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Feature

The patients bringing lived experience to research teams

BMJ 2024; 386 doi: https://doi.org/10.1136/bmj.q1406 (Published 03 July 2024) Cite this as: BMJ 2024;386:q1406
  1. Eva Amsen, freelance journalist
  1. London
  1. eva.amsen{at}gmail.com

Patients are increasingly acting as co-leads on research projects, but the infrastructure to support these collaborations is still in its infancy. Eva Amsen reports

In 2015, when the Cambridge Clinical Trials Unit launched its Patient Led Research Hub (PLRH) it noticed that patients were interested in research topics that weren’t covered by most published studies. “Most patient priorities were around quality of life and symptom management, whereas a lot of funded projects were about interventions and drugs,” says Laura Cowley, research lead at PLRH.

A similar discrepancy had come up in the Netherlands in 2006, when the Dutch Burns Foundation invited patients to help set their research agenda.1 It learnt that this group’s main concern was itching, which wasn’t a research priority at that time. “It’s such a good example to show that people with lived experience ask very different questions,” says Sanne Steenhuisen, programme secretary of the participation and citizen science initiative at the Netherlands Organisation for Health Research and Development (ZonMW).

Many organisations and patient groups around the world, including PLRH and ZonMW, are now creating better ways for patients and researchers to collaborate.

Beyond box ticking

“Patient led” now means that research is driven by patients or by people with lived experience. This might be in consulting roles or as co-researchers. It goes further than merely encouraging researchers to incorporate patient and public involvement (PPI) in their work—something that is already becoming common practice at many medical research funders.

Funders that ask for PPI plans include Australia’s National Health and Medical Research Council, the Canadian Institutes of Health Research (CIHR), the UK’s National Institute for Health Research, the National Institutes of Health in the US, ZonMW, and many others, including charitable research funders.

While asking funding applicants about their PPI plans encourages researchers to think about patient involvement in the early stages, there is often very little follow-up2 and no guarantee that researchers will actually involve patients. “It’s often seen as a box ticking exercise and we want to change that,” says Steenhuisen. “Although funders say they want a PPI plan or evidence of PPI, it’s still very tokenistic,” agrees Cowley.

PLRH is taking a more active approach to getting patients involved. Its current focus is on delivering clinical studies in collaboration with rare disease patient groups.

“Originally, the ethos was to take on any research idea from any patient group,” says Cowley. “But that got too big too quickly.” Since most of the feasible research ideas came from rare disease groups, it decided to limit the scope. PLRH now takes research suggestions from rare disease patient support groups and facilitates the formation of equal partnerships between patients and research groups if the idea is feasible.

Patient groups are becoming research partners

Organisations involved in patient led research are exploring different ways of giving patients an active role. Some initiatives are led by patient communities, such as the Patient-Led Research Collaborative for Long Covid3 (see box 1). Others are spearheaded by research or funding organisations that take research topic suggestions from community and patient groups, involve them as equal research partners, or both.

Box 1

Long covid

Long covid research has been patient led from the beginning.

People who did not fully recover after a covid infection found each other online and created communities to drive awareness. One of these online forums branched out into the Patient-Led Research Collaborative, which facilitates patient led research into long covid by working closely with a group of researchers.

Other members of the long covid community also recently organised the Unite to Fight5 conference, where researchers and others shared updates on long covid and myalgic encephalomyelitis/chronic fatigue syndrome research.

One of the speakers was Alison Cohen, epidemiologist at the University of California, San Francisco. Cohen worked with the Patient-Led Research Collaborative on a report documenting 13 case studies of people who tried nirmatrelvir/ritonavir (Paxlovid) as a long covid treatment, currently available as preprint.6 “I’m particularly proud of this work as a model of patient driven research—many of the people whose experiences are documented were also involved in the paper as co-authors,” says Cohen. “When patients and other people with relevant lived experiences are involved in research, it makes the research stronger.”

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ZonMW, for example, is currently running a process to connect researchers on youth matters with community members who have suggestions for research topics. “One of our goals is to expand this to other topics,” says Steenhuisen. Other funders are taking similar steps. Diabetes UK, which is already flexible about including patients as co-applicants on some of their funded projects, is broadening its scope to include more people with lived experience.

Meanwhile, the Strategy for Patient-Oriented Research (SPOR) Evidence Alliance in Canada routinely includes patient partners in knowledge synthesis projects, but has recently also selected several new research topics that were submitted by patients or community members who are co-leading their chosen project with a research team.

Becoming equal partners in a research team is currently more common in areas with tangible and applicable outcomes, such as knowledge synthesis at the SPOR Evidence Alliance or clinical effectiveness research, the focus of the Patient Centred Outcomes Research Institute (PCORI) in the US.

PCORI includes patients as advisers in study design and implementation; on some projects it also involves community organisations as co-leaders. “It represents a model that we may be able to repeat over time,” says Harold Feldman, deputy executive director for patient centred research programmes at PCORI.

But while all these organisations and projects are exploring new ways of collaborating and co-creating with patient communities, they’re also identifying new challenges.

A system unfriendly to patient led research

Patients can be involved as advisers in setting research directions, in providing feedback when approached, or even in co-leading research projects. That level of involvement deserves compensation, but this is not a straightforward process. Paying patients for their involvement may affect any government benefits they are receiving. That’s why some organisations give patients the choice between remuneration or gift cards, for example.

“We try to find different options to pay people for their time, and we ask what their preference is,” says Andrea Tricco, principal investigator for the SPOR Evidence Alliance and scientist at St Michael’s Hospital, Toronto.

Problems also arise when patients are co-applicants on grant proposals, because existing funding systems aren’t set up for applicants without institutional affiliations. “When patient partners apply as co-applicants, they need to submit an academic CV, which is just not appropriate,” says Cowley of UK funding systems. “And there’s no box for PPI. If you don’t have a medical background, you select ‘other.’ It’s such an unwelcoming first interaction for a patient.”

The situation in Canada is similar. “Right now, patients can’t necessarily hold funding unless they’re part of a formal affiliation or group,” says Tricco. “But I do know that that’s something CIHR is looking into.”

Another roadblock to further implementation of patient led research is that the research community is only gradually becoming aware of how it can collaborate with patients. “It has to become more normalised,” says Steenhuisen. “Why would you do research without involving the people that the research is about?”

“Unfortunately, a lot of research groups are still new to the idea of partnering with a patient or having patients involved at all,” says Dana Lewis. Lewis, who has type 1 diabetes, has worked closely with researchers for the past decade to develop and test a closed loop artificial pancreas system (see box 2). But when she was also diagnosed with exocrine pancreatic insufficiency, a digestive condition, she found it much harder to collaborate with gastroenterology researchers. “It’s like a different universe,” she says.

Box 2

OpenAPS

In 2013, Dana Lewis was not able to hear the alarms on the continuous glucose monitor that she used to manage her type 1 diabetes.

Together with Scott Leibrand she found a way to pull data from the device and build a better alarm system. Further modifications connected the system to an insulin pump to create a fully functional closed loop automated insulin delivery system.

“We spent a lot of time thinking about safety as it evolved to a closed loop system,” says Lewis. “We wanted people to have the advantage of building from the safety based design that we had.”

That was one of the reasons Lewis made everything openly available through the OpenAPS project, where anyone can download the software they need to run the system on either a small hardware device or through an Android phone. Even now that commercial closed loop systems are available, thousands of people are still using OpenAPS because it’s more accessible and affordable.

Lewis connected with researchers and clinicians at conferences where she was invited to speak. This network made it possible to fund and carry out a clinical trial with Lewis as co-investigator. The results of this trial further validated the effectiveness of the new Android based OpenAPS system.

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This lack of familiarity among the research community is something that the SPOR Evidence Alliance is hoping to tackle. “We’re trying to change the research community to be more accepting of patient engagement,” says Tricco.

Organisations and funders are learning where the bottlenecks are in managing patient led research. They’re working on building fair compensation systems, supporting researchers, and evaluating best practices.

PCORI is also funding research into the science of engagement,4 to learn more about the best ways researchers and funders can work together with patients in research planning and execution. “We need to take the evidence and the findings from those programmes,” says Feldman. “Then we can weave that back in so that we can do an even better job of facilitating and promoting patient engaged research.”

It’s a learning process for everyone and the next few years will likely see further progress. Cowley is optimistic, and says, “In general, people are much more aware and are at least having conversations around the challenges of patient led research.”

Footnotes

  • I have read and understood BMJ policy on declaration of interests and have no relevant interests to declare.

References