Late last August, autism researcher and clinical psychologist Simon Baron-Cohen announced the start of an ambitious new study called Spectrum 10K. In media interviews, he described his vision to collect saliva samples, medical information, and behavioral surveys from 10,000 autistic people and their families to better understand the condition’s genetic and environmental underpinnings. To recruit such a large cohort—the biggest in the UK’s history, according to Spectrum 10K’s website—the project required “an unusually well-publicized launch,” Baron-Cohen tells The Scientist in an email. Over the next few weeks, a team of ambassadors, many of whom had either been diagnosed themselves or were caregivers to autistic children, touted the study’s mission. Among them were television celebrities and a former president of the Royal Society, who helped bring the project into the public eye.
These appearances painted a picture of a new era of autism research, but that vision wasn’t embraced by all. Kieran Rose, an autism advocate who had himself been diagnosed by Baron-Cohen years before, wrote about the “shock and initial trauma” felt by many in the community following the announcement. “Over the course of that day, reading through all that information, you realize it was all really woolly—there [were] no real aims or outcomes for it, there was no real identifiable benefit to anybody,” Rose tells The Scientist.
Rose, along with several autism scholars, advocates, and support groups, formed Boycott Spectrum 10K, while a separate petition calling for the project to be halted collected more than 5,000 signatures. The boycotters’ concerns, outlined in an open letter posted online last September, include researchers’ failure to consult with patients and alleged obfuscation of the study’s objectives and data privacy protocols. Some people oppose the study because they fear that genetic information could be used to “cure” autism or to develop prenatal screenings. The people behind Spectrum 10K, its detractors note, haven’t been clear about how the project will avoid this misappropriation of genetic information, nor how the research will improve the well-being of participants. These issues, Rose says, could have been avoided had the Spectrum 10K team consulted with patients from the beginning. (On its site, Spectrum 10K mentions an advisory panel that includes autistic adults and parents of autistic children, and Baron-Cohen says that this panel was consulted in the years prior to the study’s launch.)
These concerns spread fast. Autism groups that had initially supported the project rescinded their cooperation, and protests sprang up outside of participating facilities. Only weeks later, Spectrum 10K’s organizers released an apology to the community and announced that they were voluntarily pausing further recruitment as they worked to address the criticisms. The UK’s Health Research Authority (HRA) has since stepped in to investigate concerns leveled at the initiative, and has so far requested undisclosed changes to the project’s supporting documentation. In an update last November, an HRA spokesperson stated that “some of the issues raised as part of the complaints process could have been considered during the original review” of the study.
If the story of Spectrum 10K is a cautionary tale, it is also a lens. Increasingly, research is done with public and patient engagement, or PPE (sometimes called public and patient involvement), as funding agencies, research organizations, and academic institutions adopt the practice. The benefits, as explored in several studies, include injecting fresh perspectives into research, tapping new sources for recruitment and dissemination of findings, and giving patients a say when it comes to what scientists study and how. Science produced in this way is a collaboration between researchers and the public, one in which researchers are encouraged to compensate engaged members of the public and patient communities for their knowledge and to treat them as equal members of the team.
“Science is so important,” Rose says, “But what’s more important is doing this the right way.”
Sharing data is so important for the work that we do . . . but I want to do it in the best way possible, where families feel like their information is being respected.—Yana Wilson, Cerebral Palsy Alliance
From the ground up
One of the immediate benefits of PPE is that it often stimulates new ideas and energizes scientists by humanizing their work, says Eric Lau, a tumor biologist at Moffitt Cancer Center in Florida. His research on tumor biology, particularly in melanoma, rarely “emotionally engages” him, he says, but a few years ago Lau’s husband, a clinician, asked if he could bring a patient for a tour of the lab. Lau expected to leave the young woman, who had an aggressive form of brain cancer, feeling more hopeful. Instead, “she flipped the whole situation around,” he says, by opening up about her own experiences with not just the physical aspects of cancer, but the emotional ones too. “By the time she was done, my whole lab was in shambles. But for the next couple of months, I have never seen them move faster.”
Lau, who hadn’t previously engaged much with patients, took away a desire to bring basic scientists and patients together as collaborators. “We realized very quickly [that] if one experience had this profound, lasting effect, we should be making this experience available to as many labs as possible.” Partnering with PPE specialists at Moffitt, Lau created a series of ongoing forums where patients share their stories—guided by patient advisors to help focus their message—and scientists brainstorm how best to fold those experiences into their work. Initially held in person, these meetings went virtual after the start of the pandemic.
As a result of these interactions, cancer researchers at Moffitt are thinking about their research in new ways, Lau says. For example, basic scientists often develop tools to kill tumor cells without considering the possible side effects of treatment, but after one patient noted the “brain fog” that accompanied her lung cancer treatment, researchers at the center began shifting from broadly toxic treatments such as chemotherapy to targeted therapies that spare brain cells.
The direction of Lau’s research has also been inspired by patients he met at these events. He recently started probing how testosterone drives melanoma after attending a forum highlighting men with breast cancer. For some reason, breast cancer survivors of any gender are at an increased risk of developing melanoma, and vice versa, compared to the general population, he says. “I would say there’s maybe four or five groups in the whole US that are studying this topic, but it’s actually a . . . profound biological effect.”
In addition to spurring new ideas, PPE can also sharpen study planning, says Yana Wilson, a genomics research officer at the Cerebral Palsy (CP) Alliance Research Institute in Australia. “Asking families what they’re interested in is really helping drive our strategy,” she says. Wilson and her colleagues recently conducted a survey of CP patients and their families on their attitudes toward genomics research. The findings suggest that many patients support the work and are understanding of prickly aspects like data sharing, but some think that scientists often stumble when crafting public documents such as study information forms and privacy policies.
Privacy in particular is a recurring issue. It can be daunting, Wilson admits, to educate people about all the ways their data could be used, and the forms that end up being sent to patients typically contain pared-down descriptions. Instead, Wilson and her nonscientist partners have developed a detailed policy that lays out explicit conditions under which information can be shared—patients might choose to release their data within a particular institution or for certain purposes, for example—and Wilson encourages researchers to speak with their study participants and reassure them that they have a say in how their information will be used. “Sharing data is so important for the work that we do, and I’m always going to support that, but I want to do it in the best way possible, where families feel like their information is being respected,” Wilson says.
That level of respect also extends to other parts of her protocols. For her dissertation, Wilson studies families in which multiple children develop conditions such as CP and autism. Working with patients means that Wilson has ironed out small procedural details before they become significant wrinkles. Asking a beleaguered parent to bring two young children to a hospital for sample collection or testing might dissuade them, so she strategizes other ways of engaging, including home visits and phone calls. Other researchers noted similar adjustments, such as mailing paperwork rather than emailing it, as some patients, particularly from older generations, are not computer literate or don’t have access to the necessary technology. “For us, that’s really changed some of the things that we implement in our work,” Wilson notes.
While many members of the public get involved in PPE to share their experiences, they often lack scientific backgrounds—but in fact that is part of what makes for fruitful research partnerships. Researchers who have incorporated PPE into their work tell The Scientist that they are often surprised at how helpful patients are in various aspects of the research process.
Kate Boddy, a research fellow at the University of Exeter whose job involves helping scientists bring PPE into their studies, understands this better than most. Often, she says, the best fits are those where participants can comment on “tangible things” such as consent documents. But she’s been looking for other ways to solicit their involvement and has incorporated PPE into projects that push the normal bounds of patient and public involvement.
Researchers who have incorporated PPE into their work tell The Scientist that they are often surprised at how helpful patients are in various aspects of the research process.
Boddy partnered with general practitioner Christopher Clark in 2014 for a meta-analysis looking into whether differences in blood pressure between a person’s arms might indicate a risk for cardiovascular disease. Because the project, called the INTERPRESS Collaboration, relied on existing studies, it didn’t require recruitment, making it an unusual choice for PPE. Yet Boddy included three members of the public as advisors. None of them had conditions relevant to the project, but they had attended an early presentation about the study that solicited public feedback. In addition to drafting a plain-language summary for the public and invitation letters to the authors of studies whose data Clark wished to include, Boddy says, the trio also evaluated the appraisal tool, called QUIPs, used to assess studies for inclusion in INTERPRESS.
Thanks the public advisors’ involvement, the research team quickly realized that some aspects of QUIPs left room for improvement—namely, that there were redundancies between the published tool and the ways that the INTERPRESS team would analyze the studies. The conversations between the scientists and the PPE group led to significant changes in how the appraisal tool was applied, and therefore how the papers were ultimately analyzed for inclusion in the meta-analysis. Although some researchers view public involvement as only minimally helpful for meta-analyses, “now that INTERPRESS is completely wrapped up, I can say that [it] has been really significant in a way that perhaps goes against that perception,” says Boddy.
Malcolm Turner, one of the three public advisors on the INTERPRESS project, says that participating was a unique experience. “There were significant areas where we did get involved, and that’s not always the case,” says Turner, who has been in several other PPE groups for various research efforts. He adds that “you get some projects where they seem to have asked people along to tick the box.” What set INTERPRESS apart, he says, is the genuine interest that the researchers showed in his opinion. “You never felt ill at ease or that what you wanted to say wasn’t going to be listened to and received.”
Working comprehensively with patients was on Kristina Medlinskiene’s mind too. As a PhD student in pharmacy research at the University of Bradford in the UK, Medlinskiene knew that she wanted to incorporate PPE into her research on factors influencing the clinical use of medications, focusing specifically on stroke-preventing anticoagulant drugs, but felt there weren’t many resources for early career scientists. Her adviser couldn’t offer guidance, and her grant couldn’t support a patient advisory group. So she applied for more funding and reached out to local patient organizations. It turned out to be a sometimes-frustrating process, and, together with three other students, Medlinskiene published a guide detailing their experiences for other early career scientists.
Medlinskiene’s persistence ultimately paid off. After an internet search led her to a support group for patients with atrial fibrillation, Medlinskiene formed a three-person advisory committee. While the members contributed to many facets of her work, Medlinskiene specifically wanted their insight as she crafted her analytical framework using a social science tool called thematic data analysis. The team created a visual map of recurring themes pulled from her interview transcripts with patients, which Medlinskiene used to develop her analysis framework. The entire exercise “added a richness to the analysis, having visited different views and perspectives, because they really saw this from their own experience of being a patient accessing a service,” she says.
Medlinskiene, now an associate professor at Bradford, adds that the experience brought another, unexpected benefit: a source of support during the early part of her career, a time that could otherwise feel very isolating. “Having your group believe in your project—because it’s close to their heart, it’s important to them, it does affect their daily lives—it motivates you to carry on even if you feel you’re struggling.”
Seeing it through
Several researchers who spoke to The Scientist note that research of any kind is most useful when it gets into the hands of those who can put it into practice. In addition to helping formulate studies, PPE is often most valuable when it comes time to disseminate results.
You never felt ill at ease or that what you wanted to say wasn’t going to be listened to and received.—Malcolm Turner, public advisor to the INTERPRESS Collaboration
Boddy treats her participants just as she would any collaborator. Following the conclusion of INTERPRESS, she worked with the advisory group to present their findings at a conference, and they continue to be involved as coauthors on manuscripts. Cerebral palsy researcher Wilson has also presented talks with her participants, and says that she sometimes sees talks given solely by patients or members of the public who played a role in the research. This is an opportunity for engagement, as are commentaries in journals coauthored by scientists and their public partners. All these forms of ongoing collaboration and engagement support PPE as a component of empirical research.
Members of the public can also be unexpected sources for sharing results beyond academic settings. Because many come to studies through support or advocacy groups, they’re often deeply embedded in their communities, says Rose, and can use their social networks to amplify news of a study’s findings. Rose’s social media presence as The Autistic Advocate, for example, gives him an audience of tens of thousands, including both scientists and members of the public. Having coproduced research into autism for many years, Rose says that he’s been able to educate others about the complex ethical issues surrounding the work, issues that Spectrum 10K has brought to the foreground.
Awaiting the results of the investigation into Spectrum 10K, Baron-Cohen tells The Scientist in an email that he has taken much of the feedback to heart. The team now recognizes that “we need to consult with a much larger group, and to ensure that this group is representative of the many perspectives within the autism community.” The researchers brought in an organization that facilitates stakeholder engagement to oversee their conversations with autistic people as they work together to restructure the study’s goals, according to a statement on the study’s website. The experience of Spectrum 10K has also changed how the University of Cambridge’s Autism Research Centre, which Baron-Cohen directs, will organize its work, he says. “Large-scale or controversial studies require large-scale, ongoing engagement, to be representative of all the opinions within the diverse community. We are now wholeheartedly embracing these new ways of working.”
Rose remains skeptical as to whether Spectrum 10K can reboot without running into many of the same problems, given that the funding has already been awarded to pursue specific objectives, but notes that autism makes a great case study for many issues in academia, including how PPE can be done successfully. After a century of patients being scrutinized by researchers far removed from the experience of the condition they’re studying, “there are some fabulous autism researchers working in the field who are now doing coproduced research,” he says. “We’re moving the field on dramatically, and that’s because all those things were missed before.”
TIPS FOR MEANINGFUL PUBLIC ENGAGEMENT
BELIEVE PATIENTS. One of the benefits of including public and patient engagement (PPE) in research is the added perspective that personal experience can bring. But patients often report having their experiences dismissed by doctors or researchers, according to Stanford University geneticist Ronald Davis, whose son was diagnosed with myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) after visiting nearly a dozen doctors. The most fruitful collaborations, notes Moffitt Cancer Center tumor biologist Eric Lau, arise when scientists take the concerns of patients seriously.
DON'T TOKENIZE. As more funding agencies require PPE, reports have arisen of researchers involving the public only as a box-checking exercise, notes Kate Boddy, a PPE expert at the University of Exeter. She recommends engaging in meaningful conversations and respecting the time and effort of your public advisors. Many established guidelines recommend compensating participants.
BRING THEM IN EARLY, INVOLVE THEM THROUGHOUT. True coproduced research requires treating patients and other members of the public as equal collaborators and actively seeking their input at each step of the research process, says autism advocate Kieran Rose, who coproduces research with academics studying the disorder. Kristina Medlinskiene, an associate professor of pharmacy research at the University of Bradford, says this can mean soliciting input during design, recruitment, analysis, and dissemination of research.
KEEP COMMUNICATION FLOWING. Yana Wilson’s work with patients with cerebral palsy and their families at the Cerebral Palsy Alliance Research Institute in Australia has reinforced how much people appreciate transparent, ongoing communication with researchers. Rather than updating patients periodically, take the time to consult with them regularly, she says, and always be courteous, consistent, and caring in your feedback.
BE FLEXIBLE. Many researchers note that the time commitments required to do PPE right remain a barrier to more widespread adoption among scientists. Budget time to apply for grants to include PPE in a project, and to meet with your partners. Christopher Clark of the University of Exeter’s INTERPRESS Collaboration held special meetings with his public advisory group before each lab gathering to prepare them for what would be discussed, says INTERPRESS public advisor Malcolm Turner. This helped avoid overwhelming nonscientists on the team with new information.
BE PROACTIVE IN SEARCHING FOR RESOURCES. Many universities are hiring staff to work with researchers who want to incorporate PPE. If your institution doesn’t yet have this type of department, consider reaching out to local patient or advocacy groups specializing in the disease or disorder you study, says Medlinskiene. They can be useful in recruiting patients and may also point you toward other helpful resources.
ACKNOWLEDGE PPE INVOLVEMENT IN MANUSCRIPTS AND PRESENTATIONS. Medlinskiene recalls how difficult she first found it to identify studies that included PPE, even when she knew the authors had involved the public in their work. She recommends being explicit about it in papers, presenting posters or talks alongside public collaborators at conferences, or coauthoring commentaries in journals. Boddy notes that this approach lends legitimacy to the work, while bringing PPE to the attention of other academics.
Correction (March 16): A former version of this piece incorrectly referred to the Cerebral Palsy Alliance Research Institute as the Cerebral Palsy Alliance Research Initiative. The Scientist regrets the error.
This article was featured in March 2022, Issue 2 of the digest