First, the company needs to worry about not getting an RTF if they make a submission.  That is not a trivial obstacle.  This site has a lot of amateur statisticians that have thrown the BS flag more than once (rightly so in my opinion), but the FDA has a small army of them, most with PhD's in stats.  If you want to see what the FDA statisticians come up with then look at some past ADCOM reamings (the warm welcome James Bianco and CTIC received comes to mind).

 

At the ADCOM meetings, FDA goes over their view of the file, including the statistical analysis.  The alpha spend will surely come up, as will the tortured history of the clinical trial with all its starts, stops, and pivots.  So yes, all of that comes up for discussion at an ADCOM, if there is an ADCOM meeting called. 

 

At the end of the day, the ADCOM is there to talk about the strength of the evidence for approval of the file.  None of the anecdotal evidence from UCLA and other postulated upsides are going to make it onto the agenda.  At the end of the day, the committee has to decide on the evidence admitted, sort of like juries who have to decide on the evidence the judge has allowed them to see.

 

Which brings us back to the key question; when is the company going to submit a file?