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
To learn more about the RWE Collaborative or to learn more about 2022 projects, please contact
Rachele Hendricks-Sturrup at email@example.com.
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
Special Advisor for Real World Evidence |Retired
Head, Global Epidemiology | Abbvie
Global Senior Principal of Real World Evidence Strategy | Cerner Enviza
Professor and Chair, Department of Biostatistics; Director, Collaborative Studies Coordinating Center | University of North Carolina
|Barbara E Bierer
Faculty Director, Multi-Regional Clinical Trials Center | Brigham and Women's Hospital and Harvard
VP, Epidemiology and Fellow | ISPE
VP, Research Consulting | Veradigm
|Nirosha Mahendraratnam Lederer
Director of RWE Strategy | Aetion
VP, Center for Observation Research | Amgen
Assistant VP, Research and Programs | National Health Council
Chief Science Officer | TriNetx
Executive Director | CTTI
Global head of RWE for Oncology | Novartis
Director, Strategic Partnerships | Evidation
SVP, Customer Success and Engagement | OMNY
Professor, Pharmaceutical Health Services Research | University of Maryland
Distinguished Research Scientist, The Healler School for Social Policy and Management | Brandeis University
Executive Director, Harvard Pilgrim Health Care Institue and Chair, Department of Population Medicine | Harvard Medical School
VP, Global Ehatlh Economics and Outcomes Research | Teva
Co-Founder; President: Board Director | Aetion
CSO and SVP, Real-World Insights | IQVIA
Senior Research Scientist | Patients Like Me
FDA Liaison, Global Regulatory Policy and Intelligence | Pfizer
VP, Scientific Affairs | Evidera
SVP, Medical Engagement and Valued Evidence and Outcomes | GlaxoSmithKline PLC
Senior VP, Medicine and Regulator Affairs | Boehringer-Ingelheim
Integrated Evidence Generation Lead | Bayer
Senior VP | Tempus
|Stacy M Holdsworth
Senior Advisor, US Regulatory Policy and Strategy | Eli Lilly and Company
Global Head of Real World Data | Genentech
Executive VP RWE and Late Phase | Merck
Chief Executive Officer for Evidence and Access | Open Health
Executive VP RWE and Late Phase | Syneos Health
Chief Science Officer | ISPOR
Senior Director of Regulatory Policy | Flatiron
Co-Founder and President | Healthcore
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
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.