What Patients Teach Us About Patient Engagement in Research

“Nihil de nobis, sine nobis,” or “nothing about us, without us,” is a phrase that dates back to the early 1500s when it was used to convey the idea that policies should not be established without full and direct participation of members of groups that would be affected by the policies. In the 1990s, the phrase was used in advocacy efforts for people with disabilities (¹), and more recently, it has been used by patients to communicate the importance of their active engagement in all stages of clinical research (²). Although the value of incorporating into research the perspectives, preferences, and expertise of people living with the condition being studied seems obvious, this has only recently become a priority. In the United States, research funded by the Patient-Centered Outcomes Research Institute (PCORI), established in 2010, requires patient engagement, which PCORI defines as “meaningful involvement… throughout the entire research process—from planning the study, to conducting the study, and disseminating study results” (³). The National Institutes of Health is increasingly involving patients in the planning and implementation of research studies, as is the pharmaceutical industry (⁴). However, despite this growing interest, patient engagement is not yet a routine component of clinical investigation.

In this issue of CJASN, Gutman and colleagues report their findings from semistructured interviews with both adult patients with CKD and caregivers, each of whom had experience with research engagement (⁵). The previous research engagement by the 23 participants (18 patients and five caregivers recruited from Australia, the United States, the United Kingdom, and Denmark) included serving on advisory groups, steering committees, and expert working groups, and occurred at a variety of stages of research, including planning and conducting research projects, evaluating results, and disseminating findings. Interestingly, despite an extensive list of research contributions, none of the participants were categorized as an investigator in prior research, a role for patients that has been increasingly encouraged and facilitated by advocacy groups and some funders (⁶,⁷). The authors used rigorous qualitative methods to identify themes and subthemes from the interviews with the goal of informing strategies to strengthen patient engagement in research. This effort complements a previous report by these authors from facilitated workshop breakout group discussions that included both patients and researchers (⁸). Fully consistent with the premise of patient engagement, the author groups for both studies included at least one patient with CKD.

From the patient interviews, the authors identified six themes that, for most patients, were framed as issues that require attention to improve patient engagement in research: (1) grappling with the diagnosis of CKD, (2) bearing the responsibility for involvement, (3) battling big agendas, (4) seeing the person behind the patient, (5) sensitivity to complexities of payment, and (6) championing the patient voice. The subthemes, the illustrative quotations, and the recommendations made by the participants are numerous, and several stand out as particularly noteworthy.

First, some participants identified stigma and shame associated with a diagnosis of CKD as a barrier to engaging in research, and expressed their sense that CKD is often considered to be the fault of the patient. The perception of CKD as a “non-sexy” disease, a description used by one participant, has been identified by professional and patient advocacy organizations as a reason for the disproportionately low funding for research in kidney disease relative to its prevalence and associated burden. Efforts to correct these misperceptions and eliminate stigma should be prioritized by the nephrology community for obvious reasons, only one of which is the negative effect on research. The authors’ impression was that CDK-related stigma and shame were concerns of participants from the United States, in particular. An additional barrier to research engagement articulated by participants from the United States is a lack of empowerment among patients. The participants attributed this to the low prioritization of patient education by large, financially motivated health systems and dialysis providers, which, in turn, leads to a passive approach by patients to health care, including research.

Second, participants emphasized a need to “change the culture” so involving patients in research is not “simply ticking a box” to meet a requirement, but rather something truly valued by researchers. Based on this editorialist’s personal experience as a researcher, it seems likely that as investigators increasingly partner with patients in designing and conducting research, the challenges will feel less significant, interacting with patients in what initially may be an unfamiliar context will become second nature, and the value of patient engagement in research will be readily evident to researchers. For both researchers and patients, practice with patient engagement in research is helpful, and comfort with this activity can be attained quickly, but is usually not immediate. Participants in the interview study expressed a desire to feel like “equals” and to have researchers appreciate and fully utilize the complementary expertise of patient partners, not only as experts in their disease, but as “more than patients.” Discomfort in “speaking up” despite a desire to contribute was described and attributed by one participant to experience as a patient in the clinical setting “on a lower level… to the doctors.” Ensuring patients engaging in research recognize the importance of their role and the value of their contributions requires active attention and should be viewed as a key responsibility of the research teams.

Third, in addition to contextual issues and patient-researcher dynamics, participants conveyed the importance of practical considerations. They emphasized the need to be explicit about the work required, including the time commitment and scope of activities. Setting expectations before initiating the partnership and throughout the duration of the activity has been identified in other studies as being critical to successful patient engagement (⁹). There is also concern that the same patients tend to get called upon repeatedly to engage in research, which risks over-burdening those who are involved and limiting the diversity of perspectives. Participants encouraged researchers to facilitate involvement of a broad pool of patients by ensuring adequate compensation and covering expenses in advance. Also noted as a potential problem are restrictions on compensating patients for their engagement work, imposed by medical disability policies that limit eligibility on the basis of income, something that is probably not appreciated by many researchers.

Fourth, participants identified some of the benefits of engaging in research. “Paying it forward,” “giving it back to the community,” turning their experiences with disease into “something positive,” meeting others with shared experiences, traveling, and professional development are examples of motivating factors highlighted by participants. Specific products or output from their engagement activities were described by some participants as sources of satisfaction and as evidence for their roles in driving innovation, one of the subthemes identified by the authors.

The report by Gutman and colleagues should be read by researchers planning patient engagement in CKD research and re-read while the engagement activities are underway. For patients, this study may validate their experiences and concerns, and, importantly, it provides strategies to improve the research engagement experience. Although many of the findings likely have relevance to the broad communities of CKD patients and researchers , it is important to recognize some limitations to transferability. As the authors acknowledge, the study participants all had previous experience with research engagement. Although this was appropriate given the specific goals of the study, insights from patients who have not previously engaged in research could help with efforts to expand the pool of patients engaged in research. Additionally, the participants were highly educated (86% had undergraduate or postgraduate degrees), most were working (78% were employed either part-time or full-time), and most (83%) were White. Eliciting perspectives from a more diverse group of patients that better reflects the population of patients with CKD should be a goal of future work to build on this effort.

The benefits of engaging patients in research are established, but experience with this in CKD is still in the early stages of development as a routine practice. Nephrology should continue to recognize that including patients early and often in the research enterprise will lead us all to ask better questions and conduct higher-quality research. Patients and researchers have much to learn from each other.

Disclosures

L.M. Dember reports receiving consultancy fees and honoraria from AstraZeneca, Cara Therapeutics, and Merck; reports receiving compensation from the National Kidney Foundation for her role as Deputy Editor of the American Journal of Kidney Diseases; and reports her spouse has consultancy agreements with Actelion, Alexion, Bristol Myers Squibb, ChemoCentryx, Genentech/Roche, Genzyme/Sanofi, and GlaxoSmithKline and received research funding from Actelion, Bristol Myers Squibb, Celgene, ChemoCentryx, Genentech/Roche, and GlaxoSmithKline.

Funding

This work was supported by the National Institute of Diabetes and Digestive and Kidney Diseases grant U01DK123813.