The U.S. Food and Drug Administration (FDA) issued its third draft guidance under the Real-World Evidence (RWE) Program on November 29, 2021. In Real-World Data: Assessing Registries to Support Regulatory Decision-Making for Drugs and Biological Products, the FDA discusses considerations for sponsors and other stakeholders when designing or using an existing registry as RWD to support a regulatory decision about the safety and effectiveness of a medicine or biologic.

The goal of the RWE program, in part, is to satisfy Congress’s mandate under section 505F of the Federal Food Drug and Cosmetic Act (FD&C Act) for the FDA to provide more guidance about the use of RWE in regulatory decision-making. We discussed the FDA’s first and second guidances, released in August and October 2021, here and here.

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The U.S. Food and Drug Administration (FDA) issued a draft guidance titled Data Standards for Drug and Biological Product Submissions Containing Real-World Data on October 21, 2021. The guidance provides the Agency’s thoughts on how sponsors can comply with the Federal Food, Drug and Cosmetic Act (FDCA) when submitting “certain” applications that contain study data derived from real-world data (RWD) sources. The FDA acknowledges that its current study data standards do not necessarily reflect a process derived from RWD sources. However, sponsors will need to convert RWD into established study data standards when submitting this information as part of a regulatory application (a process called “mapping”).

For context, study data standards are documented guidelines to help with the exchange of clinical and nonclinical study data between computer systems. They are used to provide a consistent framework for organizing study data (such as templates for datasets, standard names for variables, how to do calculations with common variables, and so on).

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The U.S. Food and Drug Administration (FDA) has issued a draft guidance for pharmaceutical companies, sponsors, and researchers sourcing Real World Data (RWD) from health records. As the FDA’s first attempt to define guidelines on sourcing RWD, the draft document provides insight into the FDA’s current thinking on the approach that companies should use to support regulatory filings for medications.

The guidance references the Framework for Real World Evidence Program, released in December 2018. The Framework is designed to regulate uses of RWD and Real Word Evidence (RWE), which are used by life sciences companies to investigate how medicines perform beyond clinical trials, and to back up clinical trial results. This draft guidance is the first of the series that have been promised by the FDA to develop the Framework. More information about the FDA Framework can be found on our blog here.

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