PPIE programme supporting a Phase III clinical trial

A protocol designed by clinicians, for clinicians

A pharmaceutical company developing a treatment for a long-term inflammatory condition was preparing for a Phase III trial. The clinical team had strong views about the primary and secondary outcome measures, based on established endpoints used in similar trials. What they had limited insight into was whether those endpoints reflected what patients living with the condition actually wanted to know - whether the treatment worked in ways that mattered to their daily lives.

NIHR guidance and the growing expectations of ethics committees and regulators made a robust PPIE programme a practical necessity. But the research team also recognised a genuine argument: patient-reported outcome measures and patient-defined endpoints produce more meaningful data and trials that are more likely to retain participants. The brief was to design and deliver a PPIE programme that would inform the trial from protocol development through to participant recruitment, with documentation sufficient for ethics submission and regulatory review.

Patients involved at every stage that mattered

Participation Studio established a Patient Advisory Group of eight individuals with lived experience of the condition, recruited through patient organisations and condition-specific online communities. Membership was deliberately diverse in terms of disease duration, treatment history, employment status, and age - factors that the team expected would produce different perspectives on what meaningful improvement looked like.

The programme operated across three phases. In the first, the advisory group was engaged before the protocol was finalised: reviewing the proposed primary and secondary endpoints, discussing what improvements in those areas would mean in daily life, and identifying aspects of their experience that the current outcome measures didn't capture. In the second phase, the group reviewed and rewrote the participant information sheet and consent form in plain English, working directly with the clinical writing team. In the third phase, participants contributed to developing the trial's retention strategy - drawing on their understanding of what would make sustained participation realistic for people living with a demanding long-term condition.

What the research involved

Protocol changes that improved the science

The consent form review produced substantial changes to both language and structure. The advisory group identified sections that created unnecessary anxiety, requests for information that participants were unlikely to understand without clinical background, and an overall length that several members described as a barrier to participation for people who were fatigued or in pain when reading it. A revised version tested more clearly with a small validation group before finalisation.

The retention strategy discussions surfaced practical factors that the trial team hadn't fully modelled: the impact of assessment visit frequency on participants with demanding work schedules, the importance of regular non-clinical contact to maintain motivation, and the communication approaches that participants found respectful versus those that felt like administrative box-ticking. Several of these insights were incorporated directly into the trial's participant support framework.

Evidence of involvement that changed the trial