Thanks for your response.
I do admire your vigourous support of the company, and the research.
Still, I am not convinced.I am uncertain.
Coming back to my post, I was clear to ask questions rather than make a definitive statement and was asking for thoughts , because much of the data, and the interpretation is not definitive, despite your analysis. The study design had perspecified endpoints, and opinion cannot change the design, nor results.
To the point:
I said “While There are many arguments to be made to explain the placebo response, as it stands, the primary endpoint of the pivotal, placebo controlled study was not met.
Failure to not meet a primary endpoint, in evidence-based medicine, and subsequently of secondary endpoints using rules of multiplicity, indicates lack of efficacy
We can debate the merits of the “placebo” and other study design conundrums, but as it stands a reviewer examining the data would be left with this conclusion, notwithstanding the lack of other treatments, and the low risk.”
I didn’t get back into rehashing the study, but I did put placebo in quotation marks, because I thought that it was self evident the argument for the possible effect, and mislabelling of placebo. In fact, was it not called a sham? Why would you call an active treatment a sham, If you believe it’s effective.
In pharmaceutical trials, they will label it a minimally effective dose, but never a sham if they believe it has efficacy.
The point I am making is, what would a reviewer be asking based on the data they have, which is the basis for the delay?
None of us know, unless one of us works for the company,FDA and has seen these documents.
We are all speculating.
Your discussion for justification of the failure to meet primary endpoint, is interesting and somewhat convincing, but it does not change the study results that it failed the primary endpoint
That is factual, and not influenced by opinion.(hence my use of the word conundrum)
You mention:Management has been clear in their press release that the questions the FDA asked would not require the additional studies. “We believe we have the data and information to address FDA’s questions”.
They may believe, and clearly do, that the study is sufficient and makes the points that you say. That does not mean the FDA agrees.
The FDA has a long history of requiring more information, or more studies, not withstanding another regulatory bodies approval. Not all drugs and devices get approved similarly, between countries.
I hope that you are correct, because that would be the quickest path to approval.
In the end, not much use debating this more, we will have to see what happens.
GLTA