Good point. If you go by the past, yes, historically controlled trials seems to let in questions that clouds the results. In reality I would disagree but I am not the FDA. FDA has up to now been fairly pacific, they want a well controlled study in adults for that indication. Off lable use would provent this from happening. Adults still would get funding from the insurer, we have plenty of evidence of effectiveness from the 70 odd patients already treated.
Waiting a few months seems good to me. MSB would be fairly well financed with around 100 children and 200 adults with GVHD that have no other alternatives. No rush for results in the P3 for adults. We could look at treatment failures over say 2 years as a primary. We could also look at cGVHD development. This would put us in a position to be the preferred 2 line treatment for aGVHD.
MSB Price at posting:
86.5¢ Sentiment: Buy Disclosure: Not Held
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