FDA Framework for Use of Real-World Evidence


Image by  Gerd Altmann  from  Pixabay

Image by Gerd Altmann from Pixabay

The U.S. Food and Drug Administration (FDA) released a Framework in December 2018 for the use of real-world evidence, which is not routinely collected during the course of a clinical trial. Under the 21st Century Cures Act, the FDA was to establish a program to evaluate the use of real-world evidence to help support a new indication for an approved drug or to satisfy post approval requirements. RWE evidence has been used by the FDA for some time to monitor drug safety, but its use to determine effectiveness has been limited. The Framework outlined the future for real-world data used in clinical trials, and most recently brought about a draft guidance, “Submitting Documents Using Real-World Data and Real-World Evidence to FDA for Drugs and Biologics Guidance for Industry.”

Definitions (source):

  • 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.” Examples of these sources are Electronic Health Records (EHR) or claims.

  • Real-world evidence (RWE) is the “clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of RWD.”

Scope of the RWE program is to:

  • add or change an indication,

  • add a population, or

  • add safety or effectiveness data.

Some considerations prior to using RWD in regulatory decisions to support effectiveness are if:

  • the RWD are fit for use, meaning the data are high-quality and reliable,

  • the study design that generated the RWE has enough evidence to answer the regulatory question, or

  • the study meets regulatory requirements.

Clinical trials may move closer to the point of care to utilize the data collected during routine care in the future. For data to become more patient-centric, data may need to be collected directly from the patient from such sources as:

  • wearables

  • ePRO

  • MyStudies app, which can collect data in a HIPAA compliant manner. It is openly available for others to adapt and use as needed in clinical trials.

With the increase in RWD generated from the expanding Sentinel System, there is a wealth of data that can generate RWE to be used to inform regulatory decisions for drug and biologic effectiveness. The FDA will consider if they need to create guidance for real-world data used for these new purposes, and also if guidance needs to be expanded for electronic systems.


- The Clinical Pathways Team

Enjoy this blog? Please like, comment, and share with your contacts.