Organising patient involvement in a research programme
Patient involvement requires a defined role in the programme, suitable ways to exchange information, operational support and continuing assessment of whether partnerships work in practice.
Communication and participation
A 2025 framework distinguished three overlapping domains across research and development, regulation, market access and publications: patients communicating to industry, industry communicating to patients, and bidirectional communication and co-creation. The framework was nonexhaustive.
Patient-to-industry input includes unmet needs, endpoints and outcomes, benefit–risk assessment, protocol design and review, and lay review of patient-facing material. Industry may invite input on its own terms, while patients can also initiate communication to ensure their voices are heard. Industry-to-patient information includes regulatory lay summaries, consent forms, patient leaflets, publication summaries, enhanced publication content and standalone plain language summary publications. These channels aim to support transparency, open science, health literacy and information equity.
Co-creation can involve patient authors, peer reviewers and researchers, research advisory boards, publication steering committees and editorial boards. Within this framework, genuine co-creation ideally exchanges knowledge between equals, recognising patients as lived-experience experts and experiential knowledge as an equally robust form of knowledge alongside other scientific knowledge. Individuals can bring multiple stakeholder identities, experiences and perspectives.
Willingness to listen, recognition of patient expertise and embedding the patient voice support inward communication; shared decision-making and plural voices support co-creation; transparency, open access, citizen science and democratising research support outward communication. Respect, diversity, equity, inclusion and environmental, social and governance goals underpin all three domains.
Programme and trial levels
Programme-level involvement begins early, using patient advisory groups or other means to understand what a participant group wants from a product. Trial-level involvement concerns the design or protocol of one particular study. In October 2025, both remained challenging to organise because many frameworks and initiatives existed without a single established approach.
Frameworks and their purposes
Patient and public involvement and engagement (PPIE) resources described in June 2025 addressed different tasks:
| Resource | Purpose |
|---|---|
| Clinical Trials Transformation Initiative | Recommendations for patient-group engagement in trials |
| European Patients' Academy, EUPATI | Patient involvement in medicines research and training across Europe, including ethics, regulation and health technology assessment |
| GRIPP2 | Checklists for reporting patient and public involvement |
| National Institute for Health and Care Research | UK public-involvement standards |
| Patient-Centred Outcomes Research Institute | US standards and an engagement rubric for patient-centred research |
| FDA Patient-Focused Drug Development guidance | Collection and submission of patient experience data for product development |
| Public Involvement Impact Assessment Framework, PiiAF | Assessment of public involvement's impact |
| TransCelerate Patient Experience initiative | Improving patients' trial experience |
TransCelerate's P-PET guide recommends incorporating patient and public input as early as possible in programme and trial protocol development.
Operational arrangements
Public contributors can include trial participants, people with the condition, patient advocacy group members, caregivers or relatives, and social-service providers.
Periodic surveys, relationships with advocacy groups and caregivers, and a maintained bank of patient insights can inform scientific staff and key opinion leaders designing trials. Suggested operational arrangements are:
- Create organisational SOPs reflecting local regulations and defining patient-partner, advocate and industry roles and responsibilities, with scope for therapeutic-area adaptation. A standard internal process can support continuing partnerships, insight banks, contextual online surveys and virtual meetings, making feedback available before protocols are initiated or changed.
- Allocate budgets, timelines and resources for infrastructure, educational materials and training, financial or nonfinancial compensation and work translating input into actionable research strategies. Implementation needs trained staff and strategic leadership.
- Specify goals and clarify expectations and rules from the outset because researchers' and patient collaborators' backgrounds, perceptions and interests may differ. Agree a simple contract of understanding, confidentiality or nondisclosure arrangements, compensation, regular contact, a plan for collecting, sharing, storing, assessing and using engagement data, and the institutional review board's role.
Sustainable implementation
A 2025 review of medicine-development engagement models found inconsistent definitions and priorities, nonbinding and nonprescriptive guidance, and sparse evidence of consistent, meaningful and sustained implementation. Barriers included insufficient training and expertise, missing practical tools and industry-specific impact metrics, behavioural resistance, gaps in skills and awareness, and unclear scope, expectations and responsibilities. Publication involvement also faced difficulties in identifying where it occurred, with consistency and best practice still evolving across industry. Positive researcher and sponsor attitudes, regulatory encouragement and patient demand had not yet produced consistent sustainable practice.
Impact frameworks require rigorous validation before they can support routine integration into medicine development. Recommendations arising from the 2025 research were to:
- Establish shared global values and principles, unified terminology and an adaptable code aligned with regulatory standards.
- Create precompetitive collaboration to reduce duplicate effort, potentially facilitated by ICH, WHO, CIOMS or professional associations such as EMWA.
- Harmonise stakeholder-specific guidance and training on an accessible digital platform, including published practice case studies.
- Test tools, frameworks and impact measures over time, including meaningful publication metrics.
- Assess digital-platform equity and content accessibility for all patients, particularly underserved communities, while supporting environmental, social and governance goals.