GUEST BLOG: Why medical research should include the patient perspective
Dr. Leana Wen discusses how PCORI–Patient-Centered Outcomes Research Institute–hopes to change how medical research is approached in the US.
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 four 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.
Dr. Leana S. Wen, M.D., is an emergency physician at Brigham & Women’s Hospital and Massachusetts General Hospital and a clinical fellow at Harvard Medical School. She is the author of the new book, When Doctors Don’t Listen: How to Avoid Misdiagnoses and Unnecessary Tests. For more information, visit her blog The Doctor is Listening or her website. On Twitter, follow @DrLeanaWen.
Editor’s Note: Lorraine Johnson, CEO of LymeDisease.org, also serves on PCORI’s Advisory Panel on Patient Engagement.
- August 17, 2013 at 9:16 pm
This is a catch concept, but the real catch will be in figuring out how to meaningfully add patient input while keeping the nuts and bolts of the scientific method intact. I supect the biggest benefit from getting patient input will be in prioritizing what problems need the most attention in the first place. It wouldn’t hurt to get more input from primary care physicians too.
- November 3, 2013 at 5:48 pm
Dr. Wen is exactly right. I can even go one step further: As a sufferer, give the research money to me and I will design the most result rich research program to date. I have had PTSD since age 12 (38 years), but although the trauma was extremely severe (unknowing being given LSD as a joke), my condition started with the LSD episode and I could I identify that as the cause of all the symptoms (at age 13), licensed therapists at Wayne State U and Lafayette Clinic in Detroit misdiagnosed me with Separation Anxiety and refused to take serious my insistence that the LSD caused conditioned fear response reminiscent of WWI Shell Shock. At age 15 (1978) I specifically identified my condition as “a shell shock like condition” (before the coining of PTSD) with me showing eight of the most serious of the criteria and pointing to them in the DSM on the doctor’s desk. Again, the psychiatrist (now a diplomat of the American Board of Psychiatry and Neurology) diagnosed me with anxiety neurosis, again ignoring the massive trauma altogether and not treating it. Finally, at about age 24, I was correctly diagnosed, but told I was unlikely to respond to treatment because it was not treated in a timely fashion. If my diagnoses where accurate at are 13 and 15 and the psychologists were so horribly wrong what good are these “experts”. A grant given to me based of my proven superior knowledge and experience with PTSD would be more productive than awarding it to any other researcher I have followed.
Patient participation is exceedingly important in psychiatric research for a simple reason: When a surgeon, for example, that uses questionable methods, a body count reveals the questionable techniques and the doctors license is revoked. In psychiatry and psychology, doctors often lie about the efficacy of their treatments and successes because no one is verifying claimed results with patients. Unlike the the example with the surgeon above there are normally no dead bodies to illuminate the treatment failures (and outright frauds) and in cases where there are suicides blame is easily directed away from any therapists treatment or conduct. Surviving patients can leave treatment and continue to suffer for the rest of their lives and there is nothing to stop the therapists for publishing a paper claiming success, as psychologist John Money famously did in the botched sexual reassignment of David Reimer.
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