The Importance of Patient and Public Involvement and Engagement (PPIE)

Dr Sylwia Migas, Head of Analytics 

Introduction: From Passive Subjects to Active Partners

For decades, clinical research has frequently marginalised the individuals it seeks to serve - patients, treating them primarily as passive subjects rather than active collaborators. This approach, rooted in historical ethical shortcomings, has hindered both the relevance and applicability of research outcomes. In response, this field is undergoing a paradigm shift towards greater transparency, inclusivity, and patient-centred methodologies, recognising that meaningful patient engagement is essential to advancing both ethical standards and scientific rigour.

Frameworks such as the Declaration of Helsinki and Good Clinical Practice (GCP) have solidified the importance of informed consent and participant rights. However, true progress lies in embracing Patient and Public Involvement and Engagement (PPIE), which places people at the heart of research design, implementation, and analysis.

As Head of Analytics at NWEH, I believe PPIE is essential not just ethically, but strategically. As a technology partner specialising in real-world clinical trials, our advanced platforms are most effective when built on insights from the very users they serve. Embedding PPIE at the feasibility stage of every study improves design, enhances participant retention, and strengthens data quality.

Enhancing Recruitment and Retention Through Lived Experience

Recruitment and retention remain among the most significant challenges in clinical research. Delays or failures to enrol participants can stall innovation and compromise the generalisability of results.

PPIE offers a solution. A recent meta-analysis showed that PPIE interventions significantly improve enrolment rates (OR 1.16; 95% CI 1.01–1.34), with even greater benefits when patients with lived experience are involved (OR 3.14 vs. 1.07; P=0.02) [1]. Though data on retention is less conclusive, it is widely understood that participants who feel informed and involved are more likely to stay engaged throughout a study.

In practice, PPIE enhances not only the clarity, cultural relevance, and practical applicability of study materials but also informs study design, ethical considerations, and dissemination strategies, while concurrently fostering participant trust and engagement. These multifaceted contributions collectively lead to improvements in recruitment efficiency, reductions in attrition rates, and increased patient-reported satisfaction, thereby strengthening overall study validity and data integrity.

PPIE as a Catalyst for Better Analytics and Higher-Quality Data

The benefits of PPIE extend well beyond enrolment. When patients help shape trial feasibility and design, the resulting data is more meaningful and aligned with real-world needs.

From an analytics perspective, this alignment is invaluable. It ensures that key variables reflect outcomes patients care about - quality of life, symptom control, daily function - not just clinical markers. The result: richer, more interpretable data and stronger models.

Retention, too, plays a key role. Better adherence and continuity lead to cleaner longitudinal data, enhancing the statistical power and validity of findings. Patient-reported outcomes (PROs), a growing standard in trial evaluation, benefit from this engagement by capturing nuanced, personal health impacts often missed by traditional metrics.

Case Study: Co-Designing FARSITE Improvements Through PPIE

To bring PPIE to life, NWEH recently conducted a formal engagement initiative to shape proposed improvements to our FARSITE system, a key tool used to identify and recruit patients for real-world clinical trials and health improvement projects [2].

Between August and October 2024, structured feedback was collected from GPs actively using FARSITE, as well as 31 members of the public. The goal: evaluate potential system enhancements, including predictive analytics, improved communication, and data transparency.

Key Outcomes Included:

• 100% of GPs agreed that proposed improvements to FARSITE were important or very important, particularly those aimed at enhancing longitudinal patient safety monitoring, supporting proactive care, and improving recruitment precision.
• 84% of public respondents rated the proposed improvements as “Very Important”, showing strong support for anonymised data use to develop treatments, monitor drug safety, and predict healthcare needs.
• GPs highlighted the importance of in-person training and robust support, while patients prioritised easy-to-understand data-sharing policies and independent security checks.
• The public overwhelmingly expressed trust in GPs and researchers to use data responsibly, especially when governed by frameworks like GDPR.
• Most participants preferred email, letters, or phone calls as methods of communication about how their data is used and how they can get involved in studies.

• 81% would be willing to participate in future trials, with a preference for invitations to be sent by letter.

For digital health technologies to succeed, they must be both functional and user-friendly. Involving GPs and patients during feasibility stages reduces the risk of costly mid-study revisions. It supports the creation of inclusive, accessible digital experiences and enhances the likelihood of technology adoption in trial settings.

This initiative demonstrated how involving users early in the process yields more relevant tools, reduces operational risk, and builds trust. Feedback directly informed development priorities, training content, and sharing information about data governance strategies.

Future Directions: Embedding PPIE in the Digital and AI-Driven Research Landscape

As clinical research becomes increasingly digitised, Patient and Public Involvement and Engagement (PPIE) must evolve to meet new challenges and seize new opportunities. Technologies like artificial intelligence, federated learning, and real-time health data capture offer immense promise but also require public trust and transparent governance to be successful.

At NWEH, we see an emerging role for PPIE in shaping the ethical design of predictive analytics and AI tools used in trial feasibility, recruitment forecasting, and risk modelling. Involving patients and public contributors in the development of these models can help ensure:

• Fair representation of diverse populations
• Transparency around data use and algorithmic decisions
• Trust in how decisions are made and communicated

There is also growing potential to scale PPIE using digital engagement platforms, allowing broader and more inclusive participation, especially from underrepresented communities. However, this requires thoughtful design to avoid digital exclusion and support informed, equitable participation.

Looking ahead, PPIE will not only support more inclusive research, but it will be essential to guide how innovation is adopted in a way that reflects real-world values, preferences, and lived experiences.

Policy and Regulatory Context in England

In England, the Health Research Authority (HRA) strongly encourages public involvement in research design. Their Participant Information Design and Review Principles [3] stress that co-production improves clarity, relevance, and ethical robustness.

Importantly, PPIE during the design phase does not require Research Ethics Committee (REC) approval, but ethical issues arising during the conduct of the study are subject to scrutiny. Proactively addressing these through early engagement reduces regulatory friction.

PPIE is not just supported by policy; it is increasingly a prerequisite for funding, ethical review, and public trust.

Conclusion: A New Standard for Real-World Research

Embedding PPIE at the feasibility stage is no longer optional; it is foundational to high-quality, inclusive research. The benefits are clear: stronger recruitment, better retention, more meaningful data, improved system usability, and enhanced public trust.

For NWEH, PPIE is a critical enabler of our mission to accelerate safer, smarter clinical trials. Our FARSITE engagement case study is just one example of how involving patients and professionals early drives measurable improvements across the trial lifecycle.

We are committed to designing studies not just for patients, but with them - because when research is shaped by the people it aims to serve, everyone benefits.

References:

1. Crocker JC, Ricci-Cabello I, Parker A, Hirst JA, Chant A, Petit-Zeman S, Evans D, Rees S. Impact of patient and public involvement on enrolment and retention in clinical trials: systematic review and meta-analysis. BMJ. 2018 Nov 28;363:k4738. doi: 10.1136/bmj.k4738. PMID: 30487232; PMCID: PMC6259046.
2. https://www.nweh.co.uk/farsite
3. https://www.hra.nhs.uk/planning-and-improving-research/research-planning/participant-information-design-and-review-principles/