Using Real-World Evidence: FDA Workshop on 13 Sep 2017

Dear All: 

There is a newly announced FDA workshop on 13 Sep on using real-world evidence. Here is the link and I’ve also reproduced the key background text below my signature.

This is of interest to the antibacterial community as our reliance on non-inferiority trials for the majority of our data means that we are often going to want to use open-label real-world data as part of the value argument for new agents. The workshop unfortunately overlaps with the CDRH workshop on diagnostic devices, but I look forward to see the output from this parallel discussion!

All best wishes, –jr

John H. Rex, MD | Chief Medical Officer, F2G Ltd. | Chief Strategy Officer, CARB-X | Expert-in-Residence, Wellcome Trust. Follow me on Twitter: @JohnRex_NewAbx. See past newsletters and subscribe for the future:

Upcoming meetings of interest to the AMR community:

Background from FDA’s web page at
RWD (data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources) and RWE (clinical evidence regarding the usage and potential benefits or risks of a drug derived from analysis of RWD) are increasingly being used by multiple stakeholders within the health care system. Payers may rely on RWD and RWE to refine formularies or assist in coverage decisions. Physicians and professional societies can utilize RWE to further tailor clinical practice guidelines and decision-support tools. Medical product developers can use RWE to further develop a product’s benefit-risk profile, monitor postmarket safety and adverse events, or generate additional hypotheses for continued clinical development.

The 21st Century Cures Act, section 3022 (Pub. L. 114-255), enacted on December 13, 2016, directed FDA to establish a program to evaluate the potential use of RWE. The framework of the program was to include information describing the sources of RWE, the gaps in data collection, standards and methods for collection and analysis, and the priority areas and challenges.

To date, RWD and RWE have been used in very specific regulatory contexts. Some treatments for rare diseases, for example, have utilized RWE as part of the historical controls used for clinical study and, ultimately, regulatory submission. Postmarket safety surveillance has also relied heavily on RWD-generating networks. As part of exploring the opportunities for enhanced use of these types of data and evidence in additional regulatory decision-making contexts, FDA is seeking input on the opportunities and challenges in using RWE to support the approval of a new indication for an already approved drug, and to help support or satisfy postapproval study requirements.

This public workshop is being held to engage external stakeholders in discussions around the current state of RWE development and potential challenge areas for using RWE in regulatory decisions beyond postmarket safety surveillance. 


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