Patient and Family Engagement in Research in Era 3

Abstract

In a March 3, 2016, JAMA viewpoint, Donald M. Berwick, MD, MPP [1Berwick D.M. Era 3 for medicine and health care.JAMA. 2016; 315: 1329-1330Crossref PubMed Scopus (199) Google Scholar], argued for ushering in “Era 3” in medicine and health care by embracing a new belief system requiring nine fundamental changes. Among the changes proposed was learning from patients “what matters most.” We propose that asking patients what matters most, as Berwick suggested, must extend beyond the examination room to the halls of academic medicine, where research questions and priorities are decided. These upstream decisions ultimately shape new knowledge, practice, and the very options available to patients downstream. We therefore propose a 10th change essential to realizing the promise of Era 3: patient and family engagement in research. In the emerging paradigm of true partnership necessary for Era 3, efforts to promote patient and family engagement in research are under way, supported by major industry thought leaders, including the National Academy of Medicine and congressionally authorized agencies such as the Patient-Centered Outcomes Research Institute. Although assessment of implementation and effectiveness is ongoing [2Forsythe L.P. Ellis L.E. Edmundson L. et al.Patient and stakeholder engagement in the PCORI pilot projects: description and lessons learned.J Gen Intern Med. 2016; 31: 13-21Crossref PubMed Scopus (182) Google Scholar, 3Forsythe L.P. Szydlowski V. Murad M.H. et al.A systematic review of approaches for engaging patients for research on rare diseases.J Gen Intern Med. 2014; 29: 788-800Crossref PubMed Scopus (89) Google Scholar, 4Guise J. O’Haire C. McPheeters M. et al.A practice-based tool for engaging stakeholders in future research: a synthesis of current practices.J Clin Epidemiol. 2013; 66: 666-674Abstract Full Text Full Text PDF PubMed Scopus (66) Google Scholar, 5Motu’apuaka M. Whitlock E. Kato E. et al.Defining the benefits and challenges of stakeholder engagement in systematic reviews.Compar Effect Res. 2015; 2015: 13-19Crossref Google Scholar, 6Concannon T.W. Meissner P. Grunbaum J.A. et al.A new taxonomy for stakeholder engagement in patient-centered outcomes research.J Gen Intern Med. 2012; 27: 985-991Crossref PubMed Scopus (237) Google Scholar], a number of barriers are clearly evident. Access to medical research is limited by technical complexity of the methodology, jargon-filled writing, and knowledge about and availability of research journals. Technical complexity is, at least in part, the result of a stated bias toward quantitative designs, as was made eminently clear in an open letter to the BMJ by more than 70 medical researchers challenging this stance [7Greenhalgh T. Annandale E. Ashcroft R. et al.An open letter to the BMJ editors on qualitative research.BMJ. 2016; 352: i563Crossref PubMed Scopus (215) Google Scholar]. For patients, jargon-filled text and complex statistical analyses are a significant barrier. Medical jargon is found not only in abstracts boasting P values and confidence levels but in the titles of the journals and articles themselves, which are not organized with consumer-friendly language and naming conventions. For example, many consumers, particularly those already at risk for health disparities, conceptualize and search for health information in plain language. Instead of “cardiology,” they search for “heart.” Instead of “pulmonary,” they look for “lung.” In an effort to address this challenge, the Patient-Centered Outcomes Research Institute requires that all funded research studies provide 500-word plain-language descriptions. Without more widespread standards for consumer-friendly language, the pool of research that is comprehensible to the average consumer remains limited. At the same time, current access to most peer-reviewed top-tier research journals remains geared primarily toward professionals, supported by subscriptions of academic institutions and organizations. Databases such as PubMed and MEDLINE provide free access to citations and abstracts with research of interest to patients and families. However, gaining access to full articles in research journals requires that patients be Internet savvy and know where to look. Armed with this knowledge, patients can access these journals in a variety of ways, including paid subscriptions for full access, single purchase of an article outright, or accessing contents for a limited time period for a fee. For some journals, access to the full text of published works is available, free of charge, only for a limited number of articles and for research older than 3 years. Others provide free access to persons in developing countries. Some publishers offer open access and free availability of research articles to nonsubscribers of journal publications for a fee, generally paid for by the authors of the article or their institutions. Improving physical access to research is just a first step. In our engagement with patients, in settings across the health care continuum, patients conceptualize access to research as more than simply expanding access to scientific journals. Patients repeatedly describe a sense of being on the periphery of the evidence base. Not only do they request full access to research articles, written to promote consumer understanding of the findings, they also ask for direction in how to apply findings in the context of their health care decisions. As one patient recently questioned during a focus group, “After all, who is the evidence base being created for?” For researchers, engagement of patients and families in research is messy. Increasing participation may complicate the process of gaining institutional review board approval and obtaining informed consent. Scheduling time for participatory input becomes more difficult. With limited time and tools to engage consumers in the process, researchers may evade participatory dialogue and make decisions on the basis of their professional prerogative. Engaging consumers can also challenge our notions of ownership and control, as well as the methodological rigor and quality of research. For example, question development is one of the most common ways of engaging patients in the research process [2Forsythe L.P. Ellis L.E. Edmundson L. et al.Patient and stakeholder engagement in the PCORI pilot projects: description and lessons learned.J Gen Intern Med. 2016; 31: 13-21Crossref PubMed Scopus (182) Google Scholar]. What happens, for example, when engaged stakeholders write double-barreled survey questions? How do researchers negotiate ownership over the process and avoid overediting contributions? It seems that certain types of research studies lend themselves to patient-family engagement more than others. This in turn limits the methodologic options available to researchers. Patient engagement in research has largely been defined as a process beginning with topic selection, conducting research, and ending with dissemination. The topic of evaluation has largely been ignored. Yet a shift in traditional conceptualizations of research evaluation is critical in the new era. Current models to assess the strength, quality, and relevance of scientific evidence have similar criteria based on a hierarchy of study designs, with randomized controlled trials and well-designed meta-analyses earning the highest rankings and observational research, case studies, and qualitative research earning the lowest rankings. Unlike guidelines to critically evaluate the rigor and quality of quantitative research, there are currently no established guidelines for critical assessment and evaluation of the growing amount of strengths-based practices, interventions, and documented professional practice around patient engagement, much of which is qualitative. Using current evaluation models, this research is ranked low in the hierarchy because of flaws in study designs that tend to rely on small convenience or snowball samples and qualitative and correlational behavioral-science approaches. Although there are examples in the literature describing how traditional evaluation models are not perfect, they nevertheless remain the standard. In the new era, evaluation tools for patient- and family-engaged research must deviate from these traditional rankings. Furthermore, new research evaluation tools must account for engagement of patients and families, not only in the research process but as evaluators of research. This means that a tool itself would be understandable and accessible and would support patients and family members in finding research, evaluating it, and putting it to use. Embracing Era 3 will require a new belief system supportive of engaging patients and families in the full research process, including topic selection, conducting the research, dissemination, and evaluation. This new belief system will require that organizations and publishers support access to research. Knowledge brokers of research must include patients and family members on boards and as reviewers of submitted research. Additionally, journal editors should establish a requirement for authors to submit abstracts free of jargon. Researchers will need to incorporate the value of patient and stakeholder time in their project budgets and relax their professional ownership over the research process. A new set of principles for evaluating the strength of the evidence that is informed by patient and family engagement and accessible to patients and families as evaluators of research is needed. This new evaluation tool would not discount the value of traditional research but rather acknowledge the inherent value of experience- and engagement-based contributions to the evidence in a more balanced approach.