In response to requirements under the 21st Century Cures Act and the sixth Prescription Drug User Fee Act (PDUFA VI), the U.S. Food and Drug Administration (FDA) is exploring the use of Real-World Data (RWD) and resultant Real-World Evidence (RWE) in regulatory decision-making. Through extensive engagement with the stakeholder community, the FDA published in December 2018 a framework for the Agency’s RWE Program. The framework outlines key considerations for using RWD and RWE for regulatory decisions related to the effectiveness for marketed products and is guided by a three-part approach:
- Is the RWD fit for use?
- Does the trial or study design used to generate RWE provide adequate scientific evidence to answer or help answer the regulatory question of interest?
- Did the conduct of the study meet FDA regulatory requirements?
This conference on Developing Real-World Data and Evidence to Support Regulatory Decision-Making is funded by FDA through a cooperative agreement with the Duke-Margolis Center for Health Policy and will bring together leading experts to examine these questions, other topics addressed in the framework, as well as emerging topics in the development of real-world data and evidence.
To inform FDA’s work in this area, the Duke-Margolis Center for Health Policy launched the RWE Collaborative to engage stakeholders on key issues and high-priority efforts aimed at improving the development and use of RWE. Workshop sessions will also highlight emerging takeaways from white papers that will be released as part of the RWE Collaborative’s ongoing efforts to identify and further unpack important issues highlighted in FDA’s RWE Framework.
Funding for this conference is provided by the FDA. Support for the Duke-Margolis Center for Health Policy is provided by Margolis Family Foundation. The Real-World Evidence Collaborative is funded by a combination of financial and in-kind contributions Collaborative members including AbbVie; Eli Lilly and Company; Genentech, a member of the Roche Group; GlaxoSmithKline; Johnson & Johnson; Merck & Co.; Novartis; Teva; and UCB.