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.
