And relative to the second BLA what more did they have to consider that they didn't have before?
Well, presumably the handful of survivers at four years (last report) are still alive and more time has passed. And presumably the sympathetic supporters Levine, Ferrara etc are still hooked into an extensive network of MAGIC data collecting -so they'd still be offering "real world" data by way of comparison. And that would still be good. The same arguments (emotional not scientific) that got votes at ODAC would still be appealing to the same folk that don't understand the science and the stats. But that's most people. Its just not usually the FDA.
I wouldn't expect survivers to cease being survivors because their immune systems I'd expect to have been essentially reset - successfully. So I'd expect they'd keep on surviving.
But none of that changes that there were only a handful maybe eleven or so patients that got a known tightly controlled dosing of the product without confounding variables - so it still has to be the case that the product that went into the patients that did show very good longevity wasn't sufficiently characterised in a statistically large enough number - I think.
Still think more data needed with new trial in adults.
If I had to bet at this point I'd bet a repeat of history - SI cranking up the rhetoric and getting ready for another go at trying to sympathy claim an outcome where the science and stats don't justify that outcome. CRL number 3 coming up most likely in my opinion.
To be fair opinions aren't worth much without evidence and chains of reasoning behind them. Even a few errors in the drafting of the announcement changes things. I don't think the board or Silviu would lie or deliberately open themselves up to legal risk - but I can't see how they cannot be either mistaken or that the FDA has made a mistake (no amount of retrospective back-filling or data mining can make any difference to what went into the handful of patients in the GVHD trial being something and not other things at a certain point in time) if the MSB board has accurately reflected the true substance of what the FDA has said to the MSB company officers.
(20min delay)
