The FDA released a draft guidance today titled Submitting Documents Using Real-World Data and Real‑World Evidence to FDA for Drugs and Biologics (here) that describes how the Agency will receive Real World Data (RWD) and Real World Evidence (RWE) and the potential use of each. After reading the draft guidance, it seems to me that the industry is still far away from being able to rely on RWD and RWE for use in supporting initial application approval, but it may be very useful for post-approval label changes, new patient populations, and for satisfying some post-approval commitments. This is, of course, just my opinion and perhaps I am a bit cynical about its utility, based on years of FDA indoctrination. It does, however, offer some promise.
The draft guidance provides information on the background of the concept, provides examples of how RWD and RWE could be used, discusses the purpose of RWE as part of a regulatory submission, study designs that could be used to generate the data, and the types of RWD sources that could be used to develop RWE.
There is also an appendix that provides a format for submitting RWE in the cover letter of the submission. The FDA notes that the use of this type of data and evidence may be supportive in the IND, NDA, or BLA stage, or in post-approval submissions.
This may be a new era for this type of data for regulatory submissions, but we will have to wait to see where this takes us and how useful the concept becomes.