Pragmatic Clinical Studies to Evaluate Patient-Centered Outcomes - Cycle 2 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.

For this solicitation, PCORI is not requiring that relevant national patient organizations, professional organizations, or payer or purchaser organizations be formally included as partners and active participants prior to contract award. However, applicants should document that they have consulted with patients and other stakeholders to identify the important decisional dilemmas and evidence needs that will drive development of the research questions, or reference previously documented decisional dilemmas. Successful applicants are required to collaborate with PCORI staff upon award of the studies to establish a project Study Advisory Committee (SAC) that is comprised of national or regional organizations that represent, at a minimum, patients or families with lived experience, relevant clinicians, payers, and health plans. 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. The SAC advises and assists the research team with refining the study questions, outcomes, and protocol. PCORI expects that most awards will be made for study designs that use randomization—either of individual participants or clusters—to avoid confounding bias. However, we recognize that exceptional opportunities may arise—by virtue of natural experiments or the existence of large registries—to address pragmatic questions using observational designs.

Please 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.