" In my opinion the Trial network attended the meeting to ask the FDA why this therapy has not been approved . They have used it and believe it to be effective. "
I think they were also proposing a win/win solution to the (apparent) quandary.
OK, I'm not sure how strongly this point would have been made - a more 'palatable' way to get the result that was needed might have been for Dr Levine, say, to point out to CBER personnel (incl. Dr Verdun) that the BMT CTN had resolved to fund and commence a much-needed SR- / RR-aGVHD trial in 2024 but, given the ODAC result, BMT CTN want to know before they lift a finger that FDA will accept as suitable MSB's newly improved potency assay (as now validated against Lonza Biologics manufacturing processes) for the qualification of the existing product intended to be used in the proposed trial. FDA gets its adult trial. BMT CTN gets to share the data. FDA gets compliance with its real-world registry data standards by the best in the USA....all FDA has to do is put the horse before the cart.
I agree with the other comments re the meeting & the importance of Dr Verdun's attendance per your post, as we've posted several times previously.
Regarding the presence of Dr Verdun, my view is that her willingness to get out of her office and attend in person, & see & hear how FDA reps acquit themselves, will also assist in managing the FDA biostatisticians better this time - especially M. Klinker's conflicted involvement (nb: he's been Chief, Cell Therapy Branch 2 at OTP since August, 2023).
But I also take a lot of heart from her personal bio, especially the fact that she's a Board-certified paediatric haematologist/ oncologist, who did her residency at a Chicago Children's Hospital and then followed that 3 years up with another 5 years as an attending physician & then haematology/ oncology fellow at the Children's Hospital of Pennsylvania.
Readers on HC: MSB also may not be aware that, despite her key administrative appointment as Director of CBER's OTP Super Office, she somehow manages to maintain her professional practise commitment and looks after kids as an Attending Physician at the Children's National Hospital in Washington DC, #5 children's hospital in the USA, which she's been doing apparently for the past 13 years in DC.
IMO this woman is one of the best persons, if not the best available, as Super Office Director for OTP that Mesoblast Ltd could have ever hoped to see in the chair when it comes to deciding on CBER's recommendations on RYONCIL for paediatric SR-aGVHD i.e. with a genuine and competent appreciation of the plight of paediatric oncology patients in the USA.
Oh, and did I mention that she did her undergrad at Duke University, & lists Duke U on her interests page. Those Duke connections...
All good IMO.
Cheers
GLTAH
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