Hi
@Pledge,
"
Why are they targeting adults "at the highest mortality risk......where 90 day survival remains as low as 20-30%"? Savings those that are less sick but will die is not enough now?
How is THAT "substantial clarity on how to bring this much needed product to these patients".
Clarity = fix those that have no hope or forget it? That's what he's taken out of the latest? "
it's not my field, but I suspect that the reason MSB would be proposing a prospective interventional study narrowly targeting this high-mortality adult group is because of something FDA has indicated/ said to MSB personnel at some late stage in the review process.
I suspect it was regarding FDA conceding an unarguable proof of the MAGIC 'pudding' (Ryoncil) as being in how it deals with unarguable salvage cases i.e. where the biostatisticians can get "comfortable" (to use CEO Itescu's tart expression) that the risk of ALL confounding factors causally intervening between the treatment and the observed effect is negligible, so that the outcomes of this prospective study must then be accepted at face value by the FDA.
Perhaps the context was the review of the EAP outcomes i.e. under Protocol 275, where the retrospective inclusion of the EAP outcomes from comparable product as comparable with MSB's GVHD-001 outcomes from the same upgraded product was being sought by MSB. I'm speculating here.
It may be that, at the end of the day, the FDA biostatistical reviewers could not bring themselves to allow that all of the possible confounding factors they could think of were addressed by the revalidation of the potency assay & its re-application to the EAP remestemcel-l product. Did the FDA approach the process commitment to MSB with genuine enthusiasm and intent to find reasons to approve (cf. reasons not to approve)? Did they have the kids in mind?
Who knows. I don't. I'm focussed on what's needed to fix the situation. That's why I'm trying to understand it.
Cheers. Hope to see you at the AGM.
(20min delay)