I'm not sure whether you've discussed it more on other threads or not, but I figured it would be interesting to hear more your thoughts about this as I think we might be on the same page here.
I did some. But frankly I'm finding HotCopper difficult to read and post into at present - the poor behavior which I see as motivated by greed (traders many of whom are mostly ignorant pumping nonsense often to the other holders) I'm finding depressing as its so wide spread.
Essentially, MSB were unable to get approval of Rem-L in paediatric Gvhd. This was/is due to the current clinical trial data being not being adequately controlled and related potency assays were not sufficiently related to the mechanism of action.
Yes
However, as I read it in the paper, SI walked into the latest meeting with the FDA and was told "actually, we’ve thought this through, and you’ve convinced us that the clinical data are very strong."
Yes. Cameron England is the reporter. I wondered if he might be a rookie or someone interviewing out of his field and so might have quoted Silviu incorrectly - that rookie and out of his field doesn't seem so likely from what I saw searching on his name. He says he's interested an reported on integrity in business or some such.
Nonetheless it still seems to me that some of what he quotes Silviu as saying the FDA said still almost beggars belief - to the point where the FDA according to Cameron quoting Silviu is almost giving business advice in pithy little quotes like (haven't got it in front of me so I'll paraphrase ('pediatrics looks good go ahead and then do the adults later').
This is...weird. Why would the FDA assess this twice, come back with the same outcome both times (you need to do another well-controlled trial), but now backflip and essentially admit their previous two assessments were wrong?
Indeed.
My best guess (and I have put no effort into researching this), is that there is a new person assessing regenerative therapies at the FDA.
I understand some of the personnel have gone - Steve Bauer for instance and some others that were at ODAC - but not I believe Matt Klinker who was the principal investigator and I though had become the head of the dept that Steve Bauer used to head. Its inconceivable to me that he could reverse himself so much without new data (to be absolutely clear - I think he was absolutely right in his science and stats assessment and I know from reading his papers with Bauer that he really did understand MSC potency at an expert level).
A new person (in my opinion they'd have to be a less capable person or persons or someone caving to political pressure from inside) because the science is clear. (I'm open to being wrong on the science too btw but I honestly don't think I am and there seems to be no-one around on the HC forums that can go with me on it to point out what I'm missing (and every time I try to engage on the science the idiot traders attack my character and motives to shut me up so they can resume pumping their bs- pfeifer seems to be the most thorough researcher and seems to understand the science stuff afterwards even to the point where he can condense what I say but I say this trying to be respectful I think he possibly hasn't got quite the same level of understanding of some cell biology technologies that would be behind things like cell sorting maybe. I'm open to being wrong on the science too - but the engagement isn't there on that. And I got the call right ahead of the CRL # 2).
TLDR - New personnel are a possible explanation. But I still think human error - Silviu misinterpreting or hearing what he wants to hear and then dumbing down so he gets quoted in more pithy sand bites for better copy (with respect to the paper) seems more likely.
Would you be interested in emailing Cameron England the reporter with me and saying saw you article and was surprised and impressed by the quotes relating to Silviu as they sound a little different to how we expect the FDA to speak would he mind confirming that that was exactly what Silviu actually said and that he didn't accidentally abbreviate him or something?
I am certainly not suggesting Cameron England would have done anything other than make an innocent mistake - but he might confirm and say - actually I'm sure I recorded Silviu and that's exactly what he said.
They are of the opinion that a trial can be "adequately controlled" with no control arm in conditions where survival outcomes on standard of care are low. This flies in the face of the FDA approval process, but short of suggesting something criminal has occurred, or that SI was just talking out his backside in the article (which I guess would also be criminal), this is the only way I can see MSB as having gotten a green light to proceed without another clinical trial.
What are your thoughts?
Well my thoughts are above. If the FDA does have new personnel and they do approve this - it would be a serious error in my opinion - like a judge getting something wrong.
They would have to validate the potency assay implicitly to give a BLA -and honestly I don't think on the science they should do that - but if they did, the US patent office would likely not second guess the FDA over a potency assay and the USPTO might patent a broad patent that doesn't actually work but could block others from using some natural occurring pathways.
(20min delay)