FDA Unveils Real-World Evidence Program Framework

Just in time for the holidays, the U.S. Food and Drug Administration (FDA) has giftwrapped and delivered the framework it plans to use to implement its Real-World Evidence (RWE) Program. FDA defines RWE as the “clinical evidence about the usage and potential benefits or risks of a medical product derived from analysis of” real-world data (RWD).

“FDA will work with its stakeholders to understand how RWE can best be used to increase the efficiency of clinical research and answer questions that may not have been answered in the trials that led to the drug approval, for example how a drug works in populations that weren’t studied prior to approval,” said Janet Woodcock, MD, director of the Center for Drug Evaluation and Research at FDA.

Among other helpful nuggets, the document offers two examples of trials generating RWE. One is a randomized aspirin-dosing related trial using RWD to assess dose response. In the second, a randomized clinical trial of a heart disease therapy uses an established registry.


Big Data, RWD and RWE: What’s the Difference and How is it Changing the Research Landscape?

Join us at ACRP 2019 in Nashville this April for a review of real-world evidence (RWE) regulatory and market impacts. Expert speaker Karri Venn of LMC Manna Research will also highlight ethical and privacy concerns and offer best practices from a clinical setting that has multiple RWE publications.

View Session Details >>


Up to now, FDA has used RWD primarily in its evaluation of safety and only in limited circumstances to inform decisions about effectiveness. The agency’s new RWE Program will focus on exploring the potential of RWD/RWE to support regulatory decisions about product effectiveness. The program will also evaluate the potential use of RWE to support changes to labeling about drug product effectiveness, including adding or modifying an indication, such as a change in dose, dose regimen, or route of administration; adding a new population; or adding comparative effectiveness or safety information.

The FDA’s framework will ultimately considering three key areas:

  • Whether the RWD are fit for use
  • Whether the trial or study design used to generate RWE can provide adequate scientific evidence to answer or help the regulatory question
  • Whether the study meets FDA regulatory requirements (e.g., for study monitoring and data collection)

The RWE Program will involve the establishment of demonstration projects, engagement with stakeholders, the use of internal processes that bring senior leadership input into the evaluation of RWD and promote shared learning and consistency in applying the framework, and the development of guidance documents to assist sponsors interested in using RWE to support their work. FDA will also explore ways to establish data standards.

Author: Michael Causey