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Back in the day we would technically define this as a, "cluster".
T-38...thx again for all you have done and for your over the horizon insights.
We must appreciate the scope of the AVXL task. We are presented with either one massive binary event (a true OMG moment) leading to effective treatments or an old school BP/WS CFIT. All the best.
Doc...thanks for grounding all of us in the reality of dealing w/regulatory bodies during times of change. Can you say ,in this context, how the new "precision Medicine" trials rules are being developed/applied. Are these guidance or are they requirements being applied to the first trials and how is RWE being treated? If new learning occurs which change the trial plans, do they stop/go back and restart or evolve as they go? TIA
How does an enterprise establish a IND protocol with these process controls in place?
[quote“As clinical trials become more virtual, sponsors will be able to ‘dial in’ what capabilities they want virtualized,” he said. “This new Apple feature will enable new capabilities that were not possible before.”]
The reported conversation with IR on IND status is very encouraging news. Some of us have suggested a scenario similar to the one described by IR. A study of what has been published by the FDA on new IND-Precision Medicine protocols also indicates we are doing what was expected working with the FDA. We could not predict how much time would be required to do the back-forth planning/critique but months(not weeks) are to be expected. Anyone with any FDA interface experience can see what is being done. Extreme caution must be exercised at the trials expectation/RWE/PR-news interface. The work must be done well and it must be reproducible. Expense and time must be forecasted while day to day conflicts are resolved. Conflicts galore.
Go look at what the FDA has already released on the topic (we have posted the links here a while back with discussion). The FDA has established a structure/framework of the trial design with required checkpoint with emphasis on what not how and RWE-patient interface-takeaways. It all reads to me like they expect tp rely heavily on pts/RWE and being as flexible as possible in interpretation. Nothing at all like conventional FDA trials. What would be unknown at this point is what clinical/pts evaluations must be done, at what point in the protocol, what will/must be measured and when, what pass/fail acceptance criteria (if any) will be used and when applied.
We must keep in mind that these CNS diseases have NEVER been effectively treated. Even though pre trial information establishes basic expectations no one has ever been here before. Not only do we need results to be precise and meaningful but we better not make an error b/c the entire pharma world has just had it's collective ass handed to it. We can intuit that something very important will eventually come from this molecules testing. Let's hope it is soon but sadly we have no way of knowing when or what. I do believe that some very exciting science is about to demonstrated.
habit...thx...somewhat encouraging to hear.
Schwab Yes, proxy mtl received about a week ago.
Thoughts on next few weeks of events (planned or likely) and what will follow? This has been a very quiet period, even by AVXL stds. TIA.
REALLY??...is that what precision means?
http://anavex.com/category/anavex-2-73-publications/
Sometimes reviewing history can be helpful.
Xena..cannot do PM. Would expect Si3N4 -silicon nitride body of knowledge to be extensive (semiconductor guys have been all over the physics for decades) compared to CNS. Should be much lower risk than AVXL. Looks very solid.
Xena...
Biostock..."
All we need is a solid-fact based set of objective evidence that the science has been validated again in some CNS context. Substantial but no need to be massive(would be great but), I'll go w/substantial and not a PR Puff piece only.
I did send another e-mail and V-mail just in case.
He is the logical compliment.
Whip...Thx for great find w/links...good stuff
https://www.nbcnews.com/health/health-news/scientists-say-they-ve-discovered-unknown-human-organ-could-help-n860601
Any thoughts from techs on what this new organ discovery means for AVXL going forward...looks like a reset in many areas or at least a rethink. TIA
Tradeherpete
Agree, that is what most of it looks like. Still looking for any new/innovative AD studies or PII trail plans being started since the recent BP stand downs.
We need to be aware of the time bubble which most biotech-pharma labs/development organizations live in. H/W medical device people(part 820) have H/W regression curves/methods which can be used to safely accelerate failures, for example. They are able to accelerate H/W failures. The bio clock variable is experiential, some may have clocks that run synched with the rest of us but often bio clocks are not synched. Consequently they might have 3 yrs to wait for results in the lab while the rest of us decompose in 1 year. All of this gets completely screwed up when someone stops the train and requires a restart(Precision medicine for example). AVXL may be slower than even the normal clocks due to the nature of CNS diseases. Hopefully, RETT will soon accelerate the clock somewhat. It is all very frustrating as we await evidence-results. IMO, the clocks will probably be reset/synched up when we start to see PII timelines/results. I have seen nothing that predicts how much time it will take to demonstrate efficacy or if the normal clocks will even matter much. Many of us expect quick feedback though.
Talon..thanks for the excellent observation here..
https://www.cnbc.com/2018/02/15/cnbc-announces-speaker-lineup-for-healthy-returns1.html
My opinion is(with the exception of Gottlieb) This would be the gene pool of geniuses that controlled/watched/approved and peered the last few decades of worthless misleading AD and other CNS trials. Do you suppose anyone will bring up the complete failures they have sponsored/witnessed while they splain to CNS patients..."HEY, we got this."
All that brilliance in one room, I am certainly impressed.
As the process for FDA IND treatment of A2-73 in the RETT trial continues to hold our attention there are a couple of things worth considering. Just what is the formal FDA process guidance for what we expect to happen, how formal is it and is there anything about the FDA history we need to understand. How did we get here and what's next? So these are a few of the questions I had as I went poking around. Maybe others here will get more than I came up with. Last week when a pharma product was put on clinical hold (killed the stock for few days). "clinical hold" sounded pretty serious for and IND, could not help but wonder since we have not heard theses terms used often, if at all. Was this a consequence of the new rules being implemented? Are we raising the bar?
See FDA MAPP 6030.1 rev 3-(Manual of Policies and Procedures-effective date 2/20/18) also see MAPP 5210.5 Rev 2-effective date 10/25/16. It is easy to become an FDA guidance document junkie b/c there is just so much of it. You will see these are typically very high level (abstract) procedures which makes everyone nervous when the FDA starts their interpretation dance and we all just listen. Remember this is all new turf. For the most part they are common sense documents which outline the fundamentals with good/basic process steps. You will not see a lot of specifics on HOW but lots on WHAT they want done.They then will judge how well you followed the what, and debate the how, etc.
I am nervous b/c we need fast and effective systems in this NPI-IND space, not a bunch of hand wringers. Since we anticipate the new "Precision Medicine" protocols will require a stronger PII protocol than might have been done in past days. This should lay the path for smaller, more precise PII trials with far fewer failures. It all makes sense to me in a PAY ME NOW OR PAY ME LATER environment. BTW, the old way cost a lot, sucked up intellectual enormous resources, emphasized the wrong science and, in the case of CNS diseases, has been a massive failure. We all know that, good riddance.
So, NOW WHAT? I thought that if I went back and studied the history of FDA guidance docs and process controls I would be able to see a set of (BEFORE-AFTER Precision Medicine guidance changes) . NOPE, so far I have not seen such although it is still a good idea. Comparing old to new FDA guidance docs should tell us what has changed and therefore what to include in any new protocol. I invite anyone to try and do the same exercise as a kind of reverse engineering of FDA changes. Good Luck. See T-38's comments on Precision Weapons, this is not a guessing game like the old days. PII-IND is the new acceptance trial base-IMO. The street will not like this-IMO.
I do expect AVXL will begin RETT and AD trials but I also expect to not apply any of the previous life experiences to what happens going forward. You can figure out for yourself what this means for you (trader/short/other).This applies both to timelines and content. I cannot but wonder how the WS/BP world is going to be impacted by the new protocols. On the positive side, we should see more BTD declarations. I am looking forward to little RETT girls being well and to Medical OTC products having fewer marketing-warning labels and some ceremonies being held to close dementia recovery homes/wards as patients go home. Best to all. What style am I you ask? Shotokan.
F1ash
Xena, thx but did not yet watch video but am not surprised BP has to change the process of discovery/development. You ID'd it correctly IMO.
Old methods required all to lock in the process before you leave the room and "just run till it breaks or you win". No one in private industry would ever sign up to develop a product with those rules. It is idiotic, no wonder they never got anywhere. ONLY BP could run using the old rules for all the reasons we discussed before. BTW, that is not a good learning technique for all the obvious reasons but the rules guys and statisticians controlled the thinking.
In fact such rules would always result in what we got. Large overhead/spending, over the cliff time line/constraints, no adjustments as you proceed based on any newly acquired knowledge. You are not allowed to apply new learnings unless you stop the trial and go back, redesign the trial, get approval, start all over again with a new budget. Under previous guidelines/rules it is no wonder the development process got the results it did and that people were forced to just EAT THE RESULTS, just figure out how to make it work. That is how you end up with brain swelling/bleeding new product solutions being the best we can do.
And, keep doing the same process b/c the rules say you have to. WTF were they thinking would happen?
Finally, some kind of common sense or real world (call it reality) stepped in. Maybe Dr.M. was one of those who wanted to go a different path. Dr. G. saw an opportunity to reset the trials process. "I Know let's call it Precision Medicine". Anyone who has been around these parts for a while will tell you that BUT, such changes do not happen at the speed of light. THAT, IMO is the issue they/we face. Now, it's gonna be ALL ABOUT TIME. Is it OK to start/stop a trial? Apply what you just learned and RESTART? How do you do that when the herd has been trained to run in a straight line until they go over the cliff? (SEE RWE)
We'll see I guess. IMO, that is part of the genius of selecting the RETT trial and possibly why we cannot get a straight answer. New trials will/may allow application of newly acquired knowledge on the run (how do you do that and remain in control?).IN my opinion that is what RWE will be/is all about. The answer is, we do that now every day everywhere else on the planet, now we have to get the FDA and others to do the same w/o developing chaos. That is why god gave us training wheels.
Rett has 2 things at least going on. First we want to/must demonstrate and validate CNS Cellular Homeostasis thesis. What it means in this context and clinical applications. Second, create and apply meaningful process control rules that can be applied to the new structure with flexibility. Like changing a tire while the car is rolling. NP.