RESUMO
BACKGROUND: The effective impact of patient engagement (PE) across the medicines development continuum is widely acknowledged across diverse health stakeholder groups, including health authorities; however, the practical applications of how to implement meaningful and consistent PE are not always addressed. Guidance for the practical implementation of PE requires granularity, and the need for such guidance has been identified as a priority. We describe the co-production and summarize the content of how-to guides that focus on PE in the early stages of medicines development. METHODS: Multi-stakeholder working groups (WGs) were established by Patient Focused Medicines Development (PFMD) for how-to guide development. How-to guides were co-produced with patients for PE activities identified as priorities through public consultation and by WGs. Guides were developed by applying PE quality guidance and associated quality criteria in an iterative process. How-to guides underwent internal review and validation by experts (ie, those with relevant experience in the particular PE activity or focus area) in specific focus groups and external review and validation through appropriate events and public consultation. RESULTS: Overall, 103 individual contributors from 38 organizations (representing eight stakeholder groups, including patients/patient organizations) and from 14 countries were organized into WGs and workstreams. Each WG comprised 15-30 contributors with PE experience relevant to the specific how-to guide. How-to guides were developed for PE in the early discovery and preclinical phases; PE in the development of a clinical outcomes assessment strategy; and PE in clinical trial protocol design. The how-to guides have a standardized format and structure to promote user familiarity. They provide detailed guidance and examples that are relevant to the individual PE activity and aim to facilitate the practical implementation of PE. CONCLUSIONS: The how-to guides form a comprehensive series of actionable and stepwise resources that build from and integrate the PE quality criteria across the medicines continuum. They will be made freely available through PFMD's Patient Engagement Management Suite ( pemsuite.org ) and shared widely to a variety of audiences in different settings, ensuring access to diverse patient populations. Implementation of these guides should advance the field of PE in bringing new medicines to the market and ultimately will benefit patients. Medicines are developed to help patients improve their health and lives. Many organizations and individuals want to ensure that medicines are developed to meet real patient needs and to address what is most important to patients. Finding out what patients need and what patients want requires good patient engagement, but knowing how to do patient engagement is not always clear. This is because medicines development is complicated, and a lot of different steps, people, and organizations are involved. Patient Focused Medicines Development (PFMD) was established in 2015 to connect individuals and organizations that are committed to making medicines not just for patients but with patients. To do this, PFMD brought together patients and other groups of people with relevant experience and good ideas on how to achieve patient engagement in the real-world setting. Together, PFMD has developed "how-to guides" for patient engagement that cover the main activities along the medicines development process. The guides are free to use and provide practical advice and examples that anyone can use in their patient engagement activities. The how-to guides will also help patients to understand medicines development and how best they can participate in this process to address their needs.