Monitoring and Evaluation of Patient Engagement in Health Product Research and Development: Co-Creating a Framework for Community Advisory Boards.

PURPOSE: While patient engagement is becoming more customary in developing health products, its monitoring and evaluation to understand processes and enhance impact are challenging. This article describes a patient engagement monitoring and evaluation (PEME) framework, co-created and tailored to the context of community advisory boards (CABs) for rare diseases in Europe. It can be used to stimulate learning and evaluate impacts of engagement activities. METHODS: A participatory approach was used in which data collection and analysis were iterative. The process was based on the principles of interactive learning and action and guided by the PEME framework. Data were collected via document analysis, reflection sessions, a questionnaire, and a workshop. RESULTS: The tailored framework consists of a theory of change model with metrics explaining how CABs can reach their objectives. Of 61 identified metrics, 17 metrics for monitoring the patient engagement process and short-term outcomes were selected, and a "menu" for evaluating long-term impacts was created. Example metrics include "Industry representatives' understanding of patients' unmet needs;" "Feeling of trust between stakeholders;" and "Feeling of preparedness." "Alignment of research programs with patients' needs" was the highest-ranked metric for long-term impact. CONCLUSIONS: Findings suggest that process and short-term outcome metrics could be standardized across CABs, whereas long-term impact metrics may need to be tailored to the collaboration from a proposed menu. Accordingly, we recommend that others adapt and refine the PEME framework as appropriate. The next steps include implementing and testing the evaluation framework to stimulate learning and share impacts.

Sustaining Meaningful Patient Engagement Across the Lifecycle of Medicines: A Roadmap for Action.

BACKGROUND: There is increased recognition that incorporating patients' perspectives and insights into the medicines development process results in better health outcomes and benefits for all involved stakeholders. Despite the increased interest and the existence of frameworks and practical recommendations, patient engagement (PE) is not yet considered standard practice. The objective of this work was to provide a roadmap to support systematic change in all stakeholder organisations involved in medicines development across Europe, patients and patient organisations, medicines developers, academia, regulatory authorities, Health Technology Assessment bodies, payers, policy-makers and public research funders, to sustain PE practices. METHODS: A mixed-methods approach was used by the EU-funded Innovative Medicines Initiative PARADIGM Consortium to co-develop the sustainability roadmap including background work to identify success factors and scenarios for sustainable PE. The roadmap development was based on the Theory of Change concept and populated with findings from (1) interviews with national/ and international institutions with the potential to increase PE uptake by other stakeholders; (2) multi-stakeholder workshops and webinars; and (3) consultations with specific stakeholder groups, Consortium members and a consultative body formed by international PE initiatives. RESULTS: This roadmap sets strategic goals for the PE community to achieve meaningful and systematic PE through changes in the culture, processes and resources of stakeholder organisations. It brings in key PARADIGM outputs to work in a coordinated fashion with existing frameworks and mechanisms to achieve system-wide sustained PE. CONCLUSIONS: The roadmap provides a framework for all stakeholders to take collective action within their organisations and across Europe to implement PE in a sustainable manner.