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

Abstract

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.

Keywords: Call to action; Medicines development; Patient engagement; Roadmap; Sustainability.

Fig. 1

Common sustainability factors categorized according to the three sustainability dimensions. Source: Informal interviews with initiatives/organisations in the open innovation field related or not with PE in medicines development

Fig. 2

Sustainability scenarios building blocks. These components were used to describe future scenarios with the greatest potential to accelerate the implementation of PE and make it sustainable. Each of these components can drive PE within stakeholder organisations and across the medicines development ecosystem

Box 1

Priority activities for PE sustainability in the CEE region

Fig. 3

PE sustainability roadmap visual summary

Fig. 4

Integrated resources, guidance and tools covering PE phases. List of existing PE frameworks to achieve sustainable PE. Top headings: Phases of patient engagement. All listed resources are available and free for use and links are available in the Supplementary information. Blue boxes: Recommendations, guidance and tools included in the PARADIGM Toolbox. Orange boxes: Tools and resources from existing patient engagement frameworks (non-exhaustive). Green boxes: PE training resources (non-exhaustive). All listed resources are available and free for use. PE patient engagement, PFMD patient-focused medicines development, CTTI Clinical Trials Transformation Initiative, PCORI Patient-Centered Outcomes Research Institute, NHC National Health Council, NIHR National Institute Health Research, EFPIA European Federation of Pharmaceutical Industries Association, ABPI Association of British Pharmaceutical Industry, EUPATI European Patients Academy on Therapeutic Innovation, EPF European Patients' Forum

Fig. 5

Platforms important for PE sustainability. Links to all listed resources are available in the Supplementary Information

Box 2

Call to action to the PE community