Pragmatic Clinical Studies to Evaluate Patient-Centered Outcomes -- Cycle 3 2016

PCORI seeks to fund pragmatic clinical trials, large simple trials, or large-scale observational studies that compare two or more alternatives for addressing prevention, diagnosis, treatment, or management of a disease or symptom; improving healthcare system-level approaches to managing care; communicating or disseminating research results to patients, caregivers, or clinicians; or eliminating health or healthcare disparities.

Proposed studies must address critical clinical choices faced by patients, their caregivers, clinicians, or delivery systems. They must involve broadly representative patient populations and be large enough to provide precise estimates of hypothesized effectiveness differences and to support evaluation of potential differences in treatment effectiveness in patient subgroups.

PCORI’s multi-stakeholder panels have identified 20 research topics and research questions. (See Appendix for list.) PCORI will give first consideration to applications that directly address one or more of the 20 PCORI identified topics. Note that PCORI is open to receiving and reviewing LOIs for studies on investigator-initiated CER questions as well. In addition, for this cycle, PCORI is indicating a Special Area of Emphasis for two topics from the topic list: community-acquired pneumonia and pelvic organ prolapse. PCORI has set aside $80 million to fund applications in response to these special emphasis topics.

For this solicitation, applicants are not required to demonstrate that patients and other stakeholders are already engaged as research team members at the time an application is submitted. However, applicants should outline how patients and other stakeholders will participate as partners in various phases of the proposed research, once awarded. Applicants should describe their plan to form a Study Advisory Committee (SAC)¹ or other appropriate engagement body, to ensure that a broad spectrum of patients and other stakeholders advise and assist the research team with refining the study questions, outcomes, and protocols. These patients and other stakeholders must include national or regional organizations that represent—at a minimum—patients, caregivers, clinicians, policy makers, and other healthcare system stakeholders. Additional representation may be recommended in collaboration with PCORI, including individual patients with lived experience and other relevant stakeholders, such as scientific and methodological experts.

Note that this funding program does not support applications to conduct cost-effectiveness analyses, systematic reviews (with or without meta-analyses), or developing or evaluating shared decision-making or decision-support tools. In general, PCORI will not cover costs for interventions that are being compared in the proposed study. (See Appendix 3 in the Application Guidelines for details.)

This announcement is a collaborative effort of PCORI’s Comparative Clinical Effectiveness Research, Communication and Dissemination Research, Improving Healthcare Systems, and Addressing Disparities research programs.

¹ The intent of the SAC described in the PFA is to ensure that a broad spectrum of patients and other stakeholders advise and assist the research team with refining the study questions, outcomes, and protocols. These patients and other stakeholders must include national or regional organizations that represent—at a minimum—patients, caregivers, clinicians, policy makers, and other healthcare system stakeholders. Additional representation may be recommended in collaboration with PCORI, including individual patients with lived experience and other relevant stakeholders, such as scientific and methodological experts. However, PCORI understands that engagement structures and approaches vary widely. Other engagement approaches, such as forming stakeholder groups, panels, task forces, working groups, and other bodies, or involving individual patient and other stakeholder partners in various ways are also permissible to employ—either in addition to or instead of—the formation of the SAC. The SAC provision is not meant to require that a separate governance or advisory entity be established beyond the study governance and advisory structure the awardee has planned, if an applicant already has an approach for including the relevant and required patient and other stakeholder partners. For clarification in your application materials and merit review purposes, please indicate which body or structure is filling the SAC requirements, including the requirements for in-person meetings at least two times per year, and appropriate budgeting.