A large amount of patient information is captured at the point-of-care or at the patient-level, information that has been described as real-world data (RWD). Health care stakeholders are enthusiastic to explore whether RWD sources and the evidence it can produced (real-world evidence or RWE) can inform and/or hasten medical product development and labeling. Importantly, RWE can inform us about patient and provider choices as well as patient outcomes to help us understand whether medical products are performing as intended in routine care. Ultimately, RWE may drive learning health systems that can benefit patients with greater precision, equity, and impact.

The Duke-Margolis Center for Health Policy engages stakeholders through the RWE Collaborative to guide high-priority efforts aimed at improving the development and use of RWE. The RWE 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 more broadly, and the RWE Collaborative strives to be at the forefront. Guided by an Advisory Group of leaders representing medical product developers, payers, research groups, providers, patient networks, and regulators, the RWE Collaborative is engaged in the following 2023 priorities:

Regulatory Acceptability of Real-World Data and Real-World Evidence…New methods of collecting data and evaluating evidence necessitates new regulatory policies and requirements. This workstream looks at where regulatory policy is and whether it needs to go to make RWD and RWE work for all stakeholders.

Regulatory Acceptability of Patient Generated Health Data…Generating high-quality evidence from patient-generated health data is one of the most promising aspects of the RWE ecosystem. Working within the Regulator Acceptability workstream’s umbrella, this project focuses on specific sources of RWD, e.g., wearable sensor data and genomics.

Leveraging Causal Inference for Real-World Evidence…The noise often associated with RWD studies can present challenges when attempting to draw conclusions. Assessing current study designs and statistical methods will help us better understand challenges, limitations, opportunities, and best practices for determining causal inference with RWE.

The Role of Real-World Evidence in Accelerated Approvals and Coverage with Evidence Development…Stakeholders are seeking to align on definitions, standards, and the appropriate role of RWE in accelerated approvals and coverage with evidence development. This workstream explores how RWD/RWE can be used to address both regulatory and payer needs for post-market evidence.

The Real-World Evidence Collaborative is led by Research Director, Rachele Hendricks-Sturrup, DHSc, MSc, MA. To learn more about the group's efforts, please contact her at rachele.hendricks.sturrup@duke.edu

Matt D'Ambrosio

Policy Analyst

Nora Emmott, MPH

Senior Policy Analyst

Rachele Hendricks-Sturrup, DHSc, MSc, MA

Research Director
Senior Team Member

Trevan Locke, PhD

Assistant Research Director

Maryam Nafie

Policy Research Assistant

Sandra E. Yankah, Ph.D.

Postdoctoral Researcher

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

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

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

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

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

A Roadmap for Developing Study Endpoints in Real-World Settings

Here, we outline a roadmap for developing, enhancing, and utilizing RWE endpoints. Duke-Margolis hosted a webinar to mark the release of the RWE endpoints paper.

Published on August 28, 2020

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

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

Brief: FDA User Fee Reauthorization and the Value of Real-World Evidence: Informed by our May Convening on the State of Real-World Evidence and our Regulatory Acceptability workstream, this September 2022 Issue Brief details the increasing use of RWE.

Brief: Cures 2.0: Building a Modern Health Data Infrastructure: In October 2021, the Real-World Evidence Collaborative published a policy brief with the Duke-Margolis Value-Based Payment for Medical Products Consortium to recommend improvements to US health data infrastructure in the wake of The 21st Century Cures Act 2.0.

Event: The State of Real-World Evidence Policy: This public conference in May 2022 provided stakeholders with a venue for reviewing progress to-date on strategic real-world data and real-world evidence policy development activities and on promising future applications of these data and evidence. Discussions covered shared themes across multiple stakeholders' public comments on recent FDA draft guidance for RWD and RWE - highlighting areas of alignment as well as opportunities for RWE moving forward.

Comment Letters: The RWE Collaborative diligently watches for guidance documents released by the US Food and Drug Administration. When the docket opens for public comment, the Duke team and workstream partners collaborate on recommendations for FDA. Below are recent comments submitted.

FDA-2020-D-2307: Real-World Data: Assessing Electronic Health Records and Medical Claims Data to Support Regulatory Decision-Marking for Drug and Biological Products; Draft Guidance for Industry

FDA-2021-D-1146: Real-World Data: Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products

FDA-2021-D-0548: Data Standards for Drug and Biological Product Submissions Containing Real-World Data

FDA-2021-D-1214: Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products

FDA-2022-D-2983: Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products

Recent Workstreams: Learning Health Care Systems: RWD for Evidence-based Decision-making and Shared Learning...the Agency for Healthcare Research and Quality defines "learning health system" as "a health system in which internal data and experience are systematically integrated with external evidence, and that knowledge is put into practice." This workstream is interested in the current state of play for LHS implementation in the United States and how RWD/E is being leveraged to advance learning health systems.

Master RWE Protocols...Traditionally, master protocols have helped clinical trials achieve greater efficiency. This workstream asks how we can bring those lessons learned to studies that use real-world data.

Real-World Efficacy: Patient Subgroups...One of the greatest advantages of RWD is that it creates evidence about groups that are traditionally excluded from clinical trials. This workstream looks at cases where real-world data and real-world evidence can advance knowledge for the benefit of specific groups of patients.

White Paper Citations Tracker: The Duke-Margolis team takes pride in our work having an impact. As thought leaders in the RWD/RWE field, the white papers published by the Collaborative have been cited in a number of different publications.

Find a comprehensive, running list here--recent as of September 2023.

"A Framework for Regulatory Use of Real-World Evidence" has been cited 98 times since publication in 2017.

"Characterizing RWD Quality and Relevancy for Regulatory Purpose" has been cited 38 times since publication in 2018.

"Determining Real-World Data's Fitness for Use and the Role of Reliability" has been cited 44 times since publication in 2019.

"Understanding the Need for Non-Interventional Studies Using Secondary Data to Generate Real-World Evidence for Regulatory Decision Making and Demonstrating Their Credibility" has been cited 20 times since publication in 2019.

"Adding Real-World Evidence to a Totality of Evidence Approach for Evaluating Marketed Product Effectiveness" has been cited 22 times since publication in 2019.

"A Roadmap for Developing Study Endpoints in Real-World Settings" has been cited 9 times since publication in 2020.

"Point-of-Care Clinical Trials: Integrating Research and Care Delivery" has been cited 2 times since publication in 2022.

"Aligning Shared Evidentiary Needs Among Payers and Regulators for a Real-World Data Ecosystem" has been cited 1 time since publication in 2022.

Advisory Group

Marc Berger Independent Consultant	Andenet Emiru Director, External Partnerships & Projects; University of California Office of the President	Eleanor Perfetto Professor, Pharmaceutical Health Services Research; University of Maryland
Elise Berliner Global Senior Principal of RWE Strategy; Cerner Enviza	John Graham Senior VP, Value Evidence and Outcomes; GlaxoSmithKline	Richard Platt Executive Director; Harvard Pilgrim Health Care Institute \| Chair, Department of Population Medicine; Harvard Medical School
Barbara Bierer Professor of Medicine; Harvard University	Matthew Harker Senior Director, Key Account Strategic Solutions; Evidation	Jeremy Rassen Co-Founder, President, Board Director; Aetion
Mac Bonafede VP, Real-World Evidence; Veradigm	Joe Henk Vice President, Clinical Program Evaluation; UnitedHealthCare	Stephanie Reisinger SVP and GM, Real-World Evidence; Flatiron
Brian Bradbury VP, Center for Observational Research; Amgen	Ceri Hirst Real-World Evidence Director; Bayer	Khaled Sarsour VP, Data Science and Digital Health, Real-World Evidence and Advanced Analytics; Janssen
Jeffrey Brown Chief Science Officer; TriNetX	Stacy Holdsworth Senior Advisor; Eli Lilly	Debra Schaumberg VP and Global Head, Strategic Development Consulting; Evidera, part of PPD clinical research business, Thermo Fisher Scientific
Adrian Cassidy Global Head of RWE for Oncology; Novartis	Ryan Kilpatrick VP and Head, Global Epidemiology; Abbvie	Thomas Seck Head of Global Regulatory Affairs; Boehringer-Ingelheim
Stella Chang SVP, Client Engagement and Partnerships; OMNY Health	Lisa Lavange Professor and Chair, Department of Biostatistics; University of North Carolina	Lauren Silvis Senior Vice President; Tempus
William Crown Distinguished Research Scientist; Brandeis University	Grazyna Lieberman Regulatory Policy and Strategy Consultant	Michael Taylor Global Head of Real-World Data; Genentech
Mark Cziraky VP, Scientific Affairs; Healthcore	Erlyn Macarayan VP, Data Science; PatientsLikeMe	David Thompson Independent Consultant
Riad Dirani VP, Global Health Economics and Outcomes Research; Teva Pharmaceuticals	Christina Mack Chief Science Officer; IQVIA \| Advisor and Representative; ISPE	Alex Vance Senior VP, Clinical Data Strategy and Operations; Holmusk
Nancy Dreyer Sole Proprietor; Dreyer Strategies \| CSO Emerita; IQVIA \| Adjunct Professor; UNC	Megan O'Brien Associate Vice President; Merck	Richard Willke Chief Science Officer; ISPOR
Omar Escontrias VP, Research, Education, and Programs; National Health Council	Sally Okun Executive Director; Clinical Trials Transformation Initiative	Bob Zambon VP, Transformational Design, Solution Architecture and Strategic Partnerships; Syneos Health

Observers

Jacqueline Corrigan-Curay, Principal Deputy Center Director, CDER, U.S. Food and Drug Administration

Carolyn Shore, Senior Program Officer, National Academies of Sciences, Engineering, and Medicine

- Please note that the above list is current as of April 2023 -

Funding

This project is made possible through the generosity of the Margolis Family Foundation, which provides core resources for the Center, as well as a combination of financial and in-kind contributions from collaborative members including AbbVie, Inc.; Amgen, Inc.; Bayer AG; Boehringer Ingelheim International GmbH; Eli Lilly & Company; Genentech Inc., a member of the Roche Group; GSK plc.; Janssen Pharmaceuticals; Merck & Company Inc.; Novartis AG; and Teva Pharmaceutical Industries Ltd.

Participation of private or non-profit organizations in Center activities does not imply an endorsement by Duke University or associated entities. As part of Duke University, Duke-Margolis honors the tradition of academic independence on the part of its faculty and scholars. Neither Duke nor the Margolis Center take partisan positions, but the individual members are free to speak their minds and express their opinions regarding important issues. Duke Margolis adheres to all institutional policies on research independence and conflict of interest.

Real-World Evidence Collaborative