The Domains of Patient Participation in Depth
PATIENT INVOLVEMENT NEEDS TO HAPPEN AT EVERY STEP
The patient community should be appropriately engaged throughout the research process so that their contributions and perspective can inform every stage of a project, from conception and design to data collection and communications. To be effective, this engagement must be inclusive; that is, a fully representative cohort of the patient population must participate. This can be challenging, since certain groups (namely, those with higher levels of health literacy at the start) tend to be easier to recruit and engage.
In both the US and Europe, it is becoming more common for pharmaceutical companies to consult patients and/or patient advocacy groups (PAGs) early on in the study design process, with the goal of creating protocols that will be more comfortable and tolerable and striving for outcomes that better align with patient needs.
Radican says, “Right from the first discussions about a research study, we should be thinking, ‘Let’s get patients involved – let’s find patients who have this disease or condition and bring them in from the very beginning.’ We want them as partners at the research table; we want and absolutely need their insights, their perspectives, and their learnings. Patients should be included from the study conceptualization to the dissemination of research results, and every step in between.”
“Patient-centered outcomes and patient-reported outcomes are increasingly accepted as standard practice within the pharmaceutical field,” says Karen Bailey, PhD, a London-based scientist who leads qualitative research at OPEN Health Evidence & Access, UK. “Less widely understood, but definitely growing, is patient engagement at the study design stage under the banner of patient-public involvement (PPI). We recently reviewed some of OH’s HEOR projects to identify examples of research that involved patients or PAGs in this way. Patients or patient representatives were invited to advise on the design of the study, particularly whether the proposed patient-reported outcome measures were appropriate and captured all the symptoms and impacts. They were also asked to look at the study material itself, providing feedback on issues like the appropriateness and time burden of interview questions.”
Bailey explains that patient engagement in a study’s early stages can go beyond advising on study design. “Another key aspect of patient engagement is asking patient participants to support recruitment efforts,” she says. “I believe that this can be entirely valid as long as it is in the context of a wider relationship, where patients can take part in discussions and be involved in the study’s design — though we should try to avoid using PAGs as nothing more than recruitment vendors. We find that if patient groups are involved in the process from the early stages, they are more likely to provide additional support on the recruitment side.”
Jasmine Malone, BSc, emphasizes that many patients have valid reasons for hesitating to participate, and an important aspect of inclusiveness is breaking down these barriers in ways that display sensitivity and understanding.
“Patients can be resistant to participating in research for a number of reasons, ranging from practical concerns to ethical concerns or their own personal views,” she says. “Commonly, practical reasons far outweigh ideological ones. These are situations where patients are already physically or emotionally burdened by the treatment or their experience of the disease itself, and they do not necessarily want to relive that for the purposes of evidencegathering. It can be hard to share — you have to be at the right part in your journey to want to talk about how a disease has affected you or your child or your loved ones — and it is often very much about finding the right patient at the right time in their journey, when they feel ready to share their experience and become an advocate for their condition and the associated unmet needs.”
Tailoring communications about a project to the health literacy level of prospective participants can increase their willingness to take part, Malone explains. “If a patient’s comprehension of their condition is low — meaning the level of information they are getting is not well-suited to their level of health literacy — they may be less confident about participating, especially in a group setting that may include patients or HCPs that they think of as more advanced or better informed,” she says. “We tailor our patient participation programs to all levels of health literacy and frame the conversations to be as inclusive and open as possible. There is no wrong statement. There is no wrong answer. We just want to hear in your own words what your answers to specific questions are — and if you don’t know the answer, that’s fine as well."
Oliver Childs, BSc, global scientific director at OPEN Health Communications, agrees that the right communications strategies – and the right recruitment strategies – are essential for inclusiveness. “Patient participation is not necessarily linked to patients’ willingness to be involved so much as their opportunity to be involved,” he explains. “The patients who work with pharma and engage with patient associations are, by definition, going to be those with slightly more health-literate backgrounds; often, they have actively identified that there is a pharma company out there that they could engage with, to offer help and potentially influence their work. These ‘professional patients’ add huge value with their personal perspectives, but less health literate patients tend to be under-represented.”
“People with the lowest health literacy tend to be the most disadvantaged,” he continues. “These are the people who are less likely to be digitally literate. They’re less likely to have access to one or more devices at home that they can use to get information about disease. Their experience will be very different than that of the patients who are easier to access. The seeming ubiquity of digital masks the fact that many of the most vulnerable and least healthy are those who are also digitally isolated. This digital divide must be addressed. Otherwise, we’re in danger of cementing inequalities through digital health rather than shrinking them. True inclusiveness goes beyond simply building usable ‘things’ — it’s just as much about work to widen digital participation.”
"Who are we to sit and design a study about a disease and not include a single patient with the disease in that design? How can we know that our studies will include all aspects of the disease and the intervention that are relevant and important to patients?"
LARRY RADICAN, PHD VICE PRESIDENT, HEOR, OPEN HEALTH