The Patient-Centered Outcomes Research Institute, known as PCORI, is an independent, nonprofit research organization that was established by Congress in 2010 to meet an unmet need. Congress was responding to the growing recognition that despite all the advances in scientific knowledge gained through traditional health research, patients and those who care for them needed better information to guide the healthcare decisions they faced every day. Patients need to know, for example, not only whether a particular drug or device “works,” but what they might reasonably expect from different options given their particular characteristics and circumstances, and what trade-offs or indirect burdens they might need to consider as part of the decision.
PCORI’s aim is to provide and promote this kind of information through funding of comparative clinical effectiveness research (CER), which compares two or more medical treatments, services or health practices to help patients and other stakeholders make better informed decisions. Our approach has three main elements, which we call Research Done Differently:
- It focuses on the research topics, questions and outcomes that are most important to patients and those who care for them.
- We work closely with a range of healthcare stakeholders to guide our research funding.
- We require that patients be true partners in the research we fund.
At the heart of this work is engagement—of patients, first and foremost—but also of caregivers, clinicians and health systems, payers and purchasers, policymakers and researchers. For PCORI, engagement means essentially democratizing the research enterprise, by bringing together and integrating in a meaningful way the variety of perspectives needed to make evidence truly useful to patients, caregivers, and the broader healthcare community. Patients, and all PCORI stakeholders, can participate in many ways, including:
- Serving on advisory panels and helping determine research topics.
- Taking part in the merit review process that determines which projects we will fund.
- Participating in peer review of draft final research reports, to provide critical perspective and nuance.
- Playing a role in the dissemination and implementation phase, where research findings are translated and communicated for use in real-world settings, including in clinical practice or policy.
To help unpack these principles and approach, it might help to share some examples of the role of engagement in PCORI-funded research, and to hear some of our stakeholder voices more directly.
Caregivers for those living with multiple sclerosis provide much-needed care to patients, but have a challenging role that can take a toll on their own health and wellbeing over many years. Most MS research focuses on the patient, however, and does not seek to capture the needs of caregivers or take advantage of the special expertise they have about MS. To address this concern, a Massachusetts-based nonprofit group, iConquerMS, is using funds from a PCORI Engagement Award to both expand its activities to address the needs of caregivers and to prepare them to participate in future CER, as well as provide opportunities to do so. You can hear directly from a patient advocate involved in this project in this video.
Another Engagement Award project funded the development of a patient-centered research agenda for women with Parkinson’s disease. Women are diagnosed with Parkinson’s less frequently than men, though tend to fare worse with certain health outcomes related to Parkinson’s. The award funded a series of forums around the country, where women with Parkinson’s, as well as caregivers, clinicians and others, could talk about the issues specific to women, such as support groups being mostly set up for men. The long-term goal is to seed ideas for more CER studies to address the needs and priorities of women with Parkinson’s.
Another good example is an ongoing study comparing three approaches to diabetes care among African Americans with diabetes. This study highlights the role of patient advisory councils and patient partners in helping to design studies that will deliver results that are important to patients. As one patient partner emphasized in this video, “I am not a doctor, but it is my body.” This study illustrates what has become a maxim in PCORI’s engagement work: “You have to meet people where they are.” Results from this study will be presented at the PCORI Annual Meeting at the end of this month (registration is free!).
In these and in all the nearly 2,000 research and research-related projects PCORI has funded to date, our goal is to generate evidence that helps real people make informed decisions about complex, real-world health issues, by engaging people in those studies at every stage of the research, through dissemination and uptake of the results.
Greg Martin is Acting Chief Engagement and Dissemination Officer at the Patient-Centered Outcomes Research Institute (PCORI). He previously worked at the National Conference of State Legislatures (NCSL) and American Academy of Family Physicians (AAFP), where he was responsible for leading AAFP’s governmental advocacy assistance to its state and territorial chapters, including research on issues such as health reform implementation, Medicaid and the patient-centered medical home.