When Effectiveness Wins, Everyone Wins: The Mission and Impact of PCORI: Interview with Dr. Joe V. Selby and Dr. Julie Panepinto

PCORI makes an effort to include studies of rare conditions that may be overlooked and underfunded, studies that address improving care and outcomes for patients with multiple chronic conditions, and studies centering on the burden of specific illnesses, as measured by their frequency, their severity, the availability or lack of other treatments, and their costs to society.

Dr. Joe V. Selby has been PCORI’s Executive Director from its start. “We fund studies that will give patients and those who care for them the information needed to choose the health-care options that may work best for them given their personal situations and preferences,” he says. “We share these results in ways that patients, clinicians, and others will find useful.” The studies PCORI funds are best described as comparative clinical effectiveness research (CER) — studies comparing how well different approaches to care work (Table 1). “We are a ‘stakeholder-driven’ research funder,” Dr. Selby asserts, explaining that the institute’s research questions don’t come only from the scientific community or from the manufacturers. Instead they emerge from patients, clinicians, delivery systems, and insurers.

Another one of the hallmarks of PCORI research is that rather than placing focus on the “average” patient or the average difference between two treatments, PCORI research emphasizes how treatment effectiveness varies among particular patient subgroups. Another point of importance is the inclusion of outcomes that other studies might not address. “In comparing drugs to treat multiple sclerosis for example,” Dr. Selby proposes, “we don’t focus just on how two drugs impact changes on an MRI or CT image, but on how the treatments compare for reducing pain, stiffness, balance, fatigue, depression, or cognitive impairment.”

Dr. Julie Panepinto with Medical College of Wisconsin has a unique perspective on PCORI study selection, not only as a hematologist, but as a reviewer of grant applications to the institute. “PCORI is interested in comparative effectiveness studies, not efficacy studies,” she says. “I like to think of efficacy trials as studies that attempt to answer the question ‘can it work?’ as with a typical phase III–type study, whereas effectiveness studies attempt to answer the question ‘does it work?’” It is the “does it work” question that gets to the core of real-world settings, where studies are designed to test two equally efficacious interventions against each other to determine which treatment is better, and especially, which is better at improving patient-centered outcomes such as physical functioning.

Drs. Selby and Panepinto stress that it is these varieties of outcomes that offer patients a clearer idea of what works better for people like themselves. This emphasis on the patient perspective is key: PCORI‐funded studies involve patients in the development of the research projects, governance and oversight, and dissemination strategy.

Patients are not the only valued resource in PCORI’s bold efforts to improve health care. Caregivers, patient organizations, clinical specialty organizations, payers, and employers are also demonstrating increasing engagement. They are becoming more involved as powerful “non-researcher” partners, who rely on more substantive evidence for their day-to-day activities. These stakeholder groups help ensure that PCORI identifies the right questions, studies them in appropriate ways, interprets the data in light of all the available information, and disseminates and implements the findings widely when studies suggest that practice should change. “We’re seeing that when patients and other stakeholders partner with scientists in choosing research questions, as well as in designing and conducting studies to answer those questions, they make it more likely that the results will be relevant to them and incorporated into daily practice,” says Dr. Selby.

To make sure that studies are well informed by these diverse stakeholders, PCORI hosts workshops convening participants from every sector. Dr. Panepinto represented ASH during a recent workshop on sickle cell disease (SCD). “There were SCD physicians, nurses, patients, representatives from pharma, the head of the National Institute for Children’s Health Quality, and representatives from the American Academy of Pediatrics and the Sickle Cell Disease Association present.” Among the goals of such events is to collaboratively shape high-level research questions that would lend themselves to specific proposals and more specific research questions.

PCORI has funded several research areas that focus on hematologic diseases, especially SCD, of which a few examples are summarized in Table 2. But what are the characteristics of a successful application? Dr. Panepinto points to three common themes: 1) evidence of the efficacy of different interventions (e.g., a therapeutic solution or procedure) that were then compared head-to-head in order to test effectiveness; 2) involvement of patients in a capacity that fits well with the overall mission of PCORI; and 3) measurement of outcomes that matter to patients, such as patient-reported functioning.

Looking ahead, the time appears to be ripe for hematologists to engage with programs such as PCORI. “I think any field that has several different acceptable methods to treat patients lends itself well to PCORI research,” noted Dr. Panepinto. For more information or to get involved, visit www.pcori.org.