MSB to dispute FDA finding in Type A meeting, page-26

If you look at this situation from both a scientific & commercial perspective, a fundamental question of the FDA is this:

Can you prove that every patient gets a uniform dose and that the dose is equally effective?

Now from one side you could say that if 70% of patients live when only 15% did previously, does it matter? More people (kids in this case) when there is no real alternative.

However, consider this:

What if the 30% who died did so because the dose was not as effective as it was meant to be?
Some patients would then have had a reduced chance of survival and them, their families or their insurers have paid for a treatment that was less effective than it otherwise may have been.

This is a pretty big question and central to the work Mesoblast now needs to do.

They either need to show the FDA how their methods of uniformity and dose are appropriate, or find a way to modify their methods to better reflect FDA custom and practise.

Note: I am a holder, I am very supportive of the company, but I am also a TGA license holder here in Australia, so do have some insight into how these regulators work.

Good luck to everyone. Lets hope updates aren't too far away.