FDA Publishes Guidance on Real-Word Data

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The final guidance document provides recommendations for designing a new registry or using an existing registry to support decisions regarding safety and effectiveness of a drug.

FDA published the final guidance, Real-World Data: Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products, to provide sponsors with direction for designing a new registry or using an existing registry to support regulatory decision-making about safety and effectiveness of a drug. It does not, however, discuss choice of study design or statistical methods used to analyze registry data.

The guidance document is in response to requirements in section 505F of the Federal Food, Drug, and Cosmetic Act (FD&C Act). Section 505F created a Real-World Evidence (RWE) Program in order to evaluate the use of RWE to support approval of a new indication for a previously approved drug under section 505(c) of the FD&C Act.

The guidance document includes considerations about a registry’s fitness-for-use, linking a registry to other data sources for supplemental information, and supporting FDA review of submissions that incude data from registries. The agency discusses using registry data to support regulatory decisions, the relevance of registry data, and the reliability of such data.


The guidance defines a registry “as an organized system that collects clinical and other data in a standardized format for a population defined by a particular disease, condition, or drug exposure.” Real-word data are defined in the guidance document as “data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources.” RWE is defined by FDA as “the clinical evidence about the usage and the potential benefits or risks of a medical product derived from analysis of RWD.”

Source: FDA