RWE for 2nd MSB COVID-19 ARDS P3 Trial, page-781

You could be right, I'm simply trying to look at the problem from the FDA's point of view, and this is what they believe

1.They don't think efficacy has been demonstrated because they don't believe the control arm is valid ( Not because they don't think it works )

2.There was no link between potency and efficacy previously.

I assume now that the company has provided in the IND update - several comparator arms for the FDA to consider, that validate the assumptions that MSB made, ontop of the new prognostic indicator paper being the MAP algorythem vs survival that Remestemcel achieved, and ontop of that the 4 years survival data.

1. The FDA now need to re-check the potency assay results against the long term survival numbers to confirm if potency is linked to survival. ( I still really don't understand how they do this in the treated arm ) They are ignoring controls, so looking at the data, if they all reacieved the same potency product and 50% died, 50% survived, how is that linked? I think they must include control data here from one of the comparator arms.

2. If the FDA tell MSB the potency assay is good - go ahead and run this 2 year trial. Then at approval Ryoncil is rejected as I stated before... because of this reason - so as not to appear hypocrites ( How can the data be good enough to say use that assay, but not good enough to approve ?). If I were the FDA and I said no more trials until the potency assay is fixed. I would not declare it " fixed" unless I was 100% certain it was fixed. Ie a formal decision as part of the CRL BLA response.


But

you could be right, and it might be some informal earlier process, I just don't think it's likley looking at the track record the FDA have.