Research Initiative Highlights

This PCORI funding announcement invites applications for high-quality comparative effectiveness research (CER) projects that will examine a critical patient-centered research question that is also relevant to decision makers and other stakeholders. For this PFA, investigators should propose an individual-level or cluster randomized controlled trial of significant scale and scope, requiring funding in excess of $10 million in direct costs. The proposed trials should address important decisional dilemmas that require important new evidence about the comparative effectiveness of available interventions. Proposed studies should compare interventions that already have established evidence of efficacy or are in widespread use. Clinical interventions (such as medications, diagnostic tests, or procedures) and delivery system interventions are appropriate for study.

This funding announcement anticipates that the proposed research projects will require an initial feasibility phase with funding support for study refinement, feasibility testing, stakeholder engagement, and infrastructure establishment. Findings of the feasibility phase should enable rapid and successful continuation to a second phase for the conduct of the full-scale or main research study. The feasibility phase may include, but is not limited to, the development and initiation of a robust stakeholder engagement plan, piloting of data collection methods, refinement of recruitment methods and estimated enrollment, or the establishment of study sites. Continued funding of the second phase to carry out the full-scale study will be contingent upon the achievement of specific milestones and deliverables in the feasibility phase.

In light of the scale, complexity, and scope of studies being solicited under this PFA, PCORI requires that funded trials include a data coordinating center (DCC) with an independent leadership role to advise on the analytical, statistical, and data management aspects of both study phases. The DCC is expected to assist in refining the final trial design and protocol, data collection and management, and the statistical analysis plan. During the full-scale study phase, the DCC is expected to manage data reporting, monitor study enrollment and retention, assure data quality and completeness, prepare reports for the Data Safety and Monitoring Board, carry out all necessary statistical analyses of study findings, and ensure a high level of data security. Potential applicants to this PFA may submit a Letter of Intent without an identified DCC but the full application must include a DCC and describe its study role and capabilities. Likewise, full stakeholder engagement may be incomplete at the time of application; applicants must, however, provide a comprehensive plan for engagement development during the feasibility phase of the project.

Investigators may request up to $2 million in direct costs for a feasibility phase up to 18 months in duration, with up to $20 million in direct costs for continuation to a full-scale study phase up to five years in duration. Total direct costs for the full proposal should not exceed $22 million. Budgets for a data coordinating center representing more than 20 percent of total direct costs must be well-justified. 

Reminder: The primary institution’s Administrative Official must review, authorize, and submit the full application by 5 pm ET on January 12, 2021.