Sunday, August 29, 2021 1:10:40 AM
"Anavex understands how well everyone is doing as over 95% signed up for the OLE, probably similar to PDD trial which showed significant numbers in a short period of time (12 weeks)"
Anavex surely has a lot of useful information out of the ongoing OLE, and the high enrollment rate is another data point. However, an OLE is an uncontrolled look, without a placebo arm, and therefore we cannot ascribe a p value to its results. That's what the trial itself is for.
"2) for an international study of 450 participants, it is strange that the first 225 were selected from only one country; this was obviously intentional with a specific, well-guarded reason for such action"
There's no reason why this wasn't based simply on logistics and/or financing. Australians have the same biology as everyone else.
"3) we don't know if Anavex is peeking or not as Dr. M does not give any hints of what Anavex is doing, other than offering a tidbit that it might peek"
Come on. There hasn't been a peek, there is no basis to believe there has been an efficacy peek, and even your own language above supports this conclusion.
"4) many people will go beyond a point in which blarcamesine might help them to recover/improve by waiting an additional 18 months for those final numbers."
That is true, but also incomplete. Regulators are measuring outcomes against an unlimited time horizon, not just the next 18 months or so. In that context, they need to know that the drugs they approve will be effective. Cutting short the proof process means that marketed drugs may not work at all, and this would clearly be at the expense of people paying for something useless (think Aduhelm as an example of a regulatory failure) or, worse, harmful (think Thalidomide in Europe in the 1960s). They require that extra time to get the decision right, and cutting out that time carries an even heavier price to public health.
"The numbers are the numbers whether a peek occurs or not."
Actually, the numbers aren't the same numbers as time goes along. First, we have numbers on one person, then two, then three, etc. Eventually we have the numbers on the n that the company and the regulators agree will provide a trustworthy answer. Ultimately, the drug may work or the drug may not work "whether a peek occurs or not." But we won't know for sure if it works unless it passes the necessary statistical tests for efficacy and safety, and for reasons that have recently been explained here, passage of the efficacy test can be imperiled by an interim peek.
"Also, has anyone provided the name of a trial and sponsor that took a peek early, which caused its number to be non-significant at the end? If so, please re-state."
I don't know where to look for that to do your research for you, but even so it would not lead to a scientific conclusion. The premise of your request is that anecdotes are proof, or a lack of anecdotes are proof. Neither is true, either in science or in other areas, such as social policy, no matter what people have been misled to believe over the last few decades.
Anavex surely has a lot of useful information out of the ongoing OLE, and the high enrollment rate is another data point. However, an OLE is an uncontrolled look, without a placebo arm, and therefore we cannot ascribe a p value to its results. That's what the trial itself is for.
"2) for an international study of 450 participants, it is strange that the first 225 were selected from only one country; this was obviously intentional with a specific, well-guarded reason for such action"
There's no reason why this wasn't based simply on logistics and/or financing. Australians have the same biology as everyone else.
"3) we don't know if Anavex is peeking or not as Dr. M does not give any hints of what Anavex is doing, other than offering a tidbit that it might peek"
Come on. There hasn't been a peek, there is no basis to believe there has been an efficacy peek, and even your own language above supports this conclusion.
"4) many people will go beyond a point in which blarcamesine might help them to recover/improve by waiting an additional 18 months for those final numbers."
That is true, but also incomplete. Regulators are measuring outcomes against an unlimited time horizon, not just the next 18 months or so. In that context, they need to know that the drugs they approve will be effective. Cutting short the proof process means that marketed drugs may not work at all, and this would clearly be at the expense of people paying for something useless (think Aduhelm as an example of a regulatory failure) or, worse, harmful (think Thalidomide in Europe in the 1960s). They require that extra time to get the decision right, and cutting out that time carries an even heavier price to public health.
"The numbers are the numbers whether a peek occurs or not."
Actually, the numbers aren't the same numbers as time goes along. First, we have numbers on one person, then two, then three, etc. Eventually we have the numbers on the n that the company and the regulators agree will provide a trustworthy answer. Ultimately, the drug may work or the drug may not work "whether a peek occurs or not." But we won't know for sure if it works unless it passes the necessary statistical tests for efficacy and safety, and for reasons that have recently been explained here, passage of the efficacy test can be imperiled by an interim peek.
"Also, has anyone provided the name of a trial and sponsor that took a peek early, which caused its number to be non-significant at the end? If so, please re-state."
I don't know where to look for that to do your research for you, but even so it would not lead to a scientific conclusion. The premise of your request is that anecdotes are proof, or a lack of anecdotes are proof. Neither is true, either in science or in other areas, such as social policy, no matter what people have been misled to believe over the last few decades.
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