An evidence base to optimise methods for involving patient and public contributors in clinical trials: a mixed-methods study

Abstract

BackgroundIn comparison with other study designs, randomised trials are regarded as particularly likely to benefit from patient and public involvement (PPI). Using mixed-methods research we investigated PPI from the perspectives of researchers and PPI contributors.MethodsRandomised trials in receipt of funding from the Health Technology Assessment (HTA) programme between 2006 and 2010 were identified. Funding applications and board and referee comments were obtained and data relevant to PPI extracted. Chief investigators (CIs), PPI contributors and UK Clinical Research Collaboration Registered Clinical Trials Units (RCTUs) were surveyed. Interviews were conducted with researchers and PPI contributors.ResultsA total of 111 trials were included. Text relevant to PPI was identified in half of the trials for which the first-stage applications were available, but only one-quarter described PPI within their development. In the second stage of the application, the majority provided some text relevant to PPI, with over half having PPI in their development. Fewer than half of referees commented on PPI, and funding boards rarely provided comments in relation to PPI. Seventy-three per cent (81 of 111) of CIs responded to the survey and 98% (79 of 81) included PPI at some stage in their trial. CIs considered high impact from PPI contributors to occur more frequently in trial setup, with low or no impact being more common during trial conduct, analysis and dissemination. Only one-third of CIs provided PPI contributor contact details but all contributors contacted completed the survey. The majority of contributors felt engaged and valued by the research team. Interviews were conducted with researchers and/or PPI contributors for 28 trials identifying two main influences on perception of PPI impact: whether or not CIs expressed personal goals and plans for PPI; and the quality of their relationship with the PPI contributors. The importance of early engagement was identified, with opportunity for input thereafter limited. Three PPI roles were identified: oversight, managerial and responsive. Oversight roles, as required by funders, were associated with low impact in comparison with responsive or managerial roles. Most researchers could see some value in PPI training for researchers, although those that had received such training themselves expressed concerns about its purpose and evidence base. Training for PPI contributors was considered unnecessary, with conversational approaches preferred, although this did not appear to provide an opportunity for role negotiation. The RCTU survey response rate was 85% (39 of 46). The majority (37 of 39) reported PPI within trials co-ordinated by their unit. Trial characteristics were used by half to determine the approach to PPI. Two-thirds reported recent developments or changes in implementing plans for PPI (21 of 33). Support to PPI contributors was commonly offered through members of staff at the unit.ConclusionsPPI is occurring in the majority of trials funded by the HTA programme, but uncertainty remains about how it is assessed and valued. Early involvement, building a relationship between researchers and contributors, responsive or managerial roles, and having defined goals for PPI were associated with impact. Efficiency could be gained by utilising the RCTU network to identify and tackle challenges, and develop a risk-based approach utilising trial characteristics. Recommendations are made to trial funders and the research community. Given the difficulties for some informants in recalling PPI contributions, future research using a prospective approach would be valuable. Ethnographic research that combines observation and multi-informant interviews is likely to be informative in identifying impact. The research community needs to give further consideration to processes for selecting PPI contributors and models of implementing PPI.FundingThe National Institute for Health Research Health Services and Delivery Research programme and INVOLVE.

Developing involvement during a programme of recovery research

“…Other forms of involvement, for example through peer review (Staley, 2016) and in trials (Gamble, 2015) are also being evaluated Overall, the impact of patient and public involvement in research is positive and increasing.…”

PERSPECTIVES: Stroke survivors' views on the design of an early‐phase cell therapy trial for patients with chronic ischaemic stroke

“…Early patient engagement is likely to be associated with increased recruitment and retention of study participants; development of research methods that are contextualized to patients' experiences with the disease; and utilization of relevant research questions and outcome measures. There is growing evidence for the value of ‘patient and public involvement in research’ (PPIR) in facilitating more patient‐focused research by offering insights into prioritization, design and implementation and making trials more effective and credible . PPIR is increasingly being mandated for publicly funded trials in many developed countries …”

“…There is growing evidence for the value of 'patient and public involvement in research' (PPIR) in facilitating more patient-focused research by offering insights into prioritization, design and implementation and making trials more effective and credible. 10 PPIR is increasingly being mandated for publicly funded trials in many developed countries. 11 Active participation of potential participants is likely to provide a sense of empowerment to people with chronic stroke.…”

Older Adults' Perspectives on Clinical Research: A Focus Group and Survey Study

“…19 For example, the Patient-Centered Outcomes Research Institute (PCORI), which funds comparative effectiveness trials in the US, requires patient input into all aspects of clinical trials, including the study design and the choice of outcome measures. 20 Older adults may offer a unique patient voice in the design of such studies.…”