PCORI helps guide use of new treatments in real-world setting

To help clinicians, patients and other stakeholders make informed health care decisions and improve healthcare delivery and outcomes, comparative research on newly approved therapeutics is needed to understand how these agents work in the real-world setting and how they fit into the treatment armamentarium.

The Patient-Centered Outcomes Research Institute (PCORI), an independent research institute authorized by Congress in 2010, was developed to fill this gap and fund comparative clinical effectiveness research that engages patients and other stakeholders throughout the research process.

“PCORI funds research that compares treatments that are already available in practice on a range of outcomes important to patients, paying particular attention to how treatments work for various patient subgroups, supporting a personalized approach to care and the aims of precision medicine,” said Joe V. Selby, MD, MPH, executive director, PCORI, Washington, D.C., speaking at the Academy of Managed Care Pharmacy (AMCP) meeting in a session entitled The Horizon of Research for Patient Centered Outcomes: The PCORI Perspective.

During the session, Selby described the five national priorities of research for PCORI:

1) Assessment of prevention, diagnosis, and treatment options,

2) improving healthcare systems,

3) communication and dissemination research,

4) addressing disparities, and

5) accelerating patient-centered outcomes research and methodological research.

He emphasized that the institute funds research that is patient-centered and engages the patient, so that patients are seen as partners in research and funded projects are aimed at answering questions or looking at outcomes that matter to patients within the context of patient preference.

In addition, the institute focuses on research of high-priority conditions: Those that affect large numbers of people across a range of population and place a heavy burden on individuals, families, particular populations and society as a whole; and rare diseases that are difficult to study.

He emphasized that research done by PCORI is aimed at addressing implementation of new therapeutics, saying that approval of new agents is just the beginning of comparative effectiveness research of these agents.

“There is much that needs to be learned about benefits and harms, how the agent works in particular patient subgroups, about longer term and rarer side effects, benefits or side effects that matter to patients but are not considered in the approval process, about adherence, about the characteristics of patients who do not benefit from the treatment and who may need another treatment developed,” he said.

Selby urged clinicians, patients and other stakeholders to submit questions and research applications to PCORI for funding, as well as to get involved with PCORI by serving on merit review panels, advisory panels, and in activities to disseminate information.