Use of Real-World Data and Real-World Evidence for Regulatory Decisions: Opportunities and Challenges

• Activity Number: 150 - Lead with Statistics in Medical Device Innovations and Beyond
• Type: Topic Contributed
• Date/Time: Monday, July 30, 2018 : 10:30 AM to 12:20 PM
• Sponsor: Section on Medical Devices and Diagnostics
• Author(s): Lilly Yue* and Nelson Lu and Yunling Xu
• Companies: U.S. Food and Drug Administration and U.S. Food and Drug Administration and FDA/CDRH
• Keywords: Real-world data; Real-world evidence; Regulatory decision-making

Abstract

There are many sources of real world healthcare data that could be leveraged in the clinical studies in the regulatory settings. While such large quantities of data reflect real world clinical practice and could potentially be used to reduce the cost of clinical trials, challenges arise concerning real-world data (RWD) quality, innovative methods for developing robust real-world evidence (RWE) from RWD, and appropriate use of RWE for regulatory decisions. This presentation will discuss such opportunities and challenges from statistical and regulatory perspectives, illustrated with real examples in both therapeutic and diagnostic device areas from regulatory submissions.