Growing Patient and Public Involvement in Clinical Trials

Patient and public involvement in clinical trials is a defining feature of modern clinical research. Sponsors, regulators, journals, and funding organisations increasingly expect patients or representatives to contribute to trial design, conduct, and dissemination.

However, the evidence suggests that while patient and public involvement in clinical trials is gaining momentum, an important opportunity to strengthen transparency, improve trial quality, and build greater trust with participants remains.

Image

Patient-Centred Research Is the New Standard

Clinical development has evolved considerably over the past decade.

Success is no longer measured solely by scientific rigour or regulatory approval. Increasingly, researchers are expected to demonstrate that trials reflect the priorities and experiences of the patients they are designed to serve.

Patient and public involvement (PPI) differs from patient participation. Rather than enrolling as study subjects, patients contribute as research partners by helping to:

• Refine research questions and study objectives
• Improve participant-facing materials
• Reduce unnecessary trial burden
• Advise on recruitment and retention strategies
• Support dissemination of research findings

These contributions can make studies more relevant, improve participant experience, and ultimately strengthen the quality of clinical evidence.

There’s Progress, but Reporting Remains Limited

To understand how widely PPI is being adopted, researchers conducted a meta-epidemiological evaluation of randomised controlled trials and their published protocols appearing in four leading international medical journals between 2015 and 2023.

Rather than evaluating clinical outcomes, the investigators examined whether studies reported:

• whether patients were involved,
• when involvement occurred,
• what activities patients performed, and
• whether published protocols and final trial reports described involvement consistently.

The findings suggest that patient involvement is still reported far less often than many may expect.

Although reporting increased steadily over the study period, fewer than one in five trial publications or protocols described any patient and public involvement.

Even among studies that reported PPI, descriptions were often brief and lacked sufficient detail to explain how patient input influenced the research.

The authors note that this makes it difficult for researchers, sponsors, and regulators to evaluate whether patient involvement was meaningful or simply acknowledged.

View interview Could Biological Ageing Be the Next Big Shift in Drug Discovery?

Could Biological Ageing Be the Next Big Shift in Drug Discovery?

“The pharma industry is beginning to look at biological age as a surrogate endpoint to improve clinical trials."

Image

Where Patient and Public Involvement in Clinical Trials Is Adding the Greatest Value 

Among studies that reported patient involvement, contributions extended across multiple stages of the clinical trial lifecycle.

These activities can improve both the relevance and acceptability of clinical trials, although inconsistent reporting makes it difficult to compare approaches across studies.

Image

Improving study design

Patients helped shape research questions, review eligibility criteria, identify clinically meaningful outcomes, and improve protocol feasibility before recruitment began.

Supporting recruitment and retention

Patient representatives contributed to participant information sheets, consent documents, recruitment materials, and communication strategies designed to improve enrolment and reduce participant burden.

Strengthening dissemination

Some studies involved patients in interpreting findings and preparing summaries for wider patient communities, helping ensure results were communicated in language that was easier to understand.

Transparency Remains Most Important for Patient-centred Research

Perhaps the most important message from the review is not that patient involvement is lacking, but that its impact cannot be fully assessed without better reporting.

The researchers found discrepancies between trial protocols and published articles. Some protocols described planned patient involvement that was not reported in the final publication, while others simply mentioned patient involvement without explaining what patients actually contributed.

For sponsors, contract research organisations (CROs), and academic investigators, this creates several challenges:

• Limited opportunities to share best practice across studies
• Reduced transparency for regulators and ethics committees
• Difficulty assessing whether involvement influenced trial decisions
• Missed opportunities to demonstrate patient-centred research

The authors therefore recommend more consistent reporting standards that clearly describe who was involved, when involvement occurred, and how patient contributions influenced trial design and delivery.

Patient Partnership Is Becoming a Marker of High-Quality Clinical Development

As decentralised trials, digital health technologies, and personalised medicine continue to grow, patient and public involvement in clinical trials is likely to become an increasingly important measure of study quality rather than simply a reporting requirement.

Meaningful patient engagement is gradually becoming more common, but transparency has not kept pace. Standardised reporting will help sponsors demonstrate the value of patient partnership, improve reproducibility, and strengthen confidence in clinical research.

Ultimately, patient and public involvement should not be viewed as a regulatory obligation, but as a strategic component of better clinical trial design.

At Pharmatica, we examine the strategies, evidence, and operational innovations shaping the future of Clinical Development. From patient-centred trial design to regulatory transformation and digital research technologies, we help life sciences leaders translate emerging evidence into practical decision-making.

Pharmatica: Insight. Connection. Impact.

Image