Doing Research Differently: The Unique Value of Engagement in PCORI-Funded Research

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Over the past decade, patient and stakeholder engagement have become increasingly ingrained in health research and the body of literature on such engagement has grown steadily. PCORI has been at the forefront of both of these developments. Our unique focus on involving patients, caregivers and others throughout the research process stems from our mission to provide people with the information they need to make informed choices about their health and health care. We have found that the best way to ensure that comparative clinical effectiveness research (CER) generates truly relevant, patient-centered, useful evidence is to meaningfully involve the people who will use and benefit from it.

In this general way, engagement makes intuitive sense; it echoes the ancient constitutional principle adopted by the disability rights and women’s health movements, among others, of nihil de nobis, sine nobis—"nothing about us, without us." Sometimes the simplest truths are the most profound.

Research Done Differently^®

In the real world, however, engagement is complex, multi-faceted and context-dependent. Doing it effectively requires an intentional and sustained commitment of time, effort, resources and level of expertise. While we certainly recognize that our work on engagement stands on the shoulders of many others, PCORI’s unique contribution has been to build a systematic approach. This approach requires research teams to develop a plan for engaging patients and others, while recognizing that across the wide range of populations and conditions, one size definitely does not fit all and applicants need latitude in how to engage research partners meaningfully. The principles in our engagement rubric—principles like reciprocity, partnership and trust—are broad enough to allow for flexibility, but focused enough to ensure that the research team remains focused on building the relationships and structures that keep all partners engaged and all voices heard.

Other innovations include pioneering the role of engagement officer—a dedicated PCORI staff person who monitors the field of engagement and supports researchers in developing their engagement plans, as well as producing a number of unique resources and tools freely available to all to support engagement in research. Our Engagement Award Program reflects our commitment to building the capacity of researchers and communities to engage in patient-centered CER, to identify the research topics and outcomes that matter most to them and to participate in dissemination of findings.

Evidence for Engagement

A decade of monitoring the “living laboratory” of the practice of engagement in hundreds of PCORI-funded CER studies has taught us a great deal. But what have been the outcomes of this commitment to the art and the science of engagement? While this is still a developing field, some recent peer-reviewed articles commissioned or conducted by PCORI provide encouraging evidence of the power of effective engagement to make results more usable by those they are intended to benefit, including those who may have been historically excluded. A few highlights include:

• An analysis of more than 125 peer-reviewed articles describing engagement activities in PCORI-funded research found that the contributions of patients and stakeholders to such elements as research design, recruitment and retention, data collection and analysis, and dissemination of results contributed to studies considered more relevant and acceptable to patients.
• A study published as part of a special supplement on engagement in the Journal of General Internal Medicine showed that engagement during the research process influenced all aspects of the design and execution of studies. Across nearly 400 identified examples of stakeholder influence, this influence led to changes that reflected the needs and preferences of patients or clinicians, as well as impacts on study feasibility, study quality, engagement scope or quality and study relevance.
• Surveys of more than 250 individuals from advocacy and community-based organizations who engaged as partners in PCORI-funded research projects found that benefiting others was their primary motivation for engaging in research, including the desire to improve patients’ lives and support effective healthcare interventions. Additional motivators included helping to meet the needs of underserved communities, believing in the intervention, wanting a voice in research and a desire to learn.
• A soon-to-be-published study explores PCORI awardee and stakeholder partner perceptions of the relationship between engagement and the dissemination and implementation of study results. Preliminary analysis indicates that stakeholder engagement enhanced the use of study findings by:
  • Focusing studies on questions important to intended audiences.
  • Ensuring findings were disseminated strategically, including through their own networks.
  • Adapting interventions and identifying implementation sites.
  • Adapting messaging about study findings to meet the cultural and linguistic needs of intended audiences.

Further, in clinical practice, engagement is being incorporated directly into quality and safety evaluations, and evidence is emerging that engaging patients and families through mechanisms like hospital patient advisory councils can enhance safety, improve clinical outcomes, reduce health disparities and save billions of dollars.

Looking to the Future

While we are excited by the advances within the field of patient engagement, we recognize there is much more work to be done. For instance, there is currently no consensus on how to define or measure engagement, and there has been little systematic study of which engagement techniques are most effective. Methods for engaging and facilitating the inclusion of historically underrepresented populations as study partners is a further critical gap. To help address those needs, we launched the Science of Engagement funding opportunity to help accelerate building an actionable evidence base that clearly identifies the methods and approaches that lead to effective engagement in CER. We are also working on a revised engagement rubric that better reflects the growing body of research in this space.

In these and other ways, we look forward to working with PCORI-funded research teams, patients and the larger health research community to enrich the evidence needed to move the practice of engagement toward maturity and continue advancing the patient-centered research we call "research done differently^®."

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