My reply on YMB, regarding the processing of data and what happens afterwards.
My opinion only on the timelines provided, and intended as a primer for people who may not know the details of the FDA decision process:
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They have several steps to go through on a bureaucratic level.
First of all, the Jan data we're expecting is simply what is called "top line data".
That is, they have to tediously going through EVERY patients' records of EVERY single dr visit and scan. Keep in mind this is in ~11 different countries, 100+ sites, from up to 4 years of data in some cases. Then they double check each of these to make sure they entered the info correctly. This is important because once they signal they are ready to calculate ANY unblinded data, the data is locked, even if they find incorrect data later - and the FDA will not accept any corrected data (called post-hoc analysis). And people complain because it takes weeks-to-months for this process!
Once they are satisfied the data is correct, they unblind the data - letting them see what arms the patients are in. Then it literally takes only minutes to calculate the PFS comparison.
So that is topline data. A yes/no on PFS 33% improvement powered at 80%. Company will get a "little" more color on stats, but probably not much. They'll get some data on stratifications - like tumor size 3-5cm vs. 5-7cm and RFA procedure types.
Your question is what happens then. They start going through the data with a fine tooth comb, teasing out all sorts of comparisons; gender, age, tumor size broken out more finely, and 180 other ways I can't even guess at. The FDA will want all sorts of combinations and it takes lots of person-hours to simply wade through these and actually digest the results.
Once they have a degree of comfort with the data, they can ask for a pre-NDA meeting (don't recall the official name for it). They'll get more clarity from the FDA on exactly what comparisons they should be looking at, for example. THAT MEETING CAN TAKE TWO MONTHS TO SCHEDULE. And that is AFTER they have waded thru the data.
I expect if we get data in Jan, we will file the NDA in July.
We do have FastTrack status which allows us to file a "rolling NDA". The NDA has several modules and the rolling part means we can file some of them early. We probably already have the preclinical and manufacturing (CMC) modules ready to file. But here's the thing: although we can file some of it early, NONE of it is reviewed until we have finished all of it and they have accepted the filing.
I expect the acceptance to come in August-ish. If accepted then because of Accelerated Approval we will be issued a PDUFA Date six months after the filing date. I thus expect a PDUFA date in January 2014.
They can approve or issue a CRL earlier, but I expect them to take approximately the whole time. I've seen every argument that the FDA "wants" this treatment but put no stock in it - bureaucracy reigns supreme. I'd love to be disappointed and again admit it could happen but MY OPINION ONLY is that we get approval sometime in Jan14.
I am relatively certain the overall process is as I describe but if anyone can fill in more details or correct any of it I would appreciate it.
Best,
Trond Hildahl
AI
