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The value of PPIE in ATMP development

A suite of resources demonstrating how early, meaningful Patient and Public Involvement and Engagement (PPIE) can create strategic and financial value for developers of ATMPs

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Patient and Public Involvement and Engagement (PPIE), also referred to as patient and public involvement (PPI) or public involvement (PI), is defined as research being carried out ‘with’ or ‘by’ members of the public rather than ‘to’,‘about’ or ‘for’ them. In the development of Advanced Therapy Medicinal Products (ATMPs), patients and the public make vital contributions by sharing their lived experience partnering with researchers and developers.

This suite of resources demonstrates how meaningful PPIE can be embedded early in development to improve study design, strengthen participant-facing materials, and support regulatory preparedness.

The first article outlines the business case for PPIE, drawing on published literature to show how early involvement can help reduce inefficiencies and support more commercially advantageous development pathways. Two accompanying case studies from UK ATMP developers — Accession Therapeutics’ first-in-human oncolytic virus trial in advanced cancer, and Rinri Therapeutics’ cell therapy programme for hearing loss — illustrate how the early and meaningful integration of PPIE can deliver these benefits in practice.

Together, these resources show that PPIE is not simply a regulatory requirement or an optional add-on, but a practical approach to enabling more efficient, participant-centred ATMP development.

Article

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Review Article PPIE Beyond Ethics

A Business Case for ATMP Developers

Case studies

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Case Study Improving Trial Design and Delivery Through Early PPIE

An Oncolytic Virus Programme by Accession Therapeutics & ECMC Network
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Case Study The Value of Early PPIE in the Clinical Development Pathways

A Cell Therapy for Hearing Loss by Rinri Therapeutics

This suite of resources was developed by:

Additional contributors are acknowledged in each resource

 

More PPIE resources

ATMP Engage

ATMP Engage PPI Directory

A directory of PPI resources including toolkits, guidelines, advice, templates and case studies, curated for developers of cell and gene therapies,
atmp engage logo

ATMP Engage

Additional resources developed by the ATMP Engage Working Group
NIHR logo

NIHR UK Standards for Public Involvement

NIHR's practical guidance, resources, and background information on public involvement in health research
PFMD logo

Patient Engagement Management Suite (PEM Suite)

A PPIE toolkit developed by Patient Focused Medicines Development (PFMD)
EPF logo

Improving Patient Involvement in Medicines Research and Development

A practical roadmap for patient involvement in medicines R&D life cycle
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