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 rachele.hendricks.sturrup@duke.edu.
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
Advisory Group
| Marc Berger Special Advisor for Real World Evidence |Retired |
Ryan Kilpatrick Head, Global Epidemiology | Abbvie |
| Elise Berliner Global Senior Principal of Real World Evidence Strategy | Cerner Enviza |
Lisa LaVange 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 |
Christina Mack VP, Epidemiology and Fellow | ISPE |
| Mac Bonafede VP, Research Consulting | Veradigm |
Nirosha Mahendraratnam Lederer Director of RWE Strategy | Aetion |
| Brian Bradbury VP, Center for Observation Research | Amgen |
Elisabeth Oehrlein Assistant VP, Research and Programs | National Health Council |
| Jeff Brown Chief Science Officer | TriNetx |
Sally Okun Executive Director | CTTI |
| Adrian Cassidy Global head of RWE for Oncology | Novartis |
Bray Patrick-Lake Director, Strategic Partnerships | Evidation |
| Stella Chang SVP, Customer Success and Engagement | OMNY |
Eleanor Perfetto Professor, Pharmaceutical Health Services Research | University of Maryland |
| Bill Crown Distinguished Research Scientist, The Healler School for Social Policy and Management | Brandeis University |
Richard Platt Executive Director, Harvard Pilgrim Health Care Institue and Chair, Department of Population Medicine | Harvard Medical School |
| Riad Dirani VP, Global Ehatlh Economics and Outcomes Research | Teva |
Jeremy Rassen Co-Founder; President: Board Director | Aetion |
| Nancy Dreyer CSO and SVP, Real-World Insights | IQVIA |
Subhara Raveedran Senior Research Scientist | Patients Like Me |
| Andrew Emmett FDA Liaison, Global Regulatory Policy and Intelligence | Pfizer |
Debra Schaumberg VP, Scientific Affairs | Evidera |
| John Graham SVP, Medical Engagement and Valued Evidence and Outcomes | GlaxoSmithKline PLC |
Thomas Seck Senior VP, Medicine and Regulator Affairs | Boehringer-Ingelheim |
| Ceri Hirst Integrated Evidence Generation Lead | Bayer |
Lauren Silvis Senior VP | Tempus |
| Stacy M Holdsworth Senior Advisor, US Regulatory Policy and Strategy | Eli Lilly and Company |
Michael Taylor Global Head of Real World Data | Genentech |
| Solomon Iyasu Executive VP RWE and Late Phase | Merck |
David Thompson Chief Executive Officer for Evidence and Access | Open Health |
| Javier Jimenez Executive VP RWE and Late Phase | Syneos Health |
Richard Willke Chief Science Officer | ISPOR |
| Brad Jordan Senior Director of Regulatory Policy | Flatiron |
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.
