Claire Murray, Patient and Public Involvement Manager
NIHR Oxford Health Biomedical Research Centre & Oxford cognitive health Clinical Research Facility
Extraction from Oxford Brain Health Clinical Trials Unit’s Knowledge and Innovation Bulletin (special edition) that was released as part of International Clinical Trials Day 2020 celebrations.
Clinical trials have come a long way since their initial conception many centuries ago. Patients and the general public are becoming increasingly involved in clinical trials and research in general. Most organisations that conduct research now insist upon having appropriate patient and public involvement to help design better research. It is a requirement of many research funders and PPI members sit on research approval bodies, such as research ethics committees. Patient and Public Involvement is the focus of Principle 4 of the UK Policy Framework for Health and Social Care Research, which specifies involvement in the design, management, conduct and dissemination of research. PPI has the potential to empower the general public providing democratic accountability which enhances the quality aspects of healthcare research and services.
Evidence associated with PPI itself has evolved quite rapidly over the last 10 years. However, there are challenges with conceptualisation, substandard quality reporting and non-standardised definitions. Systematic reviews have identified benefits of PPI on research, researchers, patients and healthcare service communities. Patient-focus research topics are now prioritised by many funders and PPI has been found to contribute to the successful recruitment of clinical trial participants especially if it includes lived experience of people with the health condition under study (2).
Healthtalk.org researchers interviewed patient and public members who identified personal benefits to being involved, including: helping others and making a difference, giving purpose after a serious illness, a route to developing new knowledge, skills and confidence, and satisfaction from being involved in an interesting project.
As we commemorate International Clinical Trials Day 2020, we wanted to take a closer look at how Patient and Public Involvement can inform clinical trials.
PPI plays a key role in the development of successful trials. It allows both patients and clinical researchers to understand the needs of one another and helps to ensure a clinical trial that is fit for purpose. Involvement activities (1) can provide important contributions to:
- Helping define the most relevant research question and outcomes
- Designing a trial appropriate to the needs and lifestyle of the patient community it serves
- Developing accessible and useful participant materials
- Conducting the trial in a participant friendly and ethical way
- Disseminating trial results to maximise and awareness and ensure the adoption of trial results in clinical practice
We asked three senior clinical researchers working with the Oxford Brain Health Clinical Trials Unit to share their experiences of patient and public involvement.
John Geddes, Head of Department of Psychiatry, University of Oxford, Director of Research and Development for Oxford Health NHS Foundation Trust, Director NIHR Oxford Health Biomedical Research Centre
“Our clinical trials are very complex and usually begin with a high-quality stock taking of the existing evidence. Questions asked by my patients have led directly to specific pieces of research that reveal the state of the science and inform the design of our clinical trials. So in this way PPI is an essential part of the process of how we develop questions for clinical trials.
It makes sure that the questions asked are right, the design is acceptable and robust, and massively helps with the impact and implementation. Only a very inexperienced – or foolhardy – researcher would fail to involve PPI in the conduct of a clinical trial.”
Michael Browning, MRC Clinician Scientist Fellow, Associate Professor, Honorary Consultant Psychiatrist
‘In my experience, the most useful aspect of PPI is that it improves studies in ways I hadn’t even considered beforehand.
During the recent set up of a trial of a medication which has quite a few side effects, the research team was focussed on how to ensure the trial was safe. We had lots of rules in place along the lines of ‘ask the patient if they have side effect X, if they do reduce and stop the treatment’. We discussed the trial with our PPI group and got the feedback — ‘if I think this drug is helping me, and you tell me that you will stop it if I have side effect X, I am just not going to tell you that I had side effect X’.
It needed the patient perspective for us to see that our safety rules were in fact not very safe because they just stopped people being open with us.”
Vanessa Raymont, Senior Clinical Researcher, Director Oxford Brain Health Clinical Trials Unit
“From my perspective, PPI is vital to clinical trials.
I would say that especially within trials for dementia as we are moving towards earlier and earlier biomarkers, in order to identify those at risk, and most importantly track any response to new drugs, the need to include PPI is really important. Without knowing what people may want to engage with when they are still well, we cannot plan such studies.
Also, we need to know now more than ever what people feel about being given a preclinical diagnosis while no effective treatments exist. This has been a cornerstone of setting up the Deep and Frequent Phenotypingstudy in particular.
Understanding the perspective of patients and potential participants will also be key, to successful recruitment for example, as we move into a post COVID-19 world, with the ‘new normal’, when people may be much more cautious about travelling and taking part in research.”
With increasing pressure and health and social care challenges, the importance of diverse thinking in order to develop innovative and sustainable clinical care solutions is vital. Therefore, it is imperative to have the support and involvement of PPI groups to harness their experience, wisdom and energy to take clinical trials to the next level.
(1) Bagley, H.J., Short, H., Harman, N.L. et al. A patient and public involvement (PPI) toolkit for meaningful and flexible involvement in clinical trials – a work in progress. Res Involv Engagem 2, 15 (2016). https://doi.org/10.1186/s40900-016-0029-8
(2) Crocker Joanna C, Ricci-Cabello Ignacio, Parker Adwoa, Hirst Jennifer A, Chant Alan, Petit-Zeman Sophie et al. Impact of patient and public involvement on enrolment and retention in clinical trials: systematic review and meta-analysis BMJ 2018; 363 :k4738