By Avni Gupta
Patient-Centered Outcomes Research (PCOR) is characterized by patients participating in various research roles other than merely the subjects. The Patient-Centered Outcomes Research Institute (PCORI), a major funder of PCOR research, defines patient engagement as including patients in all stages of research, “from topic selection through design and conduct of research to dissemination of results.”
However, while the concept of patient engagement in research and its potential for benefitting science are increasingly recognized by funders and investigators, IRBs’ comfort with patients in non-traditional roles in research protocols lags behind.
IRBs’ familiarity with patients in research has traditionally been with patients who are study participants or subjects, and are considered “vulnerable.” Therefore, many IRBs’ review process focuses on ensuring that adequate protective measures are in place to “protect” the patients, including measures such as a detailed written informed consent, continuing review, confidentiality measures, and so on.
This parochial outlook by IRBs, however, fails to differentiate between patients in traditional roles who must be protected, versus patients who are engaged in research as partners, i.e., in “non-traditional roles.” The result is that PCOR investigators and patients who want to be research partners often express frustration with the rigidity of the ethical oversight guidelines IRBs operate under.
Do the patients who volunteer to be research partners need to be subjected to the same level of protective measures as the patients who are study subjects? Perhaps not.
The ethical oversight of PCOR is further complicated by factors such as conflicts of interest of patients and the enhanced use of social media and digital media to gather and store the data.
Researchers from Brigham and Women’s Hospital, the Petrie-Flom Center at Harvard Law School, and Massachusetts General Hospital conducted a study, Patient-Centered Outcomes Research Oversight Study (PCOROS), which was funded by PCORI to develop guidelines and recommendations for adequate ethical oversight of PCOR.
PCOROS involved focus groups, case studies, interviews with stakeholders, and a national survey of IRB chairpersons to understand the numerous ethical challenges of PCOR and how they are addressed.
Frustration with IRBs’ perceived “over-protection” was clearly evident among both investigators and patients. IRBs, similarly, conceded to a lack of unambiguous guidelines to help them with reviewing these novel protocols.
In fact, in the national survey of IRB chairpersons, many acknowledged that while PCOR adds value and meaning to research, there is a need for additional guidelines for its oversight.
To fill these gaps, a modified Delphi process involving 17 content experts and patients was conducted in order to develop and generate consensus on guidelines.
Through a rigorous four-round process, these experts agreed on 21 recommendations to help IRBs review and investigators conduct PCOR, while encouraging patient engagement.
These recommendations were shared in a Symposium hosted by the Petrie-Flom Center in June, and the detailed recommendations and methodologies employed were recently published in the Annals of Internal Medicine.
The experts agreed on a need to develop a formal taxonomy to define the different non-traditional patient roles, based on their tasks in the research study. This was believed to be necessary for the IRB to evaluate the level of risk to the patients.
There was also recognition that patient partners do not necessarily need to be subjected to the informed consent process, or be required to undergo the same research trainings as other academic researchers do. There were concerns about potential conflicts of interest of patients, which might make them less generalizable to the broader patient population, and the group called for guidelines on the identification of patient partners, as well as the disclosure of their conflicts of interest. Guidelines were also considered necessary to deal with situations of simultaneous dual roles of patients in a research study (for example, when a patient is both a subject and a partner).
The patients are the foundation of PCOR. They are driven by a desire to help other patients by informing research that can account for their expectations, preferences, and priorities. A patient advocate, Paul McLean, who nearly lost child to bone marrow failure wrote in a post on Bill of Health, “There are five distinctly different patient leaders on our study’s stakeholder advisory board. I learn from all of them. Maybe what we share is a common sense of advocacy for better patient care.”
We, the researchers, along with IRB professionals, and associated policy making bodies, have an obligation to assist patients who wish to engage in research and direct the research toward more meaningful outcomes by guiding flexibility in the IRBs’ lens.
