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Exploring the purpose and meaning of patient engagement in pediatric neurodisability research

The following is text from a lecture I gave yesterday here in Toronto, at the Brain-Child-Partners conference. The conference is a joint presentation of Child-Bright and Kids Brain Health(formerly NeuroDevNet) – research networks that study child development and brain-based disabilities.

This talk borrows sections from a previous one: “Two-way street? A critical look at patient and family engagement.” Concepts developed in that lecture are applied here to pediatric neurodisability researchers. If you read both you will see a bit of overlap – however each essay makes different points.


Introduction

I’m Jennifer Johannesen, and I am a parent to a young boy, Owen, who had multiple severe disabilities all his life. He died almost exactly 7 years ago, when he was 12 years old. He had many obvious disabilities, but there was no clear cause – for his disabilities, or for his death.

I wrote about our experiences in my book No Ordinary Boy, in which I process the relentless decision-making, worries and absurdities that came with the territory. Since then, I have been interested in exploring critical issues in healthcare, particularly related to children and families in the clinical setting. As part of that exploration, I recently completed a Masters in Bioethics, and write and lecture on critical thinking in clinical practice, patient and family centered care, and patient engagement.

Owen’s official diagnosis was cerebral palsy, although there was no evidence of brain injury or trauma, and genetic testing didn’t lead to any insights. It seems to surprise people when I tell them it didn’t matter to me that we had no diagnosis. The truth is our lives did not circle around his medical conditions specifically – we were far more concerned about his experience of the world, and how he would access the things that seemed so far out of reach. We didn’t believe that, in his case, diagnosis would make a difference.

So my talk today is not about the importance of patient engagement in research. Rather, I’m interested in a broader, more critical discussion. In addition to asking ‘how do we do it better’, I propose we also need to ask ‘why do we do it at all’ – which is to say, what is the purpose and the meaning.


To get to some clarity, we’re going to explore 2 underlying questions:

Why do researchers want to engage patients?
Why do patients want to be engaged?

The answers to these questions will lead us to explore implications for research, and some possible ways forward.

A quick housekeeping note: I’m defining ‘patient engagement in research’ as patients being enrolled by institutional or academic researchers as advisors on their projects. Not as volunteers who donate labour, not as qualitative research subjects (as in focus groups), and not as peer-to-peer support. I’m talking about patients who are invited in to advisory councils, steering committees, or even as partners, to provide input on shaping research goals, design, and knowledge translation.


So let’s get started:

Why do researchers want to engage patients in research?

  1. Funding/publishing requirements

This first answer is obvious. Researchers need to meet funders’ or publishers’ criteria. While it’s not exactly law, at some level researchers understand they have to engage patients.

But this answer essentially amounts to “we need to do it because we need to do it” – which is unsatisfying. Just because something is required doesn’t make the rationale for it any clearer. We’ll need to dig deeper to get at why it’s considered mandatory.

  1. Improved outcomes?

If someone says the benefits are self-evident, this might be what they mean. Esmail et al published a literature review related to patient engagement in research, and found virtually no evidence, but lots of hypothesized (or imagined) impacts – I borrowed this list from them:

  • Better quality research (more relevant and responsive to patient needs)
  • Expanded applicability (hard-to-reach populations)
  • Empowering patients
  • Increased translation, dissemination and update of results
  • Democracy and accountability
  • Fulfilling a moral obligation

Virtually none of this has been validated, convincingly and conclusively. That’s because it’s not measured – or it’s only reported in retrospective accounts of engagement experience or informal observation.

Claims of improved outcomes are largely unproven.

  1. Good feelings

This is maybe getting closer to a truth.

Patient engagement helps patients feel they have a voice. This claim is spoken time and again. They feel included. And, regardless of whether they are or not, they feel empowered. By the way: we’re not talking about all patients, only the ones who are involved.

And I’ve heard researchers say they feel grateful and humbled that patients give their time and energy to their projects. And it can be so helpful to have collaborators to help shape and communicate the work.

So we might agree there’s a lot of good feelings and respect going around – but is that enough to justify patient engagement in research?

I’m still not satisfied with this list.

This is a discipline where evidence is everything – it’s the basis on which we make diagnoses, implement protocols, change practice – yet we have this imperative, issued by mostly by funders – to engage patients in research at advisory levels. This imperative is unsubstantiated as to its effectiveness or impact. Because no one can point to outcomes, we become overly focused on the good feelings it generates.

I don’t think this can be the whole story. ‘Patient engagement’ doesn’t’ exist on its own – it’s something people created and inserted into healthcare. It is not a random phenomenon that exists in the wild. There would be no reason to integrate it into research or practice unless there was some other benefit.

  1. “Virtue signalling”

My theory is that research projects get a considerable amount of mileage from something known as ‘virtue signalling’. This notion originated in evolutionary biology but it’s recently been popularized in the media and used in social commentary.

“Virtue signallingis the conspicuous expression of moral values done primarily with the intent of enhancing standing within a social group.”

Let’s see how this could apply to patient engagement in research.

“conspicuous expression”

When an institution engages patients and families, they communicate it – excitedly and extensively. You can always tell what efforts are “patient engagement” efforts – maybe an event sponsored by a family advisory, or a pamphlet developed ‘by patients for patients’. Patient engagement seems to only have value when its presence is communicated widely. This benefit accrues to the institution regardless of impact.

In the research context, this ‘marketing’ appears in the form of explicit statements on funding applications and in published research, backed up by letters and personal testimonies of patients.

The ‘conspicuous expression’ is clear to see.

“moral values”

Certainly, there is moral value in patient engagement, which is why I think we’re so quick to extoll its virtues without having to be specific. The actual value may vary: it conveys benevolence, or open-mindedness, or fairness, collaborative-ness – fill in the blank. It’s shorthand for ‘we care about our patients’ – because it’s principles-based, there has been no need to substantiate it via outcomes.

“intent to enhance standing”

In the wider healthcare context I hesitate to attribute intent – ‘patient engagement’ is a leaderless movement and there’s no one pulling the strings. In the case of research, however, we need to look more closely at motivations.

Imagine 2 similar research projects, side by side, one with some form of patient advisory, and one without. The one with will have better standing among peers, patients, funders and publishers. Regardless of the impact of the contribution of those patients.

Researchers get higher credibility, higher chance of obtaining funding, increased opportunities of getting published and cited – which helps to feed the demanding requirements of academic publishing. Moreso in research than in other areas of healthcare, the main stakeholders – the researchers – depend on patient engagement to support their work, reputation and even careers.

I’m not criticizing ambition or the need to meet requirements – rather, I’m commenting on the conditions under which researchers are pressured to differentiate themselves. The stamp of ‘patient engagement’ further legitimizes work, unlocks funding sources, and accrues high reputational dividends to the researcher and institution.

All things considered, patients are wanted for their symbolic value. Anything else is incidental, including the good feelings. I say this confidently because research communities have done little to indicate otherwise.


Here’s how we know that patient engagement in research is largely symbolic and not intended to be consequential:

  • Lack of literature: There is little evidence to substantially support or explain why patient engagement should be required, especially to the degree that it is.
  • No quality or protocol oversight: as in REBs for example There is no requirement for quality. No protocols to govern how patients in advisory roles are selected and trained. No mandate for patient advisors to be representative of patient populations. No direction on how their perspectives are elicited and integrated.
  • No measurement: If the actual output and influence of patients are consequential, their work should be subject to the same rigorous methods we ostensibly apply to development of protocols, ethics review, conduct of researchers, etc. This is neither tracked nor reported.
  • No compensation: Let’s assume for the moment that each person in a project feels equally passionate and dedicated to the work – and each is respected for their expertise, whether earned through education and training, or experience. Everyone is fondly called an “equal partner”. In this scenario, however, only the researcher is paid for the work and gets reputational credit in ways that are relevant to them. Despite any extra hours a researcher may be putting in, they are not volunteers. Patient partners get thanks and praises, the privilege of contributing to a greater good, perhaps an acknowledgement or even shared authorship – which is nice but has no real currency as a lay person.

Literature, protocols, measurement, funding. In a field where all of these things are foundational to integrity and relevance – none of it applies to patient engagement.

Regardless of good feelings and possible positive outcomes, patients are needed only for their symbolic value, and everything else is incidental.


I think it’s a terribly awkward moment for researchers – as ‘patient engagement’ makes its way through healthcare it’s landed in research without a mandate and without structure. It’s no surprise that many researchers don’t know how to handle it well.

This is an email I received from a researcher this summer – someone with whom I’ve collaborated on an unrelated paid project. I’ll read the first paragraph:

“Hi Jennifer,

We are putting in a research grant […], and it would be great if we had a patient advocate on the team.Obviously, I thought of you…. :-)Unfortunately, there is not great money in there, but you would obviously have an opportunity to be involved in the orientation and the translation of the research results. Could you let me know if this would be of interest to you?

In the short term, not much would be required. I am putting together a grant application for which a support letter would be sufficient. In the longer term, depending on how the project evolves, we would be expected to meet a couple of times to discuss progress, avenues, knowledge translation etc.”

About 3 weeks later I received a follow up note asking for a supporting letter, with some helpful suggestions of what to say: stress our expertise, this is relevant to Canadian patients, a high chance of success, and my role as strong advocate.

“It would be really helpful if you could submit a support letter for the final submission. Below are some ideas of what you could put in there:

  • stress our expertise particularly now that our program has started
  • state that this is a timely and important project, how it is relevant to Canadian patients (from your patient perspective) – we are trying to prevent disease
  • that this is a strong consortium with all the necessary expertise and that the project has a high chance of success

And then obviously your role as a strong patient advocate, how you can give us the patient perspective and guide us in knowledge translation, but also eventually in methods for patient recruitment etc. 

10-15 lines would be enough.


To be fair: there’s a lot of shorthand here because we know each other. There’s a feeling of ‘cutting to the chase’ – even so: it’s hard for the researcher to suppress the impulse to cajole, convince, flatter, apologize for no money, even as I’m being told exactly what to say.

Btw this request was for basic science research, using mouse models. I had nothing helpful to contribute, and we both knew it.

This is a comical example, but it’s also rather elegant in the concise way it embodies what I’ve been talking about. It simply does not matter what I contribute materially to this project.

I am being utilized for my status as a well-known patient – to help this researcher’s project move forward by accessing grant money, and to help validate the published results, whatever they may be.


So – we’ve looked at why researchers want to engage patients. Let’s move to the second question.

Why do patients want to be engaged? What do we get out of this?

  1. Gratitude

We’re grateful. Many of us feel indebted to the people that saved or treated our children. A kind of intimacy forms, and we want to express our thanks.

  1. Flattery

We are also flattered – often we’re invited to join these projects personally, and it feels good to be recognized as someone whose ideas are valued.

  1. Access

We are also granted a special elevated status – participating behind the scenes potentially gives us access to information that the general patient population doesn’t get.

And more than that: once we’re on a first name basis and working side by side with our own child’s specialist, we establish an informal personal relationship that likely has a positive affect on how we feel about our own position and the care we receive. We get to be insiders, we get special attention and appreciation, and we may feel anointed as perfect patient partners.

  1. Quest for answers

The quest for answers or clues regarding our child’s diagnosis and treatment can be all-consuming – it can lead us to participate as research subjects, and also want to be involved in other aspects of research design and delivery.

  1. Community

For some us, the hospital is our home for long stretches of time – we feel welcome, it’s familiar and even reassuring. When my son died and I no longer had the same reasons to go to Sick Kids or Bloorview again, I felt a tangible loss. Not just for the change in routine, but for the loss of a community of people who were always pulling for us. I think some of us gravitate back to the setting where we felt most supported.

  1. Altruism/charity

We are also keen to make a difference. And we know that so much of healthcare relies on volunteers to get important things done.

  1. Agents for change (added ex post facto, thanks Julie Drury!)

(As Julie mentioned in her own comments at the conference, some families experience things they hope others will never have to endure – so they may participate to spark change.)


The point is this: these motivations of ours are often borne out of deeply personal and perhaps traumatic experiences. I’m not claiming that we are being exploited, but I confidently say that we make an ideal group of people who can be relied upon to give generously and continuously.


We’ve looked at researcher and patient motivations, and landed in an interesting place.

I’m not suggesting there are no positive outcomes. Patients and researchers alike talk about enjoying interpersonal connection, learning from each others’ perspectives, and appreciating the productive dialogue that emerges from having shared interests.

But:

  • If we agree that researchers extract significantly more value out of patients than vice versa,
  • and we agree that evidence in support of patient engagement is flimsy,
  • and we acknowledge that patients – even eager ones – are a vulnerable group
  • then the least we can do is critically consider the ethical implications.

What are the implications of this?

  • First of all: patient advisors may not be representative of patient populations.
    • This is because researchers tend to invite people they know and are not recruiting a representative sample, nor are they asking that patient advisors be elected democratically.
    • As well, self-selection bias leads to the formation of a particularly homogenous group of well-educated, articulate, wealthy people who can afford to volunteer, and who consider their contributions to be charitable. It’s unseemly to ask for money, and it’s unseemly to offer. Everyone seems embarrassed by it. But the longer this goes unsolved, the more we reinforce 2 problems: inequity in the so-called partnerships between patients and researchers, and inequity in terms of who gets to benefit from special access privileges.
  • Second: Special relationships privilege certain kinds of patients who are likely already advantaged
    • Patients who are invited into the inner circle already come from a socioeconomic demographic that allows them – us – to further take advantage of own special status. This is how privilege extends and perpetuates itself. We take the space of people whose presence may actually help to redress some of that unfairness.
  • Finally: Terms of patient engagement are purely relational
    • There are no rules, so the degree and type of engagement will depend entirely on the priorities and personality of the researcher, and willingness of the particular patient.

Pediatric neurodisability

For pediatric neurodisability research, there are additional, unique concerns –our children’s conditions may be chronic and life-long, significantly affect other family members, and may be accompanied by other issues, such as co-morbidities, impact on school, family and friends, participation in community activities, and transition to adulthood. Here are some special concerns:

  • Priorities and perspectives change over time
    • This is an important concern for our population – things that feel urgent at one time give way to other concerns. Our opinions on what matters change over time. Our recollections even about factual events morph into different narratives. So which perspectives are considered and at what juncture in our lives will surely inflect research differently.
  • Parents may have insufficient psycho-social support as they engage in research projects while processing grief, gratitude or a host of other emotions
    • Studies have shown that patients sharing their story or perspective in a dialogical encounter has therapeutic value in and of itself. We might assume there are possible harmful effects if that encounter is not skillfully handled. How are we protecting, or at least not harming patients and families through their freeform participation in research?
  • Extensive engagement in research keeps families in the sphere of the biomedical.
    • Many families like mine don’t think of their problems as exclusively medical – they’re social, economic, relational, related to access and supports. If a family is involved in a years-long study on the physical effects of cerebral palsy on the skeletal structure, for example, their experience of their child may be shaped and influenced in ways we don’t readily see. So again, what supports are in place for those families?

For me it boils down to this:

It surprises me deeply that there is no oversight governing the involvement of patients and families in advisory roles in research, when the protection afforded them as research subjects is paramount. This lack of oversight is what keeps ‘patient engagement’ in the realm of the symbolic, and prevents it from become more substantial. And conversely, because patient engagement is largely symbolic, there’s no perceived need for oversight.

We don’t need to abandon patient engagement in research. I think we need to take seriously the risks these implications pose to the integrity of research. We also need to take seriously the contributions we’re asking of patients – are they consequential, or not. Are they merely symbolic, or not.

I offer some ways forward, for both researchers and for patients and families.

Ways forward for researchers

  1. Professionalize/train patients
    • Stop privileging certain patient voices over others because it’s easier, and stop trading on personal relationships. Instead, get feedback and support through patient review boards – which I know exist – or via appropriate paid training for patients
    • I place this responsibility on the research community intentionally, as I think it’s up to you to decide patient advisory is worth the investment
  2. Get clear on patient representation
    • Do your advisors actually ‘represent’ the patient population, either statistically or democratically? Are you counting on 2 or 3 people to be ‘the patient voice’? When it comes to patient advisors, get crystal clear on who and what they represent, and be transparent about it.
  3. Be accountable
    • Find ways to make absurd requirements meaningful by going beyond the mere checklist and ensuring that patient input is consequential to the project.
    • Make sure that patients understand what they are there for and what you want from them.
  4. Push for standards and protocols
    • Funders and publishers should be challenged to produce a rationale and evidence to justify any patient engagement requirements
  5. Contribute to the literature on patient engagement in research
    • Include intentional, prospective studies of patient engagement in any project in which you include patients, and publish it

Ways forward for families

  1. Don’t allow flattery to prevent you from asking important questions or facing uncomfortable truths

First: don’t allow flattery to get in the way of asking important question or facing uncomfortable truths. Ask why you were selected. Don’t accept that efforts to diversify are too hard. And don’t accept that you can’t be paid fairly for your time and contributions. No matter how discrepancies are explained, remember that researchers are being paid in some fashion, and are not volunteering. They may love what they do, but they would not carry on researching if they weren’t employed to do so.

Remember: this is deeply intertwined with lack of representation. If contributing to research is only available to those who can afford to volunteer, other voices will never be heard.

  1. Insist on accountability

Second: insist on the accountability I just spoke of. Make sure you fully understand the project. Make sure certain voices are not being privileged over others, including your own. Determine for yourself if your contribution is actually consequential.

  1. Be prepared to say no

If there is insufficient effort to justify and substantiate what is being asked of you, or it’s unclear as to why you’re involved, you can decline.


The purpose of patient engagement in research – including pediatric neurodisability – is to extract symbolic value from patients in order to access funding and enhance legitimacy of research projects. Positive outcomes and good feelings are incidental.

Of course, I may be wrong. But if our contributions are not symbolic and are in fact consequential, then we have other, bigger problems: inequitable compensation, absence of oversight to ensure appropriate and representative patient influence and contribution, lack of protection of patients and families, and unfair privileging of some kinds of patients over others.

Patients in advisory roles seem to be enablers and cheerleaders – and of course, handy free labour. Rather, I think patients should be the conscience, and the critical voice. It remains to be seen whether there is a will for change.

Jennifer

22 Comments

  1. This is awesome, thanks for sharing your insight! I hope researchers and intending researchers learn from your wealth of experience

  2. Really thought provoking and articulated thoroughly and clearly. Your views are issues we grapple with over here too in terms of genuine and effective patient and public involvement in healthcare research and co-design and co-production. In our effort to continually train staff on good patient public involvement as well as inform and activate public involvement, it would be great to hear you speak in the England if you over here at some point.

    • Thanks for commenting! It’s interesting to note how universal some of these issues are. Since writing this, I wonder if institutional efforts at ‘authenticity’ will always feel suspect, regardless how earnest – so what to aim for instead is transparency and clarity, backed by solid governance. Lots more thinking and dialogue needed!

      I’ll update on Twitter if I’m ever headed that way :) @jenjohannesen

  3. Thank you. This is a very clearly articulated and thoughtful piece that will be of great use to me in training researchers to think carefully about engagement in various forms.

    • So important to introduce and discuss these ideas during training and early years…! Thanks so much for reading and commenting.

  4. I’m a UK health services researcher currently planning a talk on patient and public participation in research – based on some consensus panel work we’ve done with our patient and public panel, and essentially making the case for better evidence and more clarity. Otherwise we’ll continue to inhabit Tokenism City. I read your piece and concluded that I should just read it out instead! Thanks for sharing.

    • What! That’s amazing :) Thanks so much for letting me know. I’ll be curious to hear how it goes, if you care to share any feedback? Hope it sparks some discussion. Good luck! Jennifer

  5. This is the most thoughtful piece I have read in a while. Thanks for making it available to a wide group.

    • Thanks Glenda, I appreciate the comment! I share my work as widely and openly as possible.

  6. Your talk is excellent. Thank you. The things you have said need saying. All power to your elbow!

    If you haven’t come across this paper you may be interested in the evidence presented. Crowe S, Fenton M, Hall M, Chalmers I. Patients’, clinicians’ and the research communities’ priorities for treatment research: there is an important mismatch. Research Involvement and Engagement 2015. 1:2. DOI 10.1186/s40900-015-0003-x http://www.researchinvolvement.com/content/1/1/2. I am encouraged by examples of the James Lind Alliance research Priority Setting Partnerships beginning to impact research agendas. http://www.jla.nihr.ac.uk/about-the-james-lind-alliance/

    • Hello Iain! Thanks for your comment and links. I was delaying responding until I had a chance to click through. I absolutely see the value in patients indicating their own research priorities. I’d be interested to know to what extent the patient side of the partnerships are truly representative of patient populations, and if there is evidence to support this partnership model over simply surveying patients. I’ll do some more reading :)

  7. Hi Jennifer, thank you for saying so well what a lot of us are thinking. We are working hard at the moment on many of these issues in a Patient Group I am on here in the UK, with Public and Researchers working together to develop some Training and Guidance. We will share your words and thoughts as widely as possible, they will help a lot. We will do this in honour of your Owen, and of the many people affected by so many different health conditions. Thank you.

    • Thank you for your kind words, and of course for the ongoing work of holding all stakeholders to account! Encouraging to hear that you and your colleagues are seeking ways forward.

  8. I have been involved as a lay person in health research for sometime. Your talk reflects many of my concerns that have developed over the years. The main one being lack of evidence. I have from time to time raised these but often this type of contribution is not discussed further or dismissed as “really interesting” but no further discussion. I have a neurological condition and as a child belonged to the paediatric neurological community.Thank you for your talk.

    • Thanks for reading and commenting Georgina – you’re definitely not alone in feeling this way. My own opinion is that “really interesting” with no action means that indeed, most patient engagement activities are not actually of consequence. I hope you eventually find someone in your circle who takes this seriously!

  9. Wonderful food for discussion… if only the appropriate people will discuss.

    Your comments on selection bias in identifying patients to engage is absolutely true for the fight against ALS in the US. It’s so convenient to pick families that have been good fundraisers for ALS organizations. It’s so easy to overlook families that will actually ask the difficult questions and challenge the status quo.

    • Thanks for your comment! My own experience is in institutional healthcare, where fundraising plays a less prominent role among patient advisors – but of course these dynamics play out everywhere. Appreciate the reminder.

  10. Hi Jennifer, as someone involved in Public, Patient Involvement in England since 2008 I found your article persuasive and thought provoking. Much of what you say can be carried through to PPI in the wider Health and social care arena.
    May I have your permission to share your article within my local PPI circle as I think many will say it articulates what we see as tick box PPI.

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