Understanding multi-stakeholder needs, preferences and expectations to define effective practices and processes of patient engagement in medicine development: A mixed-methods study.

BACKGROUND: The holistic evolution of patient engagement in medicines development requires a more detailed understanding of the needs of all involved stakeholders, and one that better accounts for the specific needs of some potentially vulnerable patient populations and key stages in medicines development. OBJECTIVE: The purpose of this convergent mixed-methods study was to better understand the needs of different stakeholders concerning patient engagement at three key stages in medicines development: research priority setting, clinical trial design and early dialogues with Health Technology Assessment bodies and regulators. DESIGN: This study brought together findings from three sources: i) an online questionnaire, ii) face-to-face consultations with two potentially vulnerable patient populations, a workshop with Health Technology Assessment bodies, and iii) three-step modified Delphi methodology. RESULTS: Overall stakeholders still need additional varied support mechanisms to undertake, sustain or measure value of patient engagement. Health Technology Assessment bodies need better rationale for patient engagement in early dialogue and tools to support its implementation. Improved awareness and understanding of the need and value that involving patients, who are often considered as potentially vulnerable, can bring is needed, as is better accommodation of their specific needs. Similarly, weighted Delphi categories were as follows: aims and objectives, and sustainability. Several additional themes were common across the three key stages in medicines development. CONCLUSION: This broad-reaching study provides the blocks needed to build a framework for patient engagement in medicines development. PATIENT OR PUBLIC CONTRIBUTION: Patients were involved in review and interpretation of data.

Optimising Multi-stakeholder Practices in Patient Engagement: A Gap Analysis to Enable Focused Evolution of Patient Engagement in the Development and Lifecycle Management of Medicines.

BACKGROUND: The PARADIGM consortium aimed to make patient engagement in the development and lifecycle management of medicines easier and more effective for all, with the development of new tools that fulfil robustly defined gaps where engagement is suboptimal. AIMS: To generate an inventory of gaps in patient engagement practices and process from existing global examples. METHODS: A large set of criteria for effective patient engagement previously defined via a multi-stakeholder Delphi method, were mapped under fourteen overarching themes. A gap analysis was then performed by twenty-seven reviewers against the resulting forty-six mapped criteria, on a sample of seventy initiatives from global databases. RESULTS: An inventory of gaps was identified including contextual information as to why the gaps exist. Our work identified general patterns where patient engagement was suboptimal-defined as; fragmented reporting and dissemination of patient engagement activities, and the fundamental principles defined in frameworks or guidance being poorly adhered to in actual practice. Specific gaps were identified for sixteen criteria. Additionally, it was also common to observe primary aspects of a process were addressed for a given criteria (i.e. training for roles and responsibilities) but a secondary context element was lacking (i.e. making training material accessible/understandable/meaningful to all participants). CONCLUSION: The results show that the evolution towards meaningful and systematic patient engagement is occurring, yet more importantly they provide clear directional insights to help enhance collaborative practices and co-design solutions. This targeted impact to catalyse a needs-oriented health system that integrates patient engagement at its core is essential.