Replies to post #774103 on NorthWest Biotherapeutics Inc (NWBO)

[manibiotech]

So the question still exists , then why haven't they applied to FDA in half a decade after data lock . 

[GPha]

NWBO has the potential to collect Real-World Data (RWD) through a compassionate use or expanded access program, assuming patient outcomes are tracked in a consistent and structured way.

According to the FDA’s finalized guidance from August 2023, real-world data includes information about patient health and healthcare delivery that is routinely gathered from various sources.

Expanded access is mentioned among those sources. When this type of data is used to evaluate treatment effects or outcomes, it qualifies as Real-World Evidence (RWE), which the FDA has been giving more weight to in recent years, especially following the passage of the 21st Century Cures Act.

[dennisdave]

Hopeforthefuture3, this is very common misconception, and often repeated Bear narrative.

aah so Dr Liau (which Hopeforthefuture3 literally quoted on this but you skipped that of course) and the the American Brain Tumor Association are bears now, according to you? Ok got it


https://www.raps.org/News-and-Articles/News-Articles/2023/5/Stakeholders-want-more-clarity-from-FDA-on-using-d

An FDA proposal to require the inclusion of patient-level data in marketing applications may prevent the use of external controls for some rare diseases, the American Brain Tumor Association said in their comment. “[I]ndividual patient-level data (IPD) is not always available for many cancers and other rare diseases,” they wrote. “This is due to many barriers that interfere with data sharing in healthcare

Dr Liau:

oh yeah let's see as far as the control group goes was
33:36
there any thought or discussion with the FDA about using external controls instead of randomization to receive both
33:43
yes we we looked at that briefly FDA would not accept any external control plan that we presented to them
33:50
um what they had offered was that we had to look at patient level external controls what that means is that every
33:56
patient on study would have to be matched with a patient from a database and as we pursued the databases that
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were out there which is kind of surprising to us there's not enough data to match these individual patients