Isn't AVII the one that brings up the FDA's broad look at the ITT population? Maybe "Intent to Treat" is not a good name for this group. The information arm are treated with DCVax-L. One must assume that doesn't happen accidentally, that there is an "Intent to Treat" prior to treatment, in spite of the fact that they are not part of the trial.
If a large number of mesenchymal patients were filtered into the information arm, then what would AVII say about the health of the information arm patients being considered by the FDA?
If analysis that includes the information arm shows a 90% CR for patients that are both mesenchymal and respond to temozolomide... and that subgroup constitutes a large percentage of GBM patients, ie 30%... then how could the FDA ignore that?
I remember watching a video of a presentation by a senior FDA member about 18 months or so ago where the official said that the FDA can and will grant early marketing authorization with required confirmatory trials, for results shown in very small trials, even without controls, for indications that have no current effective therapies. He said as small as a dozen patients, then corrected himself, and said even a much smaller numbers of patients.
Although I have not been able to find that presentation in recent searches, at least one other person, more credible than myself, remembered seeing it too.
That presentation may have been the FDA's appeasing of the public after the "Dallas Buyer's Club" gained so much attention. I believe the timing was about right. Maybe the FDA back-peddled and deleted all traces of the presentation from the internet after the furvor died down... but it is hard to forget the presentation, and hard not to wonder why the efficacy I describe above, if shown in the DCVax-Trials for mesenchymal patients that respond to temozolomide (tumor shows methylated MGMT promoter), would not beg such an early marketing approval with required confirmatory to follow.
