PPI in Research: The Patient’s Perspective

What makes someone an ‘expert’?

The word can conjure up many different images. Is it someone with a PhD? A long, illustrious career? A Nobel Prize?

Or is it simply someone with an innate and intimate knowledge of a topic?

The definition of ‘expert’ is one which can divide the scientific community. Many believe that a researcher who has spent their life investigating a topic is an expert. Others believe that a patient who has suffered through a condition should be able to claim this title. But why not both? This is where patient-public involvement (PPI) can come in – taking multiple perspectives and using them all to create better outcomes for those who need them. But we are not yet at a place where this process is the norm.

Genomics research, and a huge amount of health research in general, has been plagued by these divisions. Rifts between scientists and the community have not been uncommon, with research often missing the mark. By involving patients and their loved ones in health research, these cracks can begin to heal.

In this feature, we’ll take a look at PPI in research through the eyes of Kate, a mother who was diagnosed with breast cancer 10 years ago and who had the opportunity to contribute to research as part of a patient advisory group.

Kate’s story

Kate lives in Scotland with her two sons and her beloved golden retriever. She once loved to hike and cycle, and found joy in her job as a teaching assistant. But ten years ago, Kate’s life changed after a devastating cancer diagnosis.

“I didn’t really have many symptoms at first, so it was a bit of a shock. The doctor didn’t think it was anything to be worried about,” she says. “Luckily, I caught it early, so even after my diagnosis I felt fine. I thought ‘this cancer thing is easy.’”

But after starting treatment, Kate’s health slowly deteriorated. Struggling to sleep, eat and perform her usual activities, her mental health began to suffer.

“I had to give up a lot – hobbies, nights out… I had to watch my friends doing stuff I couldn’t do anymore, and it was tough. I’d raised my children and thought I was going to get my life back. Not that I didn’t enjoy life when they were young, but it was a very different life than I imagined.”

Kate did not have a background in science and had never been particularly interested in the subject. When her health declined following the start of her treatment, this was met with confusion and sadness. Why was this supposed miracle treatment making her feel worse? “I really struggled to understand why I had to have such harsh treatment when I didn’t feel ill. I started to wish I’d never gone to the doctor. In hindsight, I understand, but I wasn’t educated enough on cancer at the time. I was following what the experts said, and I trusted them. But what did they know about my side effects?”

Kate’s experience is not unique. Whilst cancer therapies have come a long way in recent years, many of these treatments still cause an immediate decline in the patient’s quality of life, even if it ultimately extends it. Committing to these often toxic treatments requires trust between the patient and those who develop and administer them, Many choose, as Kate did, to simply “leave it to the experts.”

Research is not reality

Research and clinical trials often do not reflect real-life scenarios and can fail to account for certain nuances. This can lead to researchers assessing endpoints that may not reflect what patients actually want or need. For example, scientists may focus on statistics rather than tangible clinical benefit.

In other words, a drug could perform well in a research setting but fail to deliver actual improvements to patients’ lives. This is often the case if researchers fail to consider side effects or cost. These two factors can far outweigh any perceived benefit to the patient, but as these issues can be hard to quantify in a lab, they can be overlooked. In Kate’s case, the side effects of her cancer treatment, both physically and mentally, were worse than her early experiences with cancer itself. She felt betrayed by the very people who should have been helping her.

Couple these issues with confusion around results, and it is obvious why patients and their loved ones can sometimes feel less than pleased with their treatment, and the research process that led them there. These problems have led to bold claims that the majority of research that makes it to the clinical trial stage is not useful to patients, despite the time, effort and money put forward¹.

So how do we start to mend this trust and bridge the gap between patients and researchers?

Engaging patients in research

One day, during a routine appointment, Kate was told about a PPI group looking for women with her diagnosis.

“I actually thought at first that I would be receiving a treatment. That I would be a lab rat, basically, which I would have been happy to do! Then I remember being told, ‘no, they want to hear from you’.  I remember thinking, ‘what could they want to hear from me’? I’m no scientist!”

Kate laughed at the prospect. Her confidence in her own knowledge of cancer was shockingly low, despite having lived with the disease for five years. “I have plenty to say, but surely they know it all already?” she questioned.

PPI is defined as research ‘with’ patients and the public, not ‘for’ them. This is a particularly important distinction, as a patient (or their loved ones) can provide perspectives that often differ greatly from that of a researcher, especially one who has never suffered from the condition. PPI is becoming far more common in the research sphere, with a shift in attitudes about who the expert in the room is.

The positives

It may seem obvious, but let’s examine the reasons for undertaking PPI.

A primary goal of PPI is to reduce fear and misunderstanding. Science is often communicated poorly, with information trickling down from researchers to patients.

Appropriate PPI gives agency to the people most impacted by the research. Having patients involved from the start means that they can better understand the content and highlight any issues. Patients can also play a large role in further communication of the research they have been involved in, ensuring it is digestible for the wider community. This can help to prevent misconceptions that can arise from traditional communication of research.

Arguably, the greatest benefit of patient-public involvement is actually for the researcher. Patient perspectives and those of their family members can be invaluable in the research process and can educate the scientist on the true value of the work. Some researchers have reported that their experience communicating with patients has enlightened them and revolutionised their attitude and approach to research. This can improve efficiency and ensure the work is meaningful, which can equate to more funding in the future³.

This is a significant reason why many patients choose to volunteer when the opportunity arises, as their input can help not only with the current study but also to ensure that research continues and doesn’t dry up after one publication. In this sense, PPI is mutually beneficial; patients may be more willing to provide samples or contribute to trials if they know you are open to listening to them and have already created a solid relationship with the community².

Why are researchers reluctant?

PPI is yet to become the standard in research, despite growing calls for increased engagement. Despite what appear to be obvious benefits to the research community, many researchers are reluctant to engage patients or other “non-experts” in their research. There are concerns over the true value of PPI and tangible evidence of positive outcomes can be limited. Often, studies that integrated PPI only describe the methodological aspects of the process and do not properly report the outcomes, meaning the benefits are unclear³.

Kate reiterated this point: “We did some good work and had some good discussions but I’m not sure how much of it really ended up getting used in the research.” With the research field already plagued by overworking and underfunding, it is perhaps not surprising that scientists may shy away from something they can’t put a price on.

Researchers can often find the practice time-consuming and costly, and whilst a survey of principal investigators revealed that they did not feel a negative impact of integrating PPI into their work, they were not sure it provided any real benefit. That said, in the same survey 87% of respondents said they would happily partake in PPI again⁴.

The survey also reported that the main cost of PPI to researchers was time-related, but financial costs were also reported – these were typically attributed to reimbursement for the individuals involved. Another, perhaps more overlooked, cost is an emotional one. Researchers have reported high levels of emotional labour when collaborating with those directly impacted by their work, although most said they were happy to pay this price⁴.

Some researchers have also faced criticism by patients for treating the practise like a box ticking exercise, further alienating the very people who should be at the heart of the work. Some individuals who have taken part in PPI have reported that the researchers were arrogant, but many patients have acknowledged that this seems to be a changing pattern as younger researchers make their way through the ranks⁵.

This was reflected in Kate’s experience, who believes the young post-doctoral researcher leading the study was ‘doing it for the right reasons’. Her boss, however, was a different story. Described by Kate as a dinosaur, she believed he was treating PPI as a box ticking exercise to obtain funding.

“To listen to us would be to admit that there was something he didn’t know, and he didn’t like that. It’s sad, it’s really sad. Because there’s so much you can learn from seeing other perspectives.”

On the other side of this, some researchers have stated that it was upsetting to have their expertise brought into question and to be criticised by PPI volunteers. “Margaret”, a research fellow interviewed for a 2019 review on attitudes towards PPI, stated that it was a personal and professional “insult” when it was implied that she did not understand the condition that she spends her time working on⁴.

Barriers to patients

There are also participation barriers for patients, even when researchers are willing to include them. Some patients may be simply unable to participate in a study due to the severity of their condition. For example, if someone is bedbound or hospitalised then they cannot attend a meeting on a university campus.

Kate’s group chose to meet in person, an experience that she valued as it allowed her to meet with others in her position. “We met at the hospital. Just in a wee room. There were maybe 5 researchers and 5 patients. Just an hour or so every month. I didn’t have accessibility concerns at the time, but I would maybe struggle to do it now. At the time I was in a much better place so I could drive, but I couldn’t do that now. It makes me wonder if there were other people who wanted to be there but couldn’t.”

The rise of virtual meetings has somewhat mitigated these accessibility concerns, although it is important to remember that even a phone call can be exhausting for someone who is severely unwell. Patients have also discussed the isolation this can bring and the frustrating technological issues that have arisen in the hybrid era for those who are not tech-savvy. Kate is one of these individuals: “If you’re like me you don’t know how to use Zoom. I did one once and I hated it! I had to get my sons to help.”

Another significant barrier is advertising and recruitment³. Patients can often struggle to find out about opportunities, and researchers can similarly struggle to find the right people. A vast amount of advertising is based online, and this therefore assumes that patients will have access to the internet and the right forums. Often, the same volunteers will contribute to multiple studies, and researchers may rely heavily on their current connections when recruiting. This can lead to a lack of diversity in PPI groups and subsets of the community can still be left feeling ignored.

Applications can also be difficult to fill out. This alone can put people off taking part. Some patients have reported needing references for applications, abandoning forms when they reached this level of complexity⁵. These feelings often come to the surface when individuals are contributing their time voluntarily – so how can this be mitigated?

Making it worthwhile

Every effort should be made to make the PPI process as smooth as possible for those who are participating. However, given how time-consuming and tiring research can be, especially for those with a chronic illness, it is crucial that patients and the public are reimbursed for their time and contributions⁶. This has not always been the case; a 2018 survey of principal investigators revealed that just over half of those involved in PPI were financially compensated in some way and a third of PPI volunteers received expenses only. Tasks that were reimbursed included attending meetings or reviewing documents⁴. Proper financial compensation can go a long way in ensuring quality input from the public, a lasting relationship and a sense of appreciation.

However, the question of reimbursement comes hand-in-hand with concerns about the potential impact on any benefits one may receive. When dealing with severely unwell patients or their carers it is not uncommon for individuals to be in receipt of benefits that may be impacted by external payments⁶. This means that a clear and thought-out policy must be one of the first things developed when drawing up a PPI plan, whilst ensuring that any volunteers are properly compensated with minimal impact to their income.

Kate has a slightly different view on the idea of reimbursement. Her work five years ago was not subject to financial compensation, but Kate viewed her part in the future of cancer research as enough of a reward. She wanted to help people, and she feels that she did, even if it was just in sharing her story with a compassionate post-doc.

Success stories

Despite the fears of both researchers and the public, the reality is that we now live in a world where PPI it is becoming more commonplace, and researchers are becoming more willing to bend their usual practices to be more inclusive.

A good example of a PPI success story is that of the 2015 study by Gilbody et al., which assessed the use of computerised cognitive behavioural therapy in the treatment of depression. Cognitive behavioural therapy can be a controversial treatment for mental health conditions and obtaining this treatment via computer rather than in person with a trained professional is even more divisive. The team included patients with depression at each stage of the research, being careful to look out for particularly vulnerable individuals, and took their perspectives on board⁷. The study has been used as a good example of PPI statements in the BMJ PPI in Research document⁸.

A more recent example of effective PPI is the COVID Public Involvement Matching Scheme. This was set up in the wake of the COVID-19 pandemic to combat a decrease in PPI opportunities as well as a lack of PPI in COVID-19 research itself. The scheme helped to “match” volunteers to COVID-19 studies, in situations where the research was too urgent to go through the usual relationship-building and approval processes typically involved in PPI. Six months into the scheme, PPI was part of 85% of COVID-19 grant applications. It highlighted the need for public involvement in health research and the reliance researchers have on their current connections for recruitment⁹.

Kate is unsure of the status of the study she was involved in. After her contributions, she suffered a relapse. Being generally uninterested in science, she stepped back from papers and manuscripts and decided to enjoy life with her family. But she would describe the experience as success story. “Even if all I did was help that post-doc get a paper published or earn a prize, then it was worth it. I’m glad I did it.”

What does the future hold?

Many funding bodies are now expecting PPI to be integrated in research and included on grant applications¹⁰. Although this may seem a forceful approach, with concerns raised over tokenism and box-ticking, researchers are now consistently responding to surveys stating that they are happy to participate in PPI. Misconceptions around the practice are beginning to be resolved and researchers are starting to feel more comfortable questioning their own expertise. Jennifer, another researcher interviewed about their experiences⁴, stated that collaboration with patients “raises [her] enthusiasm to battle the challenges.”

There are still problems to be addressed, such as the need for better reporting to quantify the value of PPI. For example, in a 2020 metastatic myeloma clinical trial¹¹, the team assessed the role of PPI in practice. Overall, the study reported positive results and the involvement of patients in the research process was seen as beneficial. However, a lack of real evaluation tools meant that the impact of PPI could not be accurately reported. The researchers had gathered their data via a workshop and through conversation with the participants, but this method of evaluation was deemed insufficient to gather real opinions, especially when the comments made were not anonymous.

To combat these issues, various groups have suggested frameworks for reporting on the costs, benefits and outcomes of PPI. One of these is GRIPP2, the first internationally recognised guidance on PPI reporting¹². The framework was developed in both a short- and long-form, containing 5 and 34 questions respectively. The reporting guideline includes questions related to the aims of the study, the methods and results and the context of the research. The goal of such frameworks is to encourage consistent reporting across different fields to define real benefits of PPI.

So, who is the expert?

Kate had never considered herself an expert. “Maybe an expert on side effects!” she joked. “When you think ‘expert’, you usually think of someone who studied it their whole life. I’m just someone who was unlucky enough to have to have cancer treatment.”

But looking back on the experience, Kate’s mind changed. “How could I not be an expert? I could tell you all the symptoms, all the treatments, I’m definitely an expert!”

The world of research is slowly but surely moving into a new era. Researchers and patients are, now more than ever, putting their differences aside to produce meaningful work and combat tireless misconceptions. It is clear that, even if you are an expert, there is always more you can learn.

As for Kate, she’s found new joy in her life. She has taken up knitting, and her sons have moved home to be with her. She is happy and has no regrets about her journey.

References:

1. Heneghan, C., Goldacre, B. & Mahtani, K.R. Why clinical trial outcomes fail to translate into benefits for patients. Trials 18, 122 (2017). https://doi.org/10.1186/s13063-017-1870-2
2. NHS Health Research Authority. Public Involvement. Available at: https://www.hra.nhs.uk/planning-and-improving-research/best-practice/public-involvement/
3. Blackburn, S., McLachlan, S., Jowett, S. et al. The extent, quality and impact of patient and public involvement in primary care research: a mixed methods study. Res Involv Engagem 4, 16 (2018). https://doi.org/10.1186/s40900-018-0100-8
4. Boylan, A.M., Locock, L., Thomson, R. and Staniszewska, S., 2019. “About sixty per cent I want to do it”: Health researchers’ attitudes to, and experiences of, patient and public involvement (PPI)—A qualitative interview study. Health expectations, 22(4), pp.721-730.
5. HealthTalk. Patient and public involvement in research. Available at: https://healthtalk.org/patient-and-public-involvement-research/difficulties-and-barriers-to-involvement
6. NIHR. 2022. Payment Guidance for Researchers and Professionals. Available at: https://www.nihr.ac.uk/documents/payment-guidance-for-researchers-and-professionals/27392
7. Gilbody, S., Littlewood, E., Hewitt, C., Brierley, G., Tharmanathan, P., Araya, R., Barkham, M., Bower, P., Cooper, C., Gask, L. and Kessler, D., 2015. Computerised cognitive behaviour therapy (cCBT) as treatment for depression in primary care (REEACT trial): large scale pragmatic randomised controlled trial. Bmj, 351.
8. BMJ. Reporting patient and public involvement in research. Available at: https://www.bmj.com/sites/default/files/attachments/resources/2018/03/PPI_in_Research.pdf
9. NHS Health Research Authority. 2021. Public Involvement in a Pandemic. Available at: https://s3.eu-west-2.amazonaws.com/www.hra.nhs.uk/media/documents/8948_Public_Involvement_in_Pandemic_Research_Report_V9_-_Accessible.pdf
10. Gray-Burrows KA, Willis TA, Foy R, Rathfelder M, Bland P, Chin A, Hodgson S, Ibegbuna G, Prestwich G, Samuel K, Wood L. Role of patient and public involvement in implementation research: a consensus study. BMJ quality & safety. 2018 Oct 1;27(10):858-64.
11. Skovlund, P.C., Nielsen, B.K., Thaysen, H.V. et al. The impact of patient involvement in research: a case study of the planning, conduct and dissemination of a clinical, controlled trial. Res Involv Engagem 6, 43 (2020). https://doi.org/10.1186/s40900-020-00214-5
12. Staniszewska S, Brett J, Simera I, Seers K, Mockford C, Goodlad S, Altman DG, Moher D, Barber R, Denegri S, Entwistle A, Littlejohns P, Morris C, Suleman R, Thomas V, Tysall C. GRIPP2 reporting checklists: tools to improve reporting of patient and public involvement in research. BMJ. 2017 Aug 2;358:j3453. doi: 10.1136/bmj.j3453.