To mark PCORI’s third anniversary, we invited representatives of three of our key stakeholder communities – researchers, clinicians, and patients – to submit guest blogs talking about some of the experiences that have led to their interest in patient-centered outcomes research and their hopes for this field. In this blog, Leana Wen, MD, MSc, an emergency physician and member of PCORI's Advisory Panel on Patient Engagement offers her perspective. The views expressed in this blog are those of the author and not necessarily those of PCORI.

What’s wrong with the following picture?

Two researchers at a major academic center collaborate to study a disease. They come up with the research question, design the project, obtain grants, and collect data. Their results are published in a scientific journal and presented at several medical conferences. On the basis of this first study, the researchers start another cycle of idea generation, data generation, and publication. I presented this scenario during lectures to several groups: medical students, research fellows, clinicians, and patients.

They all had the same response to my question. “I don’t get it,” they said. “Isn’t this the way research is done?” That’s exactly the problem. This is traditional research, the type of research that all of us have come to accept as the “way that things are done.” Yet, such research leaves out a critical stakeholder—the patient—and sidelines the important voice of the clinician.

Bringing New Voices to the Research Process

Last April, I had the honor of participating as a member of PCORI’s inaugural advisory panel on patient engagement. PCORI is the Patient-Centered Outcomes Research Institute, an independent, non-profit research institute authorized by Congress to figure out how to meaningfully involve patients in comparative effectiveness research.

Some may argue with the necessity of such an institute. After all, isn’t research ultimately done to help patients? While grants and publications advance careers, few researchers go into such grueling fields to make money or gain fame. Isn’t research already serving the public good—doesn’t that mean it counts as being patient centered?

But here’s another way to think about it. In traditional research, patients are subjects. Their sole purpose is to participate in research trials, and their decision making is confined to whether to participate in the trial (setting aside, for now, the many throughout history who were subject to research against their will). Very rarely, if ever, do patients participate in the research design, data analysis, or result dissemination. Very rarely, if ever, do patients envision the research question and initiate the project.

Yet isn’t the patient—the one with the disease—the most knowledgeable about what needs to be studied and how? And what about the clinician—can she be involved, too, in coming up with research questions and implementing the results? No doubt, the researcher has irreplaceable expertise, but aren’t the patient and clinician also vital partners in the process?

Shifting the Model

In this time of health system transformation, where the patient is finally recognized as a necessary stakeholder and patient-centered care as emerged as a goal, the creation of PCORI is an attempt to shift the paradigm of how medical research is done. Rather than having patients as invisible, nameless subjects, PCORI requires them to be involved in the research design from the very beginning. Patients are expected to be equal partners in deciding what projects to fund and figuring out how to design studies with a patient-centered focus. They—and their clinician/providers—are expected to help figure out what’s important to study and how to get out the results beyond the realms of scientific journals and medical conferences.

Much about this concept of medical research is common sense. Most would agree that precious time and tax dollars should be devoted to what really matters to people. Yet, this paradigm shift is such a departure from traditional research that it is not without its doubters. Researchers, not used to involving patients, question whether they are sophisticated enough to understand the research process. Patients, too, doubt whether they have the expertise required. Fueling this is mutual mistrust: Will this new patient-centered approach derail existing research? Will patients end up being used for some nefarious ulterior motive?

Looking Ahead

Being part of PCORI’s advisory panels has reinforced the importance of patient engagement in research. I’m optimistic and excited about the potential of PCORI and hope that it will take into consideration the following points of guidance:

• PCORI emphasizes that it aims to engage people, not just patients. So far, PCORI has done admirable work to involve many patient advocacy groups. However, these groups represent only a small portion of patients and may not represent the much larger proportion of people who are not traditionally captured in research involving patients. PCORI needs to go beyond hearing the loudest voices—many of whom may also have their own motives—and engage a broad range of people.
• Researchers are smart and savvy and will come up with ways to tailor their proposals to PCORI’s guidelines. PCORI needs to be similarly smart and savvy to ensure that they differentiate researchers who truly have a patient-centered mentality versus those who are just checking a box to affirm that yes, patients are involved.

• Clinicians represent critical stakeholders that are often forgotten in the discussion of patient-centered research. PCORI needs to engage clinicians, not just as the conduit to recruit patients, but in partnership with their patients and with researchers.

• PCORI needs to address issues neglected in traditional research that are vital to our health care, such as preventive medicine, patient-clinician communication, and overuse and misuse of health care. This is necessary, as PCORI is on its way to becoming an organization that aims for “research done differently.”

Recognizing that there is a fundamental problem with the traditional conduct of research is a critical first step to making change. PCORI has opened a door that’s been shut for far too long. It is now up to all of us— as clinicians, as researchers, and most of all, as patients—to ensure that medical research and medical care focus on, and originate from, the patient.

Wen is an attending emergency physician, Director of Patient-Centered Care Research at The George Washington University, Washington, DC, and a member of PCORI’s Advisory Panel on Patient Engagement.

The views expressed in this blog are those of the author and not necessarily those of PCORI.