Basza, I appreciate the well thought out response. Honestly appreciate it, and this is why I post, to have a discussion with someone who not only believes in the product but also has done research, has thought their position through and is willing to engage.
There's a reason that placebo controlled studies are gold standard. And they always will be gold standard. However, you are right, they aren't always ethically appropriate and often the next best thing must be done.
That being said, when you aren't able to do the gold standard, you have to be ready to accept the flaws in your method, and the risks of bias or error in the results.
This means the results may not be able to be replicated. They may not be true results. That's the risk, and it may mean that people may be more critical or skeptical of the results.
It's also important to look at the studies, rather than just the numbers, as it's difficult to interpret the significance of the data otherwise.
For example, looking at the numbers, the survival rate of children in the high risk biomarker subgroup of 64% vs 10% looks amazing! Why aren't we scrambling for Remestemcell? Why is the FDA dragging their heels?
The key is twofold:
1) the control group is taken from another trial, matched by age and disease and similar poor biomarkers. This was done due to the ethics of performing a proper RCT, but is still at higher risk of bias or confounding factors (were there other reasons the children in this group had such poor outcomes, other than the treatments?)
2) The absolute numbers looked at in the study were so small, the treatment arm and the control arm only had 12 and 10 patients respectively. As we saw with the original Covid trial, even a hugely significant mortality benefit seen in such a small cohort may simply be due to chance. They had significant P values, but with such small cohorts you are unable to draw firm conclusions at this stage.
Science is complex, but I've spent a lot of time analysing research in my old job and during my various degrees, and I've become very skeptical, which isn't a bad thing when investing.
As I've always said there may be something here but my goodness I would not bet the farm on it at this stage.
MSB Price at posting:
99.5¢ Sentiment: None Disclosure: Not Held
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