Co-creation of practical “how-to guides” for patient engagement in key phases of medicines development—from theory to implementation

Abstract

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. Plain English summary 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.

Patient involvement in priority-setting for medical research: A mini review of initiatives in the rare disease field

“…There is a growing recognition of the importance of ensuring that patients are given a greater say in medical care, in settings ranging from research and development to clinical care, as the incorporation of “patient voice … is becoming the expected norm and, in some instances, a requirement” ( 14 ) in many contexts. Notable examples from the United States include the establishment of Patient Listening Sessions by the Food and Drug Administration (FDA), through which patients and advocates are invited to speak to representatives of the FDA ( 15 ), and patient-focused drug development, where patients are asked to use their lived experience to share insights relevant to drug development, regulation, and clinical trials ( 16 ).…”

A practical ‘How-To’ Guide to plain language summaries (PLS) of peer-reviewed scientific publications: results of a multi-stakeholder initiative utilizing co-creation methodology

“…The ‘How-To’ Guide has been developed using an iterative and robust methodology [ 11 – 13 ] to meet an identified need for a practical, ready-to-use tool for multi-stakeholder co-production of PLS. The methodology adhered to key principles of co-creation [ 11 ] and included internal reviews (through WG5 workshops) and external review (at the Patient Engagement Open Forum workshop in September 2019; Fig.…”

“…The methods used for co-creation of the ‘How-To’ Guide have been previously described in detail [ 11 – 13 ]. Briefly, a landscape analysis and a public consultation were carried out and the feedback was used to develop the Patient Engagement Quality Guidance (PEQG).…”

“…Feedback from each step in the review process was consolidated, divided into themes, and prioritized based on the proportion or amount of feedback per theme and WG assessment of its relative importance. This feedback was used to refine the draft ‘How-To’ Guide, which was then validated through additional consultation [ 13 ]. Specific milestones along the PLS co-creation process are shown in Fig.…”

Practices of patient engagement in drug development: a systematic scoping review

“…With discussions about PE in drug development intensifying, we witness the emergence of regulatory initiatives aimed at facilitating PE, as well as efforts to develop guidance for undertaking PE in practice [ 11 , 12 ]. Scholarly attention has turned to exploring attitudes to PE among various stakeholders, ascertaining effects of particular PE instances, and developing tools for evaluating PE [ 13 – 15 ].…”