The U.S. Food and Drug Administration (FDA) released its final guidance on the use of real-world data in regulatory submissions, detailing how drug companies should assess data from electronic health records and medical claims to support their applications.
The guidance document is the finalized version of the draft released on September 30, 2021.
The FDA is issuing the document as part of its Real-World Evidence program, noting that it builds on the agency's 2013 guidance, "Best Practices for Conducting and Reporting Pharmacoepidemiologic Safety Studies Using Electronic Healthcare Data Sets."
While the agency noted in its guidance that it doesn't provide recommendations on the choice of study design or the types of statistical analysis, data source, or study methodology, it emphasizes the reliability and relevance of the data included to support a regulatory decision. To be reliable, the data must be accurate, complete, and traceable; to be relevant, the data must cover key study variables (e.g., exposures, outcomes, covariates) and include sufficient numbers of representative patients for the study.
The FDA also stressed that the guidance should be viewed as recommendations based on the agency's current thinking rather than as legally enforceable responsibilities unless regulatory or statutory requirements are cited.
