The inclusion of the patient voice in research is not a new concept. But while joint decision making in a clinical setting is common, it is less so in research particularly towards the 'fundamental' end of the pathway. Fields such as mental health, HIV and cancer have done better than most - progress wouldn't have been possible without patient and carer input on which health challenges need to be addressed and how to conduct research in a way that doesn’t burden participants further.
In the UK, we are working towards the inclusion of the patient voice in all aspects of public health, termed patient and public involvement (PPI). The research arm of our National Health Service, the National Institute for Health Research (NIHR), requires the input of patients across all aspects of its funded research and supports the UK's national PPI advisory group INVOLVE.
INVOLVE defines PPI as when “members of the public are actively involved in research projects and in research organisations". It is research "carried out with or by members of the public rather than to, about or for them".1
There are many forms of PPI. For example: reference groups, lay reviewers, steering group members and research partners. Benefits for health research2,3,4 include :
- identifying and prioritising the most relevant research and health topics
- setting endpoints which have greater real-life relevancy
- securing funding
- the accessibility and sensitivity of language in lay summaries and public/patient-facing information
- clinical trial management, including improvements in recruitment and retention rates
- providing real-world context to data interpretation
- broader dissemination of results
There are reciprocal benefits for patients and the public too: greater awareness and understanding of research; more open attitudes and trust in research/ers; a greater feeling of being supported and listened to as well as a sense of personal growth and achievement.3
So then why, when a case for PPI across all stages of the research pathway has been established, 2,3,4,5 are we not seeing more PPI in research?
The challenges in conducting PPI include limited time and resources, lack of definition of everyone’s roles, unclear goals as well as difficulties in establishing trusted relationships with clear communication.3,5 Good PPI practice must often be learnt as you go and developed over time. Commitment and open-mindedness to the process is what leads to tangible benefits in the end.5 Engaging early is paramount as is genuine intent. Involving people from the design stage, or even better when setting the initial question, invests everyone in a common goal giving time to learn how to work together effectively throughout. Where motivations are tokenistic, and PPI is seen as a ‘box ticking’ exercise to meet funding requirements, it at best under delivers or worse doesn’t deliver at all, wasting everyone’s time and making people less inclined to be involved in the future.5
I believe that PPI is as valuable to pre-clinical research as it is to clinical research. Many bench scientists can't see where a non-scientist's perspective could be of benefit and feel that scientific understanding is needed to derive meaningful input. I argue this is a self-imposed barrier and have seen first-hand that scientific understanding and good PPI don't necessarily equate. INVOLVE provide practical guidance on implementing PPI and Parkinson’s UK and the UK national voluntary sector’s Shared Learning Group on Involvement have both developed case study-led guidance on bringing valuable patient voices from ‘bedside to bench’.1,6,7
There are various examples of PPI in place at our joint UCL Institute of Ophthalmology and Moorfields site. Our research strategy is underpinned by the Sight-Loss and Vision Priority Setting Partnership, the largest ever audit of the public’s priorities for ophthalmic research.8 The patient information sheets for our clinical trial programme of gene therapy for inherited retinal disease were reviewed by a past trial participant to ensure they addressed patient questions and managed expectations to improve the experience of future trial participants. At our recent patient information day on Stargardt’s disease, a patient and family panel co-developed our agenda to cover the most relevant patient questions about research. And, through patient and researcher discussions on the day, we identified that patients want more research on the factors influencing progression of Stargardt’s alongside current treatment development programmes.
In my opinion, PPI is particularly important for the future development of gene and cell therapies. Early phase studies have shown tolerable safety profiles overall for gene and cell therapies in the shorter term, but the rigorous follow-up and data collection common to these protocols are often onerous. This may bring unexpected challenges for recruitment and retention as patient numbers increase for Phase III trials. Longer-term implications of gene and cell therapy interventions for many conditions are still unclear. The incidences of leukaemia that occurred with the first X-SCID gene therapy, and more recently the reports of vision loss seen in AMD patients receiving intravitreal injections of adipose tissue-derived “stem cells”, highlight why patients must be involved in deciding what is an acceptable balance between risk and benefit.9,10
To me PPI should be part of our research methodology and a key influencer of research strategy. While the first steps on the road to PPI can start off challenging and uncertain, once established the benefits of PPI can be substantial.
- NIHR INVOLVE Coordinating Centre - Briefing Notes for Researchers
- NIHR INVOLVE Coordinating Centre - Resources Library
- Brett J, et al. (2010) The PIRICOM study: a systematic review of the conceptualisation, measurement, impact and outcomes of patients and public involvement in health and social care research. Report for the UK Clinical Research Collaboration
- Domecq J.P, et al. Patient engagement in research: a systematic review. BMC Health Serv Res. 2014; 14:89
- Buck D, et al. From plans to actions in patient and public involvement: qualitative study of documented plans and the accounts of researchers and patients sampled from a cohort of clinical trials. BMJ Open. 2015; 4(12):e006400
- Parkinson’s UK - Patient and Public Involvement: A Resource for Researchers
- Shared Learning Group on Involvement - Involving people in laboratory based research: a discussion paper
- Sight Loss and Vision Priority Setting Partnership - Full Report
- Check, E. Gene therapy: A tragic setback. Nature 2001; 420: 116-118
- Kuriyan A.E, et al. Vision Loss after Intravitreal Injection of Autologous “Stem Cells” for AMD. N Engl J Med. 2017; 376:1047-1053