The anecdotal evidence is what tells you that a therapeutic works and this one has been used to positive effect in many worst case situations.
These cases are where the rubber hits the road and show that when the disease progression is far enough along, the difference in the live or die situations become more pronounced.
This is where there is no ambiguity to muddy results.
for CD10 the argument was.." These people would have gotten better on their own". Except when they don't. Over 400,000 people would have a difference of opinion, if they were alive to voice it.