is the MOA 100% clear for every FDA approved product?
NOI agree.
was all other scientific data known before mass recommendations? NOTrue.
e.g. Covid-19 vaccines - was long term study done before the bulk of the population taken the jabs?No.
Those Qs and As you provide are correct but literally straw men. And I think you know that.
The mechanism of action is important (in part from memory) because a controlled trial wasn't done with a control group - so statistical proof of the usual (older more conventional type of proof - ie involving comparing two groups one that got treatment and one that did not but that were otherwise the same) wasn't available.
Cell products are complicated so mechanisms of action matter because its important to know what in a cell product is doing the healing when it works.
The development of medicines is a human enterprise. Risks versus benefits have to both be considered.
If there isn't a good potency assay able to predict product potency in place and a BLA was granted there could be families getting loans on houses to afford to buy their GvHD kids medicines (or what they hoped to be medicines) costing hundreds of thousands of dollars that might not actually contain an active ingredient.
The FDA leaves some wiggle room, something akin to the product doing more good than harm - that’s where Ryoncil is at - reliant on the totality of evidence!Agreed largely. More good than harm. (an expensive ineffective medicine would be harmful to families that struggled to afford it). Totality of the evidence (is or should always be the case - which is precisely why there shouldn't be questions like - how is INF-gamma accounted for - left unanswered.
The potency assays are important for medicines that are going to cost people a lot of money because otherwise (mums and dads of sick kids) they'll be financially worse off and no better in terms of health (of their families) if the medicines are not potent.
@reginaldp - when an announcement is marked price sensitive its the whole announcement. Not necessarily some detail in the announcement.
I personally believe getting feedback from the FDA/CBER that they'd accepted the potency assays would be price sensitive but its also possible that an announcement that is made instead of an expected BLA resubmission could be price sensitive precisely because it amounts to a delay on the timeline that would have occurred had the expected BLA resubmission happened as expected.
To say its was the potency related aspects of the MSB announcement that made it price sensitive seems a bit presumptive to me. Its not unreasonable but its not the only explanation of what might have made it price sensitive. In my opinion.
@Lopez - I never said High School Biology - you brought that up. I'm not even sure which part of which MSB announcement you are suggesting I'm contradicting. I'm not sure I'm contradicting anything MSB have said in announcements - I believe I'm interpreting what they've said, that I can recall - did read them at the time - differently to some holders but that doesn't mean I'm contradicting what MSB have said in announcements.
On potency assays I believe MSB had said things like they believe they've addressed them - they've said they looked at old EAP data. They've said the potency assay used in the trials was still used -but they described it as optimised - they've never said they've added another new potency assay - which they could have said if they did.
(20min delay)