Anyone else buy in after todays dip/crash?, page-172

@Southoz, in many cases the FDA will not object to a single arm trial at the design stage - but it will always be subject to challenge on submission. MSB took a punt on this, and time will tell whether they are right or not (and it's a commercial punt, there is no reason other than cost why children could not be enrolled, just as they were in the earlier Osiris trial).

I've even seen one instance where the FDA pre-agreed that a particular historical control group was acceptable and that providing the drug (in this case a cell) met the pre-specified endpoints against the historical control it was approvable. But the agency changed its mind on the submission, partly because as it turned out the treated group did not sufficiently match the literature control.

So there are no guarantees, even when there are!