PCORI funds five patient-centered comparative effectiveness research studies

The Patient-Centered Outcomes Research Institute (PCORI) Board of Governors today approved awards totaling more than $64 million to fund five large patient-centered comparative effectiveness research (CER) studies that will answer critical clinical questions about care for cancer, back pain, and stroke.

The awards are the first to be made through PCORI's Pragmatic Clinical Studies Initiative, an effort to produce results that are broadly applicable to a greater variety of patients and care situations and can be more quickly taken up in routine clinical practice.

Ranging from $7.75 million to $14.5 million each, the five awards will fund studies involving thousands to tens of thousands of patients in efforts to fill critical evidence gaps. They will compare:

• primary care versus prompt referral to physical therapy combined with cognitive behavioral therapy to see which more effectively prevents acute low back pain from progressing to chronic pain.
• whether breast cancer screening tailored to women's individual risks and preferences detects cancer and minimizes screening-associated harms as or more effectively than annual screening.
• the ability of more intensive versus less intensive surveillance via CT scanning to minimize side effects and accurately detect lung cancer in patients with small, potentially cancerous growths discovered by screening scans of their lungs.
• whether a comprehensive package of transitional care and in-home support services is more effective than usual care at improving stroke survivors' functional abilities and preventing hospital readmissions.
• whether use of a standing-order entry system advising physicians when to prescribe colony stimulating factors, a medication to prevent infections, reduces over- and underuse of this medication and reduces rates of potentially serious infections among patients with breast, lung, and colorectal cancer.

Each of the studies will engage key patient and stakeholder groups, such as national advocacy organizations, major professional societies and associations, and payers, in their research design and implementation.

"We're truly excited about the topics and the quality of this first set of studies we're funding through our pragmatic clinical studies initiative," said PCORI Executive Director Joe Selby, MD, MPH. "We believe these studies can answer major gaps in our knowledge and have the potential to change practice and improve patients' outcomes. A critical feature of these studies is that they will involve all major stakeholder groups as partners, increasing the chances that the studies will be implemented in a useful way and analyzed with an eye toward implementation, and, if warranted, their results will be put to use in practice much more quickly than most research."

Pragmatic clinical studies differ in key ways from traditional clinical trials, which test whether a care approach works under optimized conditions with carefully selected patients in research centers. However, health care is rarely delivered in such idealized situations and settings. Pragmatic clinical studies test a care option's effectiveness in "real-life" practice situations, such as typical hospital and outpatient care settings, and they involve more diverse patient populations. As a result, these studies' findings are more likely to be applicable to a wide variety of patients.

The five studies were selected from proposals submitted to PCORI's first funding announcement issued through its pragmatic clinical studies initiative last February. PCORI has issued three funding announcements through this initiative to date, seeking proposals that focus on a series of key CER topics identified by PCORI's multi-stakeholder advisory panels, the Institute of Medicine, and the Agency for Healthcare Research and Quality.