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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.
Thanks Mongoose
https://www.msn.com/en-us/health/medical/alzheimers-treatment-costs-and-death-rates-are-both-rising-but-why/ar-BBKy9We?ocid=spartandhp
Yet another PR/news on the trends-costs of AD and the destruction it brings. Why would it be increasing at such a rate, even if elderly population remained relatively stable? Could it be that all the other BP crap we put in our systems are degrading our immunity and consequently normal CNS health???
FWIW I also sent this post to Anavex IR...will advise
Thanks Mongoose for your comprehensive and common sense response. I agree with every word you wrote and would add some emphasis to a few points you bring up.
1. I do agree we are caught in some kind of parallel universe comm. zone time warp made up of FDA and Govt/politics and the enormous need for REAL HONEST MEDICAL SCIENCE. Three years ago before I ever heard of A2-73 I would have defended the FDA and the BP systems established for clinical trials and scientific methods in play. Over time w/study I have begun to seriously question how the process is working and in fact am now convinced the process is seriously flawed, perhaps even criminal in areas. Having spent time as a lab rat and much time around regulatory practice/financial reality I now see the failures around AD and other CNS diseases as poorly executed, expensive, political, frauds on taxpayers and sick people. A child could ask some basic questions in retrospective analysis and conclude they should have stopped what they were doing 10-20 years ago. The good news is that entire level of thinking and statistical modeling is gone or is going away (they will just not admit it yet). We need medical/scientific facts, not statisticians and smoke. Trials will now be managed w/science and thankfully using RWE, which must be easily and credibly explained and proven.
2. AVXL is a very small company and would easily be destroyed by just one error in judgement. Timing, scientific events, PR's, political , financial, mistaken sequence...and the list goes on. The problem we have in dealing w/this environment is we have very little ammo and there are lots of targets. Dr. M. must find a communication tactic that works for him and that is effective, being silent is not a communication strategy, even though he is good at it. IMO, eventually silence becomes a bad habit and eventually it adversely impacts credibility.
3. Dr. M. must capture and present the context we are in more effectively. He can do this w/o betting the farm by being open about our A2-73 testing strategies and methods. And he must do that right away while all the competition are still down in their bunkers. The FDA and BP are clearly scared shatless about making a wrong move right now on ANYTHING . Musashi was a great warrior his famous poem on the .."The Pen and the Sword , in ACCORD" , has never been more meaningful than right now in this space. It is time to ACT with superior tactics which IMO will require generous use of RWE and precision medical science. One strong case made w/the FDA will save them from continued humiliation and will rewrite the future on CNS medical science. IMO, AVXL is capable of showing the way.
IMO, the window will close if he does not act soon.
Mongoose...thanks for your positive message. Many of us here have similar thoughts on the promise of legitimate CNS clinical work and our planned trials. Would you please expand your comments/insights on trials timing ,when will they really begin? What are some of the key event/metrics/pass-fail criteria you will be looking at?
There are so many reasons for wanting A2-73 to be an effective CNS spectrum treatment. Certainly, helping these dear little kids has to be at the top of the list. We wish them all the best.
If Kund turned optimistic on AVXL it would be a miracle, but hey, stuff happens. We need a few exceptional days in a row here to get back on track...good luck
Really...and BTW, have a nice day...
Thx...I noticed that too late...link was from previous posters ihub post
Power...agree they did they not get the AD memo on Amyloid Plaques. Also, a quick review of the topics from last weeks conference in Italy says that 50%+ of the AD presenters did not get it either.
https://www.bizjournals.com/boston/news/2018/03/14/shares-of-solid-biosciences-crater-as-fda-halts.html?ana=yahoo&yptr=yahoo...
Example of what is to be a new hurdle for IND...see text near last para as "Clinical Hold". Caused this stock to drop last week.
It all fits If one considers new trials are based on precision medicine then we should expect a much higher bar for IND-NDA. So, the back and forth w/FDA mentioned for A2-73 RETT is more than likely the new way forward. IMO, We are about where we should be as it is clear the FDA are REALLY tuned in here on making this process work.BTW this new regulatory model was effective (2/18) about the time we were at threshold.
https://www.fda.gov/downloads/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ManualofPoliciesProcedures/UCM082022.pdf
Any thoughts on what this new FDA process means going forward? For example; will the new Precision Medicine protocols and guidance/requirements result in a deeper and longer look at IND and NDA research data?
both statements are true.
Thanks doc328...
https://www.fda.gov/downloads/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ManualofPoliciesProcedures/UCM082022.pdf
As new IND processes get defined based on Oncology like detailed Precision Medicine for pharma's we should expect exactly what we are seeing. Probably a good thing as compared to the hold process.
Short interesting read from FDA on history of the trials process. Yes, there have been historical instances of trials process redo's. Clearly, that is what is happening now. Includes seemingly rational, fact based reviews of what worked and what did not. I am even more convinced the recent shut downs of all BP AD trials was a top down decision. No clue on who drove it except any kind of credible retrospective review of results begs the question..."What the hell took you so long??"
https://www.fda.gov/aboutfda/whatwedo/history/FOrgsHistory/evolvingpowers/ucm304485.htm
https://finance.yahoo.com/news/why-multiple-sclerosis-promising-risky-151203400.html....FDA tightening NDA rules on MS IND ?
See comments on new labeling applied after AE reports and note last para or so when they kicked back a new MS IND.
Bob Dylan ahda song about this...."The times they are A..Changing"...
Someone needs a hug...please continue with your very welcome contributions here
https://finance.yahoo.com/news/biogen-present-2018-advances-alzheimer-120000313.html
More evidence that BIIB did not get the memo.
Great information and inspirational message.
If in fact BIIB is doing more pre-clinical studies then AVXL must be in the loop. This may also be a mechanism for AVXL to get research done and to obtain data/knowledge which it might otherwise be unable to manage itself. Done correctly such an arraignment serves both parties. But, if that were the case it is risky.
[quoteThe applications, testing and restrictions are laid out in legally binding terms within an MTA.][/quote]
Agree, that seems clear enough, although no one has said that from AVXL. From a different point of view, is AVXL obligated to report any material event/results or new information from BIIB testing which might have a negative effect on sp in a typical MTA. Does any SEC rule set take precedent?
McM...