Real-World Evidence Collaborative

Real-World Evidence Collaborative


Randomized controlled trials (RCTs) are traditionally used by the US Food and Drug Administration (FDA) to evaluate a product’s safety and effectiveness. While they are the gold-standard from a research perspective, RCTs are resource-intensive and require investing significant financial, time, and human capital. Coupled with unmet clinical needs and drug-access challenges, the demands of RCTs have fueled a call to action from patients and legislators. The regulatory system needs new ways to optimize the drug development process, so the delivery of effective drugs to the marketplace accelerates—while public safety remains ensured.

A large amount of patient information is captured at the point of care. Health care stakeholders are enthusiastic to explore whether that information can inform and hasten new drug development. Called real-world data (RWD), analyzing this burgeoning data source has the potential to provide real-world evidence (RWE). In turn, that RWE can tell us about the use of drugs, drug outcomes, and it can help us understand whether drugs are performing as intended in routine care.

The 21st Century Cures Act and the sixth Prescription Drug User Fee Act (PDUFA VI) set milestones for FDA to explore the use of RWE in regulatory decision-making. To inform FDA as it works to meet these milestones, the Duke-Margolis Center for Health Policy RWE Collaborative engages stakeholders to guide high-priority efforts aimed at improving the development and use of RWE. The Collaborative also strives to drive progress in the use of real-world data and evidence to improve patient treatment options and outcomes more broadly. Guided by an advisory group consisting of leaders representing medical product developers, payers, research groups, providers, patient networks, and regulators, the RWE Collaborative is interested in the following 2022 priorities:

  • Regulatory Acceptability of Real-World Data and Real-World Evidence
  • Learning Health Care Systems: RWD for Evidence-based Decision-making and Shared Learning
  • Master RWE Protocols
  • Real-World Efficacy: Patient Subgroups

 


racheleimage

To learn more about the RWE Collaborative or to learn more about 2022 projects, please contact

Rachele Hendricks-Sturrup at rachele.hendricks.sturrup@duke.edu.

 


Our Work

A Framework for Regulatory Use of Real-World Evidence

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

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Characterizing RWD Quality and Relevancy for Regulatory Purposes

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

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Determining Real-World Data’s Fitness for Use and the Role of Reliability

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

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Understanding the Need for Non-Interventional Studies Using Secondary Data to Generate Real-World Evidence for Regulatory Decision Making, and Demonstrating Their Credibility

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

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Adding Real-World Evidence to a Totality of Evidence Approach for Evaluating Marketed Product Effectiveness

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

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A Roadmap for Developing Study Endpoints in Real-World Settings

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

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Point-of-Care Clinical Trials: Integrating Research and Care Delivery

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

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Aligning Shared Evidentiary Needs Among Payers and Regulators for a Real-World Data Ecosystem

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

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Advisory Group

 

Marc Berger
Special Advisor for Real World Evidence | Retired

Carol Koro
Executive Director, Pharmacoepidemiology, Center for Observational and Real-World Evidence  | Merck

Elise Berliner
Global Senior Principal of Real World Evidence Strategy | Cerner Enviza

Lisa  LaVange
Pofessor and Chair, Department of Biostatistics; Director, Collaborative Studies Coordinating Center | UNC

Barbara E Bierer
Faculty Director, Multi-Regional Clinical Trials Center of Brigham and Women’s Hospital and Harvard | Harvard University (Multi-Regional)

Christina Mack
Vice President, Epidemiology and Fellow | ISPE

Mac Bonafede
Vice President, Research Consulting | Veradigm Health

Nirosha Mahendraratnam Lederer
Director of RWE Strategy | Aetion

Brian Bradbury
Vice President, Center for Observation Research | Amgen

Sally Okun
Executive Director | CTTI

Jeff Brown
Chief Science Officer | TriNetx

Bray  Patrick-Lake
Director, Strategic Partnerships | Evidation

Adrian Cassidy
Global Head of RWE for Oncology  | Novartis

Eleanor Perfetto
Professor, Pharmaceutical Health Services Research | University of Maryland

Stella Chang
SVP, Client Engagement and Partnerships | OMNY

Richard Platt
Executive Director, Harvard Pilgrim Health Care Institute and Chair, Department of Population Medicine Harvard Medical School | Harvard University (Medical School)

Bill Crown
Distinguished Research Scientist, The Heller School for Social Policy and Management, Brandeis University | Brandeis

Jeremy Rassen
Co-Founder; President; Board Director | Aetion

Riad Dirani
Vice President, Global Health Economics and Outcomes Research, Teva Pharmaceuticals | Teva

Subhara Raveedran
Senior Research Scientist | Patients Like Me

Nancy Dreyer
Special Advisor for Real World Evidence | Retired/ISPE

Stephanie Reisinger
Senior Vice President and GM, Real-World Evidence | Flatiron

Andrew Emmett
FDA Liaison, Global Regulatory Policy & Intelligence   | Pfizer

Khaled Sarsour
VP, Data Science Analytics and Insights | Janssen

Omar Escontrias
Vice President of Research, Education, and Programs | National Health Council

Debra Schaumberg
Vice President, Scientific Affairs | Evidera

John Graham
Senior Vice President, Medical Engagement and Value Evidence & Outcomes | GlaxoSmithKline PLC

Thomas Seck
Senior Vice President, Medicine & Regulatory Affairs | Boehringer Ingelheim

Scott H Kollins
Chief Medical Officer | Holmusk

Lauren Silvis
Senior Vice President, Tempus | Tempus

Joe Henk
Vice President Clinical Program Evaluation | UnitedHealthCare

Michael Taylor
Global head of Real World Data | Genentech

Ceri Hirst
Integrated Evidence Generation Policy Lead | Bayer

David Thompson
Chief Executive Officer | Open Health

Stacy M Holdsworth
Senior Advisor, US Regulatory Policy and Strategy | Eli Lilly and Company

Richard Willke
Chief Science Officer, International Society for Pharmacoeconomics and Outcome Research | ISPOR

Ryan Kilpatrick
Head, Global Epidemiology | AbbVie

Marcus Wilson
Co-Founder and President, Healthcore | Healthcore

Bob Zambon
VP, Transformational Design, Solution Architecture & Strategic Partnerships | Syneos Health

 

Observers

Jacqueline Corrigan-Curay, Director, Office of Medical Policy, CDER, U.S. Food and Drug Administration

Amanda Wagner-Gee, Program Officer, National Academies of Sciences, Engineering, and Medicine

 

- Please note that the above list is current as of June 2022 -

 

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; Amgen; Boehringer Ingelheim; Eli Lilly and Company; Genentech, a member of the Roche Group; GlaxoSmithKline; Merck; Novartis; Pfizer; and Teva.

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.