NorthWest Biotherapeutics Inc (NWBO)

[exwannabe]

Gzzi62, re smithonstocks.

So I clicked on the first NWBO post I saw under "recent blogs" and it was wrt the FDA guidance on use of external controls for clinical trials.

So I took at look at that. Here are a couple issues:

1) The trial only has summary level data:

Per the FDA:

Sponsors must include in their marketing applications relevant patient-level data (i.e., data on each participant and patient in the externally controlled trial), as required under FDA regulations,for both the treatment and external control arms. If sponsors do not own the data used for the external control arm, they should structure their agreements with the data owners to ensure that patient-level data can be provided to FDA in support of the marketing application. Sponsors should also ensure that FDA has access to source documents and source data for the external control arm as part of an FDA inspection or upon request.

And the above has a footnote that this is not just guidance, but actually in formal regulations: 21 CFR 314.50(f) and 601.2.

There is a valid reason why LL and the other authors note the lack of patient level data as a limitation of the trial. And there is a reason why the FDA insists on this.

2) The ECA was not designed prior to initiation of the trial.

Per the FDA:

Reducing the potential for bias in externally controlled trials is best addressed in the design phase, in that well-chosen design elements increase confidence in the interpretability of study results when appropriate analytic methods are applied to estimate treatment effects. Sponsors should finalize a study protocol before initiating the externally controlled trial, including selection of the external control arm and analytic approach, rather than selecting an external control arm after the completion of a single-arm trial

That issue is just a "should", so not an absolute. But anytime the FDA says a sponsor should do something and they do not, it i a huge risk.