| Name: | Description: | Size: | Format: | |
|---|---|---|---|---|
| 1.62 MB | Adobe PDF |
Advisor(s)
Abstract(s)
As agências reguladoras de todo o mundo estão a considerar a forma como podem
incorporar evidência do mundo real e dados do mundo real nos seus processos de tomada de
decisão. A evidência do mundo real, tem sido amplamente utilizada para gerar dados de
farmacovigilância. Recentemente, reconheceu-se que, para além do seu papel nas avaliações
de segurança, os dados do mundo real têm outras aplicações em diferentes fases do ciclo de
vida do medicamento, dado o seu papel crucial no apoio ao desenvolvimento de
medicamentos e na tomada de decisões regulamentares. Além disso, esta evidência pode ser
utilizada para otimizar e complementar os ensaios clínicos randomizados.
O objetivo do presente documento é analisar e discutir o papel dos dados do mundo
real no processo de tomada de decisões regulamentares, na perspetiva das autoridades
reguladoras do medicamento e da indústria farmacêutica, na União Europeia e nos Estados
Unidos da América. O principal objetivo desta análise é mapear as orientações e iniciativas
regulamentares existentes acerca de evidência do mundo real publicadas pela Agência
Europeia do Medicamento (EMA) e pela Food and Drug Administration (FDA) para apoiar os
processos regulamentares.
Esta monografia abrange as orientações regulamentares e as iniciativas de dados do
mundo real publicadas nos websites da EMA e da FDA. A pesquisa foi complementada com
artigos científicos do PubMed, ScienceDirect e Google Scholar, abrangindo o período de 2018
a 2024. Para explorar a perspetiva da indústria sobre o assunto, foram analisados os websites
da Federação Europeia das Associações e Indústrias Farmacêuticas (EFPIA) e da
Pharmaceutical Research and Manufacturers of America (PhRMA).
A análise revela que a EMA lançou cinco orientações/iniciativas desde 2006, enquanto
a FDA emitiu dez, incluindo sete documentos de orientação finais e três documentos de
rascunho até 2018. Por outro lado, a documentação da indústria farmacêutica é limitada,
incluindo dois documentos oficiais da EFPIA (2021 e 2023), uma declaração (2018) e duas
publicações oficiais (2022 e 2024) da PhRMA.
Tanto a EMA como a FDA estão a incorporar cada vez mais a evidência do mundo real
nas decisões regulamentares. Apesar do progresso, as diferentes definições e expectativas
criam desafios. Esta pesquisa destaca a perspetiva da indústria farmacêutica sobre a
necessidade de definições uniformizadas, colaboração aprimorada e critérios específicos para
a uniformização das práticas regulamentares.
Regulatory agencies worldwide are considering how they may incorporate real-world evidence (RWE) and real-world data (RWD) into their decision-making processes. RWE in the form of post-marketing surveillance has been extensively used for generating pharmacovigilance data. Recently, it has been recognized that, beyond its role in safety assessments, RWE has additional applications across different stages of the medicine’s life cycle, as their crucial role in supporting drug development and regulatory decision-making. Also, this evidence can be employed to optimize and complement the design of randomized controlled trials (RCTs). The aim of the present document is to analyse and discuss the role of RWD/E into regulatory decision-making process from the perspective of drug regulatory authorities and the pharmaceutical industry, across European Union (EU) and United States of America (USA). The main objective of this review is to map the existent RWD/E regulatory guidance and initiatives issued by the European Medicines Agency (EMA) and the Food and Drug Administration (FDA) to support regulatory jurisdictions. This monograph covers the regulatory guidance and RWD/RWE initiatives published on the EMA’s and FDA’s websites. The research was supplemented with scientific and relevant articles retrieved from PubMed, ScienceDirect, and Google Scholar, covering the period from 2018 to 2024. To explore the industry's perspective on the subject, the websites of the European Federation of Pharmaceutical Industries and Associations (EFPIA) and Pharmaceutical Research and Manufacturers of America (PhRMA) were analysed. The analysis reveals that the EMA has launched five guidelines/initiatives since 2006, while the FDA has issued ten, including seven final and three draft guidance documents by 2018. Conversely, there is limited documentation from the pharmaceutical industry, comprising two EFPIA position papers (2021 and 2023), one statement (2018), and two official publications (2022 and 2024) by PhRMA. Both EMA and FDA are increasingly incorporating RWE in regulatory decisions. Despite progress, differing definitions and expectations create challenges. This research highlights the pharmaceutical industry's perspective on the need for standardized definitions, improved collaboration, and specific criteria for RWE design and acceptance to harmonize regulatory practices.
Regulatory agencies worldwide are considering how they may incorporate real-world evidence (RWE) and real-world data (RWD) into their decision-making processes. RWE in the form of post-marketing surveillance has been extensively used for generating pharmacovigilance data. Recently, it has been recognized that, beyond its role in safety assessments, RWE has additional applications across different stages of the medicine’s life cycle, as their crucial role in supporting drug development and regulatory decision-making. Also, this evidence can be employed to optimize and complement the design of randomized controlled trials (RCTs). The aim of the present document is to analyse and discuss the role of RWD/E into regulatory decision-making process from the perspective of drug regulatory authorities and the pharmaceutical industry, across European Union (EU) and United States of America (USA). The main objective of this review is to map the existent RWD/E regulatory guidance and initiatives issued by the European Medicines Agency (EMA) and the Food and Drug Administration (FDA) to support regulatory jurisdictions. This monograph covers the regulatory guidance and RWD/RWE initiatives published on the EMA’s and FDA’s websites. The research was supplemented with scientific and relevant articles retrieved from PubMed, ScienceDirect, and Google Scholar, covering the period from 2018 to 2024. To explore the industry's perspective on the subject, the websites of the European Federation of Pharmaceutical Industries and Associations (EFPIA) and Pharmaceutical Research and Manufacturers of America (PhRMA) were analysed. The analysis reveals that the EMA has launched five guidelines/initiatives since 2006, while the FDA has issued ten, including seven final and three draft guidance documents by 2018. Conversely, there is limited documentation from the pharmaceutical industry, comprising two EFPIA position papers (2021 and 2023), one statement (2018), and two official publications (2022 and 2024) by PhRMA. Both EMA and FDA are increasingly incorporating RWE in regulatory decisions. Despite progress, differing definitions and expectations create challenges. This research highlights the pharmaceutical industry's perspective on the need for standardized definitions, improved collaboration, and specific criteria for RWE design and acceptance to harmonize regulatory practices.
Description
Trabalho Final de Mestrado Integrado, Ciências Farmacêuticas, 2024, Universidade de Lisboa, Faculdade de Farmácia.
Keywords
Real-world evidence Real-world data Regulatory decision-making European Medicines Agency US Food and Drug Administration Mestrado integrado - 2024