Unfortunately, NameHere's analysis is wrong for several reasons. I'll walk you through what happened to explain why his hopes, assumptions, and "between the lines" are not correct, and this is just a simple research failure.
Before a study starts, one of the biggest questions, and one of the hardest to answer is: how many people to enroll in the study. Recruiting, treating, and following people is the main expense of a trial, so choose a too big number, and it is too expensive, but if you end up with too few people, then your trial is underpowered (there are not enough results to determine an outcome). There is no mathematical formula which will tell you the right number of people to enroll, because this number depends on the results you see.
For example, if everyone in the treated group is cured, and no one in the placebo group is cured, then you don't need too many people, because the results are so stark. However, if 80% of the people are not cured, and some people get better in the treated group, but a few people get better in the placebo group, then you need a much larger trial in order to get a clear answer. So you don't know how many people you need until you see the results, and you don't see the results until you have finished the trial. So it requires some very human experience, skill, and common-sense.
So these researchers though they needed a 300 person clinical trial. They organized and funded a 300 person trial. However, when they got half way through, they decided to check their progress by doing an analysis of what had happened to the first 150 people. The results were not clear. Maybe too many people were not cured (in both treated and placebo groups). Maybe too many got better in the placebo group or got worse in the treated group. We will never know. However, with the data from 150 people, and comparing the treated to placebo groups, they could see that they would not get a statistically significant result, even with 300 people.
My real summery is this: After 150 people, they ran a statistical test of the results so far. They realized that the current results were unsuccessful, and furthermore, were so unsuccessful, that even if the second 150 people were better, they would not be good enough. They knew they had an unsuccessful trial, so they cut their losses, and stopped right then.
This happens all the time. I've seen it many times. The only unusual thing is that they did an analysis at the half way point. Many studies don't and get all the way to the end before they announce failure.
I think you were confused by this statement:
Press Release said:
The interim analysis was conducted by an independent statistician, with the sole purpose of re-estimating the treatment group size required to detect a statistically significant clinical effect of larazotide, utilizing patient data from the study.
The treatment group needed to be bigger because the results were so bad. They are choosing their words very carefully in order to confuse non-researchers, but the meaning is clear: the size will not work, because the clinical effect is so small/bad. They can't detect a good clinical effect in the people already in the trial.
So lets go through two of your thoughts:
My understanding is that it did not fail; instead, the company lacked the resources to actually conduct the Phase 3 trial.
No. The phase 3 study started, and even completed the efficiency phase with 150 people. At that point, statistical analysis showed that it had failed with 150 people, and would likely have failed with 300 people, so they stopped it. Of course, the company could have tried for a 450 person study, a 600 person study, etc. However, since they failed at 150, it seems dumb to continue. You could argue that if they had 3x or 4x the money, they could have continued the trial longer and longer. Sure, but that is not the real problem. They real problem was bad results from the first (pretty large) group of people.
Further, when I am reading between the lines, the message is this: "The resources needed to do the Phase 3 exceeded the potential profitability of Larazotide as a drug."
Absolutely not. There is no information on potential profitability of Larazotide. What there is, is a finding that it did not work in the first 150 people treated, and therefore was unlikely to work for the next 150 people treated. I guess you could argue that a treatment that does not work will not be profitable, which is true, but not what you meant.
Remember, in real life, in real clinical trials, and especially not in diseases like ME/CFS: not everyone is cured and not everyone gets better or worse. In real life, some get better, and some worse, and some stay the same. What you want, overall, is more people in the treated group doing better than those in the placebo group. This is the heart of statistical significance. You are comparing better, worse, the same, in two different groups. If the results are stark a small sample size will show it. If the results are small, you need a larger size. If the results are not successful, then a large sized study will still be unsuccessful.
