FDA’s recently finalized its guidance document, Real-World Data: Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products1, as part of its Real-World Evidence program. This guidance provides a brief overview of key factors when considering the use of registry data to evaluate a specific safety risk, to characterize long-term effectiveness outcomes, or to serve as an external control for an interventional study. Strengths and limitations of registry data for these purposes are reviewed, reminding researchers of key epidemiologic principles such as representativeness of the registry population, data standardization and consistency, and understanding missing data and loss to follow-up. Important considerations for ensuring that a registry includes relevant and reliable data when designing a de novo registry as well as considerations when evaluating existing registries’ fitness for use in answering specific study objectives are noted. The guidance also provides a useful high-level list of important data elements integral to a robust registry.
UBC’s experience in designing and conducting disease and exposure registries, as well as integrating data from multiple sources for individual patients or from multiple registries to increase the power of a study, can provide deeper insights into the concepts highlighted by FDA in this guidance and assist Sponsors in the most efficient and effective ways to create and use registries.
UBC helps biopharma mitigate risk, address product hurdles, and demonstrate safety, efficacy, and value under real-world conditions. Bringing over 30 years of experience, UBC is uniquely positioned to develop end-to-end integrated evidence generation strategies, identify fit-for-purpose data sources, operationalize planned studies, and ensure regulatory-grade, publishable outputs.
ABOUT THE AUTHOR:
Annette Stemhagen, DrPH, FISPE
Dr Stemhagen, UBC’s Chief Scientific Officer, has a long history of registry involvement, having served as the Director of the New Jersey State Cancer Registry and the Principal Investigator for a National Cancer Institute Surveillance, Epidemiology, and End Results (SEER) registry. She currently provides strategic consultative services and program oversight to pharmaceutical and biotech clients in epidemiology, safety surveillance, and risk management, focusing on observational research and real-world evidence.
1. U.S. Food & Drug Administration. Real-World Data: Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products. Guidance for Industry. December 2023. Accessed January 9, 2024. https://www.fda.gov/media/154449/download