OBJECTIVES : To conduct a global consultation of perceived relevance and priority of patient engagement (PE) activities in medicines development across multiple stakeholder groups.

METHODS : Patient Focused Medicines Development (PFMD, a global multistakeholder coalition) reviewed published literature (2011-2017) to identify a preliminary list of PE activities. A 22-question online survey covering each phase of medicines’ development was designed for stakeholders involved/experienced in PE in medicines development. Respondents were asked to prioritise listed PE activities at each phase, via questions related to communication, engagement, methodology and training/education. Consultation was conducted over 9 weeks (June-August 2018).

RESULTS : 159 PE activities were included: research/discovery (n=35); preclinical (n=34); clinical (n=44); regulatory (n=22); post-approval (n=24). 183 respondents completed the survey, representing 10 different stakeholder groups and 26 countries (23% from outside EU-US axis). Respondents’ PE experience (per phase) was: 61% (early); 52% (preclinical); 61% (phase I-III); 34% (regulatory); 48% (post-approval). Overall, 87% of PE activities were rated as moderately/highly relevant. Prioritised PE activities (per phase) included: collection of patient insights on disease experience, use of patient experience to design research methodology (early); patient participation in design of patient information materials, trial design and operations (preclinical); patient participation in research questions, trial design, patient-reported outcomes methods design, study logistics and accessibility issues (phase I-III); testimony for public hearings and advisory committees, input to lay summaries and medical information (regulatory); patient community feedback including in post-marketing research, lay communication and outreach, adverse events reporting (post-approval). Activities rated highly relevant across phases included: understanding patient disease experience; lay patient communication and outreach; input into research aims and methodology.

CONCLUSIONS : This global survey provides new insights to guide the focus of future PE implementation support efforts. PFMD and multistakeholder volunteers are facilitating co-creation of “how-to” modules for prioritised PE activities, using PFMD PE Quality Guidance methodology (Deane et al. ISPOR Europe 2018; poster PHP14).