How to Submit Documents with Real-World Data to FDA

To facilitate FDA’s internal tracking of submissions that include real-world data (RWD) and real-world evidence (RWE), the agency published a guidance on how to identify the use of RWD / RWE in submission cover letters. The cover letter should describe the purpose of using RWD / RWE, types of study designs that include RWD / RWE, and RWD sources used to generate RWE. The guidance applies to submissions for investigational new drug applications (INDs), new drug applications (NDAs), and biologics license applications (BLAs) that contain RWD / RWE intended to support a regulatory decision regarding product safety and / or effectiveness.

What are RWD and RWE?

According to the FDA,

  • RWD are data relating to patient health status and / or the delivery of health care that are routinely collected from a variety of sources (e.g., data obtained from digital health technologies).
  • RWE is the clinical evidence about the usage and potential benefits or risks of a medical product derived from analysis of RWD. RWE can be generated from RWD using many different study designs, for example, interventional studies (clinical trials) or non-interventional (observational) studies.

Examples of study designs that may be included in regulatory submissions are:

  • Randomized clinical trials that use RWD to capture clinical outcomes related to safety or effectiveness
  • Single-arm trials that use RWD in an external control arm
  • Observational studies, such as observational cohort and case-control studies, that generate RWE intended to help support an efficacy supplement
  • Clinical trials or observational studies that use RWD or RWE to fulfill a postmarketing requirement (PMR) or postmarketing commitment (PMC)

The agency recommends that sponsors and applicants not identify submissions that contain RWD / RWE if those data are not intended to support product labeling. For example, submissions using RWD only to generate hypotheses or to plan a clinical trial (e.g., to identify potential trial participants), studies incorporating RWD only in exploratory modeling or simulations, studies using RWD only to validate an endpoint, or studies using RWD only in the qualification process for Drug Development Tools should not be identified in submissions to FDA.

More information is available in FDA´s guidance document Submitting Documents Using Real-World Data and Real-World Evidence to FDA for Drug and Biological Products.

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