FDA discusses RWD, RWE with industry, academia
Industry, academia and FDA officials gathered Friday in Bethesda, MD to discuss the use of real-world data (RWD) and real-world evidence (RWE) in the premarket and postmarket settings.
Jacqueline Corrigan-Curay, director of CDER’s Office of Medical Policy, kicked off the joint FDA-American Association for Cancer Research meeting to discuss the FDA’s framework for using RWE, which was unveiled in December 2018. The framework is meant to guide the FDA on evaluating the potential use of RWE to support changes to labeling, including adding or modifying an indication or adding comparative effectiveness or safety information.
“Transparency on study design and analysis before execution is critical for ensuring confidence in the result,” Corrigan-Curay said, noting that RWE may be able to detect long-term but infrequent outcomes.
More recently, the FDA issued draft guidance on the types of RWD and RWE information that might be submitted. Corrigan-Curay also offered several examples of when RWE has informed effectiveness determinations.
She further discussed how companies should decide if data can be considered fit for use and how the quality of the data can be assured by the FDA.
Work on RWE duplication demonstration projects is also underway, with investigators from Brigham and Women’s Hospital building an evidence base for real world data through large-scale replication of randomized controlled trials. The investigators said their goal is to understand for what types of clinical questions RWD analyses can be conducted with confidence and how to implement such studies.
In addition, the FDA is working on a demonstration project for the FDA’s MyStudies mobile app, designed to facilitate the input of real-world data directly by patients which can be linked to electronic health data supporting traditional clinical trials, pragmatic trials, observational studies and registries.
Looking forward, Corrigan-Curay pointed to efforts to increase internal FDA education on RWE and RWD, continuing to hold public meetings and obtain experts’ input, and to consider mechanisms and standards to assess RWD quality. “We welcome input on ideas related to the regulatory science of using this data,” she said, especially as the FDA identifies additional guidance and evaluates what situations can rely on observational studies.
Other sessions throughout the day discussed premarket and postmarket use cases, as well as large genomic databases and future directions for RWE.
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