do you think MSB absolutely must have an active comparator (concurrent control) for the adult trial? I can readily agree with you that the enrolment of RR-resistant patients makes off-label use of RYONCIL more likely on paediatric approval, but I expect it'lll be a multi-site trial anyway given it's BMT CTN & will have enormous recruitment interest and pull, so I was hazarding that willing trial participants would be on Rux & other SOC anyway & could simply quickly enrol when their attending physicians see them getting to that point (if they make the inclusions list) & save themselves any need for consideration of paying for off-label support. That could be done quickly if its going to be done through the same major centers as will be administering treatments on paediatric approval. I mean, for those without the money or who are unwilling to enrol, Mesoblast might even run an EAP but if MSB is going to supply product at low or no cost it would prefer to get the data out of it.
I do appreciate the value of an active comparator, but I thought that the thing to take from CBER biostatisticians giving up their efficacy objections to RYONCIL, i.e. on the 'null hypothesis' bullshit point vs indication for paediatric aGVHD, was that the FDA was 'demonstrating' even more of an interest in natural history-based controls, at least for aGVHD treatments. You know, actively embracing real world evidence instead of just arm waving and doing nothing more than talking about it (despite hypocritically approving RUX for adults for Novartis/ Incyte on just that basis i.e. REACH-1 on an open-label,single-arm, multicentre Phase 2, but lets not go there again).
All this provided they could get the rigour they justifiably want to see from other things e.g. randomisation, blinding (as far as possible) etc to reach the 'adequate and well-controlled' bar.
I could have been reading this wrong. Interested in your thoughts here.
Cheers
MSB Price at posting:
86.5¢ Sentiment: Buy Disclosure: Held
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