Celebration Thread! Biogen is going to the FDA with Aducan.
Posted: Fri Oct 25, 2019 3:27 pm
A futile futility analysis?
Are they kidding?
What if the futility analysis wasn't futile after all?
For me, I have to draw the line-- that would be too nuts.
Life can only be so weird, and not more.
I'm going to stick with the futile futility analysis interpretation.
Well we'll take a win no matter how the billiard ball wound up in the right pocket.
This is amazing!
Where is everyone on this?
As a forum elder, I cannot remember any AD news being this big over at least the last 5 years; actually I am unable to remember any AD news that has ever been this promising.
After being a member of the dementia underclass for the last 10,000 years, I wake up and find that I have been promoted to the upper class? Not bad.
I think this calls for and deserves some celebration.
Of course, it is not unreasonable to expect that the 3 thousand + patients in the phase 3 trials and those who had started the EVOLVE phase2, and those on the phase 1b extension will be able to provide more clinically important cognitive data once they get back on site. If it takes a while for the amyloid to clear, then might not the last 7 months have given those who received active treatment a chance to make further cognitive gains relative to those who were on placebo? It is still something of a mystery as to why they have taken 7 months to finally disclose this. The news reports appear to indicate the analysis team had concerns within weeks after the futility analysis was announced. Wouldn't it have been better to then use the precautionary principle and relaunch the trial? The online data at 78 weeks for the Emerge trial look fairly strong, why would it have been necessary for both to have demonstrated efficacy at this stage?
My hunch would be that this treasure trove of clinical information that will be gleaned when the patients return to the clinical trial sites will definitively answer the question of efficacy, so that there would be no lurking uncertainty. However, that would have to be one of those higher levels calls as at least for this round once you would have to pre-specify the outcome and the ball would really have to drop in the right pocket (perhaps after a little hesitation on the rim). I hope that is the game plan that the FDA has in mind, though it might take a little bit longer than a month or two to extract all of this important information from the cancelled trials.
For those on the forum, this is enormous news. If there is an actual product with efficacy then a self-sustaining Alzheimer research economy can emerge. It is so difficult when a problem is seen as an endless money pit with no clear endpoint. Unending exhortations for yet more money with little actual evidence of progress eventually wears down everyone. If we finally have something in which the financial resources of the dementia community can flow into, then a self-sustaining wealth base could emerge that will progressively move the research agenda forward.
The market potential is obviously large and one would have to think that rolling back treatment to those with MMSE equivalent to ~40 would be the next step as this would increase the potential customer base enormously while also likely reducing the risks associated with maxing out dosage as this is already known to cause ARIA especially in epsilon 4s. Hopefully this roll back will begin sooner instead of later as we have all seen how years can become decades with clinical research, only to find that the futility analysis or something was incorrect.
Very very pleased with this news.
Clearly it is somewhat premature to party, but would it really be sinful to celebrate without definitive evidence?
I seem to recollect other instances on this forum in which we have celebrated results which might best be described as
wishful thinking. The results from Biogen are clearly much more than that.
Are they kidding?
What if the futility analysis wasn't futile after all?
For me, I have to draw the line-- that would be too nuts.
Life can only be so weird, and not more.
I'm going to stick with the futile futility analysis interpretation.
Well we'll take a win no matter how the billiard ball wound up in the right pocket.
This is amazing!
Where is everyone on this?
As a forum elder, I cannot remember any AD news being this big over at least the last 5 years; actually I am unable to remember any AD news that has ever been this promising.
After being a member of the dementia underclass for the last 10,000 years, I wake up and find that I have been promoted to the upper class? Not bad.
I think this calls for and deserves some celebration.
Of course, it is not unreasonable to expect that the 3 thousand + patients in the phase 3 trials and those who had started the EVOLVE phase2, and those on the phase 1b extension will be able to provide more clinically important cognitive data once they get back on site. If it takes a while for the amyloid to clear, then might not the last 7 months have given those who received active treatment a chance to make further cognitive gains relative to those who were on placebo? It is still something of a mystery as to why they have taken 7 months to finally disclose this. The news reports appear to indicate the analysis team had concerns within weeks after the futility analysis was announced. Wouldn't it have been better to then use the precautionary principle and relaunch the trial? The online data at 78 weeks for the Emerge trial look fairly strong, why would it have been necessary for both to have demonstrated efficacy at this stage?
My hunch would be that this treasure trove of clinical information that will be gleaned when the patients return to the clinical trial sites will definitively answer the question of efficacy, so that there would be no lurking uncertainty. However, that would have to be one of those higher levels calls as at least for this round once you would have to pre-specify the outcome and the ball would really have to drop in the right pocket (perhaps after a little hesitation on the rim). I hope that is the game plan that the FDA has in mind, though it might take a little bit longer than a month or two to extract all of this important information from the cancelled trials.
For those on the forum, this is enormous news. If there is an actual product with efficacy then a self-sustaining Alzheimer research economy can emerge. It is so difficult when a problem is seen as an endless money pit with no clear endpoint. Unending exhortations for yet more money with little actual evidence of progress eventually wears down everyone. If we finally have something in which the financial resources of the dementia community can flow into, then a self-sustaining wealth base could emerge that will progressively move the research agenda forward.
The market potential is obviously large and one would have to think that rolling back treatment to those with MMSE equivalent to ~40 would be the next step as this would increase the potential customer base enormously while also likely reducing the risks associated with maxing out dosage as this is already known to cause ARIA especially in epsilon 4s. Hopefully this roll back will begin sooner instead of later as we have all seen how years can become decades with clinical research, only to find that the futility analysis or something was incorrect.
Very very pleased with this news.
Clearly it is somewhat premature to party, but would it really be sinful to celebrate without definitive evidence?
I seem to recollect other instances on this forum in which we have celebrated results which might best be described as
wishful thinking. The results from Biogen are clearly much more than that.