"It sounds close and there is hope that they pull this off without another trial"
This is where I disagree... Here's my take on it ... Trials are expensive. It's useful for both MSB and FDA to agree on all the trial parameters. There's no point doing a big trial on something, measuring a bunch of stuff, collating results and then going to the FDA only for the FDA to say,
"uuhhh ... why on earth did you do the trial in that manner? We don't agree that what you're measuring or how you're measuring it is a good indicator of the dynamics you're trying to resolve"
I'm not saying that MSB have done this ... I'm simply demonstrating that defining the "parameters of acceptable data and evidence" is good to establish beforehand.
So what does this have to do with the OTAT meeting? ... please note - this is just MY interpretation of what happened ....
FDA : "look, we want to work with you, we tend to believe your product does in fact work, but let's ALL agree on a bunch of parameters BEFORE we go any further"
MSB : "ok fine. How about we use ABC as the markers for Chemistry, Manufacturing and Controls (CMC). We believe that this would make a reasonable Critical Quality Attribute (CQA) ... would you accept THOSE VARIABLES as good evidence / indicators? ... Do we all agree that measuring the CMC and CQA is a good proxy / indicator for drug quality and performance?"
FDA : "yes that is reasonable. We agree that those variables / data ARE a good yard stick for performance / evidence etc etc"
MSB : "Phew ... ok great ... so if we bring that new data and combine it with our previous trial, we can re-submit the BLA and, since you've already indicated that you liked the previous trial and NOW you're saying that you agree with these new 'yard-sticks' we can safely assume that you'll judge the performance of the trial (which was good) in the context of the new variables?"
NOW! .... here's where there are two potential options (there are probably seventeen options but let's just keep it to two for now).
The FDA can answer in two ways.
1. FDA : "Yes, exactly ... we're happy with the previous Phase 3 trial and the data and so forth. We just had concerns about whether or not we truly understood WHY you got those results, HOW we might predict future drug quality, and whether the quality measurement (whether high quality or low quality) IS IN FACT, a good indicator of whether the drug will perform, in the real world, as the medical world folds it into their standard of care. We just need to add a bunch of CMC and CQA data to it, and look at the entire picture with fresh eyes to establish whether or not the RESULTS from that trial can be attributed to those new variables"
MSB : "Phew! ... ok perfect! ... we can definitely do that! We've already started in fact ... we've got all the protocols and measurements and lab equipment all set up ... we've already got engineers working on how to randomly select samples from the production facility and test for quality. We've already got engineers working on how to tune and maintain the measurement machines so that they're calibrated properly ... We can get that data in a few months ... Let's re-schedule another meeting for July, or whenever (the data you're requesting will definitely be done by then) so that we can present this new data you're asking for and describe HOW it folds into and explains the previous Phase 3 trial results"
FDA : "yep, sounds good ... if you're confident you can get that data by July ... then, yes, we can meet then"
OR ....
2. FDA : "WHAT?! NO!? ... You can't just staple new data to old data! .... we're saying that now that we've ALL AGREED on the new 'yard-stick' variables (and we've previously agreed, years ago, on the phase 3 trial design parameters so that's all fine and good), you need to REPEAT the 3rd trial WITH these new CMC and QCA data / variables integrated into it and come back to us with the results ... but don't worry - we're pretty confident that it will all work ... we just need to make the case rock solid"
MSB : "Bugger!"
Now here's my question to the forum ..... Suppose the FDAs response was option #1 ...
If it HAD BEEN option #1, do you really think we would all be here reading and re-reading the ASX statement that MSB wrote and trying to work out what EXACTLY the future steps are? ... If I had received such a response from the FDA, I would write an announcement that spells out EXACTLY what transpired, EXACTLY what was agreed to and EXACTLY what steps I'm taking to meet the objectives agreed to.
NOW, this is NOT evidence of either option being correct.... In fact - let me be very clear on what I AM saying.
I am saying that IF option #1 (or something SIMILAR to that - I'm not trying to predict with perfect accuracy - I'm just trying to paint a general picture) had transpired, the announcement MSB released this morning is unforgivably vague.
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