Real-World Evidence Collaborative White Papers
The Duke-Margolis Institute for Health Policy engages stakeholders through the RWE Collaborative to guide high-priority efforts aimed at improving the development and use of RWE. The Collaborative’s work is intended to drive progress in RWE policy and help inform regulatory agencies, including the FDA, and other key stakeholders with a vested interest in informing the present and future of RWE policy. Using RWD and RWE can improve patient treatment options and outcomes, and the RWE Collaborative strives to be at the forefront of new developments.
Our research has culminated in 15 seminal white papers. Each offers expert thought on a burgeoning RWD/E policy area and attempts to build consensus on these topics, setting the stage for further inquiry and policy refinement.
Operational Tools and Best Practices to Support Electronic Health Record-Sourced Data Quality, Relevance, and Reliability at the Data Accrual Phase
Our first EHR paper focuses on data accrual. It provides a landscape of tools, best practices, use cases and considerations to help stakeholders understand how EHR-sourced real-world data may be useful for regulatory decisions.
Published on February 14, 2025
Applying Real-World Data and Real-World Evidence for Accelerated Approvals and Coverage Decisions
The Accelerated Approvals paper looks at how real-world data and evidence can contribute to the knowledge-based used to make regulatory and payer decisions about medical products that participate in the US FDA's accelerated approvals pathway.
Published on July 25, 2024
Real-World Evidence to Support Causal Inference: Methodological Considerations for Non-Interventional Studies
The ability to infer causality is crucial for determining whether a study can inform a decision about a medical product's safety or effectiveness. The Causal Inference paper discusses how to promote RWD/E studies capacity for measure and determine causality.
Published on June 18, 2024
Regulatory Fit-for-Purpose Considerations for Patient-Generated Health Data
A critical source of real-world data are patients themselves. The Patient-Generated Health Data paper summarizes how to determine if PGHD is fit-for-purpose, and it offers practical considerations to support medical product development with PGHD in mind.
Published on May 31, 2024
Lessons from Learning Health Care Systems and Recommendations for Successful Implementation
The learning health care systems (LHS) paper features original research on how U.S. health systems use real-world data to inform their processes and decision-making. Recommendations based on these findings are proposed, so LHS principles can be further applied across organizations.
Published on October 30, 2023
Harnessing the Potential of Real-World Evidence Master Protocols
Despite being a well-established method of testing multiple hypotheses within a single clinical trial, master protocols are not fully conceptualized to support RWE studies. In the Master Protocols paper, we describe key characteristics of RWE MPs and how they can support the development of critical evidentiary needs for medical products.
Published on October 30, 2023
Improving Patient Subgroup Representation with Real-World Data
Patient-level data generated in RWE studies can support evidence generation on traditionally underrepresented and understudied patients. The Patient Subgroups paper describes opportunities and challenges for using RWD to improve care across the breadth of all patients.
Published on September 28, 2023
Aligning Shared Evidentiary Needs Among Payers and Regulators for a Real-World Data Ecosystem
The Shared Evidentiary Opportunities (SEO) paper uses disease use cases to explore building a robust real-world data ecosystem. This white paper pools the expertise of both the RWE Collaborative and the Center's Value for Medical Products Consortium.
Published on July 20, 2022
Point-of-Care Clinical Trials: Integrating Research and Care Delivery
In our Point-of-Care (POC) paper, we set a baseline for what defines and constitutes a point-of-care trial and explore the current impediments to implementing these burgeoning research methods.
Published on May 11, 2022
Adding Real-World Evidence to a Totality of Evidence Approach for Evaluating Marketed Product Effectiveness
In the totality of evidence paper, we explore the feasibility of overcoming the challenges faced by conducting interventional studies in the real world. How do we address ethical, operational resource barriers? What must be considered when demonstrating the credibility of a non-interventional study using secondary data?
Published on December 19, 2019
Understanding the Need for Non-Interventional Studies Using Secondary Data to Generate Real-World Evidence for Regulatory Decision Making, and Demonstrating Their Credibility
Non-interventional studies using secondary data can better reflect broader patient populations, care settings, and drug uses found in routine clinical practice. However, questions remain as to when these types of studies are applicable. This paper about credibility and observational methods explores several of those questions.
Published on November 25, 2019
Determining Real-World Data’s Fitness for Use and the Role of Reliability
By focusing on electronic health records and patient generated health data, the data reliability paper describes a framework for how researchers and reviewers can systematically evaluate whether RWD are fit for use in regulatory decision-making.
Published on September 26, 2019
Characterizing RWD Quality and Relevancy for Regulatory Purposes
Not all RWD is created equal. Regulators need access to ample, well-sourced, fit-for-purpose data, and the data quality paper explains what that means.
Published on October 1, 2018
A Framework for Regulatory Use of Real-World Evidence
In our first white paper, we define real-world data and real-world evidence and provide context to its role in regulatory decision-making. Consider this a foundation upon which subsequent white papers are built.
Published on September 13, 2017
