Patient and Public involvement in the design of an international clinical trial: Real world experience
Authors: Simons Gwenda , Jones Helen , Clarke Ian , Davies Firoza , Grealis Stacey , Insch Elspeth , Kahn Hameed , Lloyd Joanne , Richards Al , Rose Hayley , Williams Ruth , de Wit Maarten , Woodcock Clarissa , Romaniuk Leigh , Bardgett Michelle , Pratt Arthur G , Falahee Marie 
Published: September 2024

Background

The value of patient and public involvement (PPI) during the earliest stages of clinical trial development, and prior to the award of substantive funding, is widely recognised. However, it is often under resourced and PPI processes during this phase are rarely reported in detail. Having benefitted from seed funding to develop an international clinical trial proposal, we sought to describe and appraise PPI activities and processes that support pre-award co-development.

Methods

A 12-month “accelerator” award facilitated development of a substantive funding application to deliver the Rheumatoid Arthritis Prevention PlatfORm Trial (RAPPORT), conceived to prioritise preventative interventions for people at risk of RA. PPI partners, including individuals at risk of rheumatoid arthritis (RA), RA patients, relatives and members of the public, provided feedback on key trial design issues through online meetings, a feedback form and emails. PPI processes employed during the one-year accelerator project were thereafter evaluated by PPI partners using an anonymous online feedback form with reference to National Institute of Health and Care Research (NIHR) UK standards for public involvement in research.

Results

Sixteen out of the 25-strong PPI partner panel completed an online feedback form (64%). Respondents perceived PPI processes positively in relation to all NIHR standard domains. Several key facilitators and challenges were identified, including the need for adequate PPI funding during pre-award phases of research, strategies for creating an inclusive environment, flexibility around levels of involvement, and challenges in achieving representatively diverse participation, and the importance of communicating transparent processes for role-assignment and time-reimbursement.

Conclusions

In general, RAPPORT was considered an example of PPI well done, and in line with UK standards for public involvement in research. Facilitators and challenges of relevance for the development of future translational and clinical trial funding applications are highlighted.

PLAIN ENGLISH SUMMARY

Patient and public involvement (PPI) in the development of funding applications to deliver clinical trials is desirable, but the PPI activities and processes involved at this early, “pre-award” stage are rarely reported. In the current paper we describe such activities during a 12-month project to develop a grant proposal for a substantive, international clinical trial. Three PPI partners were co-recipients of “seed funding” to conduct the 12-month Accelerator project, an additional 22 PPI partners being subsequently recruited to co-develop the funding application for the trial, entitled the “Rheumatoid Arthritis Prevention: catalysing PlatfORm Trial (RAPPORT).” PPI partners contributed through meetings, email discussions and the completion of feedback forms. The PPI processes used in the project were evaluated by 16 of the PPI partners using an anonymous online feedback form. The form asked about the areas covered by the UK Standards for Public Involvement.

PPI partners indicated that PPI in RAPPORT was done well in relation to all areas of the UK Standards. PPI partners felt they were heard, and their input valued, and that the communication was effective. Furthermore, they appreciated online format of the PPI activities, the flexible levels of involvement offered and the support from staff with expertise in both research and PPI. Some areas for potential improvement in future initiatives were also identified, which are discussed alongside challenges to co-development of projects during the “pre-award” stage in general, and the benefit of seed funding to support effective PPI.