Does breast cancer screening based on a woman’s individual risk for breast cancer detect cancer as effectively as annual screening for all? Does prompt referral to physical therapy keep back pain from becoming a chronic condition? Is it better for stroke patients to go home from the hospital sooner, with special planning, and recover there?
We look forward to seeing those and other critical research questions answered within the next few years through projects just approved by PCORI’s Board of Governors in the first round of our Pragmatic Clinical Studies Initiative. We’ll fund three more rounds of these exciting studies in the next year and expect to approve up to nine projects, at up to $10 million in direct costs apiece, in each round.
Unlike many past clinical trials, these pragmatic studies are intentionally conducted in routine clinical settings. They are large, so that we can have precise results, and diverse so that we can ask whether treatments work the same or differently in different patient groups. They are designed to provide information that can be directly and quickly used in practice. They have longer terms and larger funding commitments than our previous awards.
Choosing the Strongest, Most Important Studies
When we issued our first pragmatic clinical studies funding announcement last February, we invited proposals for large-scale research projects comparing two or more alternatives for addressing prevention, diagnosis, treatment, or management of a disease or symptom; improving healthcare system–level approaches to managing care; or eliminating health or healthcare disparities. In doing so, we sought the research community’s best ideas for studying critical topics identified by PCORI’s multi-stakeholder Advisory Panels. We invited submissions based on priorities identified by the Institute of Medicine and the Agency for Healthcare Research and Quality. We also invited the research community to make the case for other high-priority research questions.
We’ve taken an important step toward that goal in these first five awards. All of the projects approved for funding are randomized clinical trials, and each proposes to enlist at least 2,000 patients across a variety of clinical settings. The two largest studies each target more than 45,000 participants. Many of the funded studies build on existing infrastructure so that their work can be carried out efficiently.
These studies, like all the research proposals we receive, underwent rigorous review based on our five merit review criteria: impact of the condition on the health of individuals and populations; potential for the study to improve health care and outcomes; technical merit; patient-centeredness; and patient and stakeholder engagement. To ensure use of appropriate methodologies, we performed an additional review of selected applications.
Three of the five approved projects address some aspect of cancer care, one focuses on stroke, and one on back pain. Two address improving healthcare systems, and two align with PCORI priority topics selected by our multi-stakeholder advisory panels. We will apply the active portfolio management processes we have developed to monitor all our project contracts.
We require that each research team partner with relevant patient, clinician, and other stakeholder organizations that strongly endorse the proposed study. Throughout the conduct of the study, these organizations and individual stakeholder partners will guide the teams to make research questions and outcomes relevant to clinical practice and findings likely to be disseminated and implemented.
Extensive Funding Program for Large Pragmatic Studies
We believe our Pragmatic Clinical Studies Initiative to be unique among US research funders; few organizations support such large pragmatic clinical projects. We have posted funding announcements for additional pragmatic studies and plan to select as many as 20 more projects through the end of this year. Watch our Funding Center for more information. We are eager to learn from the initiation of the first five studies to refine our process in future funding cycles.
We’re strongly committed to support large, ambitious projects that will provide patients and those who care for them with the useful, authoritative evidence they need to make the better-informed decisions that will lead to better outcomes.
We want to know what you think about this funding initiative—and all our work. Please send your comments and suggestions to [email protected].