Project Summary

Background: PCORI promotes comparative effectiveness (CER) research, using various designs and data, to inform patient decisions. Researchers, policy makers, and regulators currently are establishing standards and structure for expanded real-world evidence (RWE) use. As trusted arbiters of comparative evidence for their members, patient groups are key to optimal RWE use for CER. While some groups understand RWE, it is new for most. Knowledge gaps and fears persist, and training and tools are needed to enhance RWE engagement and use. The training and tools patient groups desire do not exist, but are needed to enhance patient centricity of CER based on RWE in an environment of RWE proliferation.

Proposed Solution to the Problem: Led by the NHC and in collaboration with the Duke Margolis Center for Health Policy RWE Collaborative, the project team will create a training program with the desired resources/tools for patient groups to increase capacity to engage in RWE co-development and use. The training will be informed by an advisory committee that includes patient groups.

Objectives: Develop a RWE training program targeted at patient groups with patient­group-identified resources/tools for enhancing their capacity to engage in RWE co­development, use, and dissemination with application to CER.

Activities: There will be four phases to this project: needs assessment; curriculum development; delivery; and evaluation. The advisory committee will co-develop and approve all final materials to ensure the curriculum is patient-driven.

Outcomes and Outputs (projected): Long­term goal: improve patient healthcare decisions through their improved use of rigorous CER. The primary outcome will be increased patient­ community capacity to co­-develop, understand, use, and disseminate RWE. More specifically, in 2020, the team expects to train over 100 staff members from patient groups. It is expected that increased involvement of patient organizations in RWE will improve quality and relevance of CER to real-world patients, resulting in improved patient decision making and subsequent health outcomes. The curriculum will be developed with supporting resources that can be used by others and shared by PCORI.

Patient and Stakeholder Engagement Plan: The multidisciplinary advisory committee and NHC patient-group members will guide the work and take part in co-creation and co-delivery and will participate in the training. Participants from PCORI-funded research projects will serve as case examples of CER co-development using RWE.

Project Collaborators: This project is led by staff from the National Health Council and Duke-Margolis Center for Health Policy. It is guided by a multidisciplinary advisory committee of representatives from: Academy of Managed Care Pharmacy; Accelerated Cure Project for MS; Foundation for Prader-Willi Research; Go2 Foundation for Lung Cancer; Health eHeart Alliance and Rowan Tree Perspectives, LLC; International Society for Pharmacoeconomics and Outcomes Research; National Pharmaceutical Council; National Organization for Rare Disorders; People-Centered Research Foundation; University of Maryland School of Pharmacy; University of Washington.

Project Information

Eleanor Perfetto, MS, PhD
National Health Council

Key Dates

18 months


Project Status
State State The state where the project originates, or where the primary institution or organization is located. View Glossary
Last updated: November 30, 2022