What planet are the FDA on, page-95

The current SOC results in 90% mortality for grade C/D aGVHD in peds. FDA are suggesting parents should line up those kids and ask them for a 50/50 chance of getting the active arm. The specialist clinicians told the FDA during ADCOM, this is not feasible and they won't initiate such a trial. So for FDA to even mention children and RCT in the same sentence is both belligerent and disrespectful to those professionals who are responsible with setting up and running these trials.

The null hypothesis of 45% was perfectly acceptable for another drug which the FDA approved based on a single arm trial for the same indication in over 12 year olds. They couldn't convince the FDA for U12 use due to its massive safety profile which will kill them before the drug works. This is of course not mentioning the life long permanent damage their drugs do to any 'lucky' survivors over 12.

This is not an efficacy issue as this was the only question posed to ADCOM and after much discussion by experts in the field (including clinical statisticians) they voted 9-1 in favour of efficacy based on current trial data.

FDA had their mind set before meeting and were hoping for confirmation of their position. Instead they got the opposite and it appears they still feel justified and are prepared to cop the heat. And they will be getting pressure from all-comers on setting this precedent. I expect their boss is prepared to back them because they are currently looking like a bunch of hicks given this drug is already approved by highly respected regulatory authorities in the world over 4 years ago.