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Session 3: Novel Randomized RWE Trial Designs to Inform Regulatory Decisions
Session Chair(s)
Jingyu (Julia) Luan, PhD
Senior Director, Global Regulatory Affairs, BioPharmaceuticals R&D
AstraZeneca, United States
James Harnett, PharmD, MS
Executive Director, Health Economics and Outcomes Research
Regeneron Pharmaceuticals, Inc. , United States
In recent years, regulators around the world encourage the industry to explore different ways to enhance the use of RWE for demonstrating effectiveness in regulatory submissions. Adopting pragmatism into Randomized Clinical Trial (RCT) design is considered a highly promising strategy of utilizing RWE in regulatory decision-making. The recent COVID-19 pandemic has drawn increased attention to novel approaches for generating evidence and will expand our understanding of the role of novel RWE trial designs. In this session, speakers and panelists from regulatory agencies, industry, and academia will discuss the design, implementation, regulatory acceptance, opportunities and challenges of novel RWE trial designs from policy, methodological, operational, and technical perspectives. Real-case studies (Registry-based Randomized Clinical Trial) that have been accepted by multiple Health Authorities will be shared.
Learning Objective : At the conclusion of this session, participants should be able to:
- Describe the basic features of novel RWE trial designs
- Identify opportunities and challenges of novel RWE trial designs
- Review case studies of novel RWE trial designs, e.g., Registry-based Randomized Clinical Trial (R-RCT)
- Discuss the possible boundaries in consideration for novel RWE trial designs
Speaker(s)
A Case Study of Registry-based Randomized Clinical Trial (R-RCT) for Registration Purpose
Charles Lee, MBA, MS
AstraZeneca, United States
Executive Regulatory Science Director
Randomized Pragmatic Clinical Trials Utilizing RWD: Myths & Realities
Frank W. Rockhold, PhD, MSc
Duke Clinical Research Institute, Duke University Medical Center, United States
Professor of Biostatistics
Panelist
Robert J. Temple, MD
FDA, United States
Senior Advisor for Clinical Science, OCD and Immediate Office, OND, CDER
Panelist
Stefan James, MD
Uppsala University, Sweden
Professor of Cardiology
Panelist
Frank W. Rockhold, PhD, MSc
Duke Clinical Research Institute, Duke University Medical Center, United States
Professor of Biostatistics
Panelist
Representative Invited
National Medical Products Administration (NMPA), China
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