por procedimento centralizado pela Agência Europeia do
Medicamento (EMA).
Metodologia: Foi realizado um estudo qualitativo descritivo, com recurso a dados secundários provenientes de bases de dados da EMA, do INFARMED I.P e da base de dados de medicamentos de uso humano - INFOMED. As variáveis avaliadas incluíram os medicamentos autorizados pela EMA, os medicamentos com apresentações registadas em Portugal, bem como todos os PAPs ativos ou indeferidos durante o
período de estudo. Os dados permitiram analisar o acesso e padrões de aprovação de medicamentos em Portugal através de PAPs.
Resultados: No que diz respeito à legislação e procedimentos de PAPs e AUEs, identificou-se uma reformulação relevante da legislação sobre AUEs com a Deliberação nº 840/2023. Esta Deliberação simplificou e clarificou os procedimentos relativos à AUE, dividindo-os em dois grandes grupos: as AUE relativas a grandes grupos populacionais e as AUE relativas a doentes específicos.
Discussão: O estudo, através de uma análise qualitativa e quantitativa, analisou os medicamentos autorizados no âmbito dos Programas de Acesso Precoce (PAP), englobando todos os PAPs deferidos e os indeferidos, registados no site do INFARMED, I.P. a 20 de julho de 2024. A análise incluiu dados desde janeiro de 2015,
com particular atenção às patologias abrangidas e aos medicamentos disponibilizados.
Este enfoque permitiu avaliar o impacto dos PAPs no acesso a tratamentos para diversas condições clínicas, fornecendo assim uma visão abrangente dos medicamentos disponíveis e das áreas terapêuticas prioritárias.
Conclusões: Este estudo reforça a importância dos PAPs como mecanismo fundamental para assegurar um acesso acelerado aos medicamentos aprovados pela EMA, desde janeiro de 2015. Esta análise demonstra que os PAPs aumentam temporariamente o acesso, permitindo a utilização precoce de medicamentos inovadores durante os processos de avaliação de financiamento público.
Access to medicines represents a significant public health challenge, particularly in the context of serious and/or chronic diseases where there are no therapeutic alternatives. In Portugal, Early Access Programmes (PAPs) and Exceptional Use Authorizations (AUEs) are mechanisms that allow access to medicines before a public financing decision is made, as long as it can be demonstrated that there is no therapeutic alternative where the patient is at immediate risk of death or serious complications (Decreto-Lei n. º 115/2017, 2017). The main objective of this study is to characterize access to medicines in Portugal through PAPs, between 1 January 2015 and 20 July 2024. In addition, the study also aims to describe the procedures and legislation currently in force for PAPs and AUEs, carry out a qualitative and quantitative analysis of the medicines authorised under the PAP and assess the impact of PAPs on access to medicines approved by the European Medicines Agency (EMA). A descriptive qualitative study was developed using secondary data from the EMA, INFARMED I.P and INFOMED databases. Variables under assessment included medicines authorized by the EMA, medicines with marketing authorization in Portugal, as well as all active or rejected PAPs during the study period. The data made it possible to analyze access and approval patterns for medicines in Portugal through PAPs. With regard to the legislation and procedures for PAPs and AUEs, a significant reformulation of the legislation on AUEs was identified with Resolution n. º 840/2023. This Deliberation simplified and clarified the procedures for AUEs, dividing them into two large groups: AUEs for large population groups and AUEs for specific patients. (Deliberação nº 840/2023, 2023). The study, through a qualitative and quantitative analysis, analyzed the medicines authorized under the Early Access Programmes (PAP), encompassing all PAPs granted and those rejected, registered on the INFARMED, I.P. website on 20 July 2024. The analysis included data from January 2015, with particular attention to the pathologies covered and the medicines available. This approach made it possible to assess the impact of the PAPs on access to treatments for various clinical conditions, thus providing a comprehensive overview of the medicines available and the priority therapeutic areas. This study reinforces the importance of PAPs as a key mechanism for ensuring accelerated access to medicines approved by the EMA since January 2015. This analysis demonstrates that PAPs temporarily increase access by enabling the early utilization of innovative medicines during public funding evaluation processes in Portugal.
Access to medicines represents a significant public health challenge, particularly in the context of serious and/or chronic diseases where there are no therapeutic alternatives. In Portugal, Early Access Programmes (PAPs) and Exceptional Use Authorizations (AUEs) are mechanisms that allow access to medicines before a public financing decision is made, as long as it can be demonstrated that there is no therapeutic alternative where the patient is at immediate risk of death or serious complications (Decreto-Lei n. º 115/2017, 2017). The main objective of this study is to characterize access to medicines in Portugal through PAPs, between 1 January 2015 and 20 July 2024. In addition, the study also aims to describe the procedures and legislation currently in force for PAPs and AUEs, carry out a qualitative and quantitative analysis of the medicines authorised under the PAP and assess the impact of PAPs on access to medicines approved by the European Medicines Agency (EMA). A descriptive qualitative study was developed using secondary data from the EMA, INFARMED I.P and INFOMED databases. Variables under assessment included medicines authorized by the EMA, medicines with marketing authorization in Portugal, as well as all active or rejected PAPs during the study period. The data made it possible to analyze access and approval patterns for medicines in Portugal through PAPs. With regard to the legislation and procedures for PAPs and AUEs, a significant reformulation of the legislation on AUEs was identified with Resolution n. º 840/2023. This Deliberation simplified and clarified the procedures for AUEs, dividing them into two large groups: AUEs for large population groups and AUEs for specific patients. (Deliberação nº 840/2023, 2023). The study, through a qualitative and quantitative analysis, analyzed the medicines authorized under the Early Access Programmes (PAP), encompassing all PAPs granted and those rejected, registered on the INFARMED, I.P. website on 20 July 2024. The analysis included data from January 2015, with particular attention to the pathologies covered and the medicines available. This approach made it possible to assess the impact of the PAPs on access to treatments for various clinical conditions, thus providing a comprehensive overview of the medicines available and the priority therapeutic areas. This study reinforces the importance of PAPs as a key mechanism for ensuring accelerated access to medicines approved by the EMA since January 2015. This analysis demonstrates that PAPs temporarily increase access by enabling the early utilization of innovative medicines during public funding evaluation processes in Portugal.
Description
Dissertação para obtenção do grau de Mestre no Instituto Universitário Egas Moniz
Keywords
Acesso a medicamentos Financiamento público Utilização excecional