Traditional Research Leaves Out a Critical Stakeholder: Patients
| June 13, 2013
What’s wrong with the following picture?
Two medical researchers at a major academic center collaborate to study disease X. 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. Based on this first study, the researchers start another cycle of idea generation, data generation, and publication.
I presented this scenario to three Harvard medical students working with me in the ER. They all looked at me blankly. “I don’t get it,” one of them said. “Isn’t this the way research is done?”
That’s exactly the problem. This is “traditional” research, and traditional research leaves out one critical stakeholder: the patient.
A few weeks ago, 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, and is a new federal institute mandated to figure out how to meaningfully involve patients in 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, which by definition is 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 the extent of their decision-making is confined to whether to participate in the trial (let’s set 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 initiate the research and envision the research question itself.
Yet isn’t it the patient—the one with the disease—the most knowledgeable about what needs to be studied and how? No doubt, the researcher has irreplaceable expertise; but isn’t the patient also a vital partner in the process?
In this era of healthcare reform where the patient is finally recognized as a necessary stakeholder, 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 are expected to help figure out what’s important to study, and how to get out the results beyond the realms of scientific meetings and medical journals.
Much about this “new” 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 would be 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?
As part of PCORI’s outreach efforts, it solicited applications for 4 advisory panels. Over 1,000 people applied—many of whom are representatives of patients and patient groups. Twenty-one of us were selected for the advisory panel on patient engagement, and met over two days in Washington D.C.
While many of us are optimistic and excited about the potential of PCORI, we also raised several questions, including:
* How can researchers switch their mentality to become patient-centered such that they are not just simply checking a box to affirm that yes, patients are involved?
* How can PCORI itself ensure that it adheres to its goal of “research done differently” and go really tackle the concept of patient-centeredness?
* While it is admirable that PCORI has taken great strides to involve many patient advocacy groups, these groups represent only a small portion of patients. How can PCORI go beyond hearing the loudest voices—many of whom may also have their own motives—and really engage people?
* How will PCORI address issues neglected in traditional research that are vital to our healthcare, such as preventive medicine?
* How will PCORI really engage people around the country to come up with research questions and redirect priorities?
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 physicians, researcher, and most of all, as patients—to ensure that medical research, and medical care, focuses on and originates from the patient.
