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Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making

Real-World Data (RWD) are data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources.

Real-World Evidence (RWE) is the clinical evidence about the usage and potential benefits or risks of a medical product derived from analysis of RWD.

The guidance discusses the applicability of FDA's investigational new drug (IND) application regulations under 21 CFR part 312 to clinical study designs that use real-world data (RWD) and clarifies the agency's expectations for sponsors submitting new drug applications or biologics license applications using RWD to support the safety or effectiveness of a drug in clinical studies that are not subject to 21 CFR part 312.

For non-interventional (observational studies), which are not subject to FDA’s IND regulations, the draft guidance outlines the agency’s expectations regarding the design and conduct of such studies including access to relevant datasets, study monitoring, maintaining case records, and early discussions with the relevant review divisions.

FDA is issuing this guidance to assist stakeholders in meeting its expectations and to ensure that the agency can appropriately evaluate non-interventional studies that are submitted in marketing applications to support the safety and effectiveness of a drug.

The draft guidance includes recommendations for sponsors, researchers, and other interested stakeholders proposing to use data from electronic health records (EHRs) or medical claims to help support a new use of an FDA-approved drug or biological product or to help support or satisfy post-approval study requirements.

The recommendations in this draft guidance are relevant for all study designs aiming to use EHR or medical claims data to help demonstrate a drug or biological product’s safety or effectiveness for regulatory purposes.


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