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 2021 priorities:

  • Embedding Practical Trials in Routine Care Delivery
  • Identifying Shared Real-World Evidentiary Opportunities
  • Developing Master RWE Protocols for Key Research Questions
  • PGHD Pilot Implementation Case Studies

 


racheleimage

To learn more about the RWE Collaborative or to learn more about 2021 projects, please contact

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

 


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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Advisory Group

 

Aylin Altan

Senior Vice President of Research, OptumLabs

Carlos Garner

Vice President, Global Regulatory Affairs, Eli Lilly and Company

Eleanor Perfetto

Senior Vice President, Strategic Initiatives, National Health Council

Marc Berger

Co-Chair, Special Advisor for Real World Evidence, ISPOR

John Graham

Senior Vice President, Medical Engagement and Value Evidence & Outcomes, GlaxoSmithKline

Richard Platt

Executive Director, Harvard Pilgrim Health Care Institute and Chair, Department of Population Medicine, Harvard Medical School

Barbara Bierer

Faculty Director, Multi-Regional Clinical Trials Center of Brigham and Women’s Hospital and Harvard

Solomon Iyasu

Vice President, Head of Pharmacoepidemiology, Center for Observational and Real World Evidence, Merck & Co.

Jeremy Rassen

Co-Founder, President, Chief Science Officer, Aetion

Brian Bradbury

Vice President, Center for Observational Research, Amgen

Ryan Kilpatrick

Head, Global Epidemiology, AbbVie

Stephanie Reisinger

Vice President and General Manager, Life Sciences, Veradigm Health

William Capra

Senior Director, Data Science, Roche Pharmaceuticals, Genentech

Christina Mack

RWE Task Force, ISPE

Debra A. Schaumberg

Vice President, Scientific Affairs, Evidera

Adrian Cassidy

Global Head RWE, Novartis Pharmaceuticals Corporation

Subhara Raveendran

Senior Research Scientist, PatientsLikeMe

Thomas Seck

Senior Vice President, Medicine & Regulatory Affairs, Boehringer Ingelheim

William Crown

Distinguished Research Scientist, The Heller School for Social Policy and Management at Brandeis University

Irene Nunes

Vice President, Regulatory Affairs, Flatiron Health

Lauren Silvis

Senior Vice President, Tempus

Riad Dirani

Vice President, Global Health Economics and Outcomes Research, Teva Pharmaceuticals

Sally Okun

SallyOkun360

David Thompson

Senior Vice President, Syneos Health

Nancy Dreyer

Chief Scientific Officer & Senior Vice President, IQVIA

Bray Patrick-Lake

Director, Strategic Partnerships, Evidation

Richard Wilke

Chief Science Officer, ISPOR

Andrew Emmett

FDA Liaison, Global Regulatory Policy & Intelligence, Pfizer

 

Marcus Wilson

Co-Founder and President, HealthCore

 

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

 

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.