Patient-Centered Research Puts Focus on Patient’s Questions, Needs

I was not really expecting to “feel the Bern,” but there he was, his SUV sandwiched between several other SUVs, red and blue lights flashing, in his blue shirtsleeves waiving to passersby on a hot summer day last month. As we approached the Dirksen Building, housing the chambers of the important Senate Committee on Health, Education, Labor, and Pensions, staff of APA’s Department of Government Relations acknowledged that security concerns might slow our entrance to the building. We were on our way to a panel discussion hosted by Sens. Bill Cassidy (R-La.) and Chris Murphy (D-Conn.) about PCORI, or the Patient-Centered Oriented Research Institute, a critically important federal agency that sponsors clinical research.

PCORI supports comparative effectiveness research, which is quite different from the efficacy randomized, controlled trials (RCT) we all learned about in residency and medical school. In efficacy RCTs, a medication, device, or psychosocial intervention is tested in a “laboratory setting,” usually a university hospital or clinic, that is staffed so patients are carefully monitored and vigorous outreach to them is possible, making it most likely that participants will stay in the clinical trial. To the contrary, comparative effectiveness RCTs are conducted in “real world” clinics or hospitals, with the crowding, staff limitations, and other attendant problems that exist in these health care settings. As well, most efficacy RCTs are conducted enrolling highly selected populations—for example, patients with mood problems but no alcohol misuse problems. Yet more than 50 percent of those with bipolar disorder and major depressive disorder have a problem with alcohol misuse. In contrast, comparative effectiveness RCTs welcome the inclusion of these more complex cases.

What does it mean for research, or treatment for that matter, to be patient-centered? For much of the history of modern medicine, clinical investigators identified what key questions still required answers and set up experiments to try to answer them. Similarly, since the very beginning of medicine, physicians related to their patients in a benign, paternalistic manner, listening closely to the patient’s history, eliciting relevant information and physical signs and symptoms, and then providing a determination of the most likely diagnosis and the best treatment. However, patient-centered clinical care has been in use for several decades now, the concept being that the patient and physician engage in a dialogue about the patient’s options for care, and the patient’s preferences are heavily weighted.

Patient-centered approaches in research, however, had been lagging, but no more. Now, institutes such as PCORI seek to fund studies in which the patients identify the unanswered questions that are most meaningful to them. In fact, PCORI requires that specifically named patients be involved in the development of research studies, and the patients are paid as consultants during the conduct of the trial. That means that when investigators are planning a study, they must integrate the patient’s point of view. What will make it more likely that patients will want to enroll in the study? What are the deterrents to participation? What are the central questions that need to be answered? Of interest, just last year, the Veterans Health Administration surveyed stakeholders, including patients in their system, to identify which mental health issues should be prioritized and queried whether gender-specific mental health services were important to them. This is an example of a system conducting research to assist in care delivery that is more in line with what patients want, rather than what the physicians think is important, and is thus patient-centered.

As we obtain more data from these patient-centered trials, evidence-based treatments that are more acceptable to the patient will become available. For example, it may be that while some patients really prefer to see their therapist in person, other patients may prefer a telemedicine approach or an online approach. Although these routes of delivery have some limitations (for example, the physician cannot appreciate the full range of the patient’s nonverbal behavior, which may provide important information), if in-person appointments will impede participation in the treatment, these are critical options to have on hand, once verified to be effective. Of course, implementation of patient-centered care is not without its challenges. Empowering patients to be active participants in their care often requires educating them about their disorder and options for therapy to ensure they are prepared to ask relevant questions and understand the answers. Certainly this process is more difficult for patients whose disorders impair their cognitive and decision-making abilities. Training physicians to have this type of communication with patients and to engage their participation in care will be essential. Nonetheless, we welcome the arrival of data relevant to patients as they exist in the real world, who have complex medical and psychiatric histories and are treated in settings that are far from ideal. ■