How about an AA for Adult SR aGvHD - at first sign of promise- how many hundreds are alive because of access via EAP?
Safety box is ticked. Manufacturing and Potency assays ticked.
I'm thinking this gets approved first via the new pathway they keep mentioning.
Tell me why a RUX refractory SR a GvHD adult should be denied access to Ryoncil ?
There is enough CIBMTR data to sink a ship- Was it 73% survival in the adult EAP ??
There is an unmet need in Grade 3/4 Adult Ruxolitinib refractory patients- that group is rare. Well not for long......
Newly installed at the agency, Vinay Prasad has committed to giving rare disease patients rapid access to “products that are even small steps forward” during his time as director of the FDA’s Center for Biologics Evaluation and Research.
“We will take action at the first sign of promise for rare diseases. We’re not going to wait,” Prasad said.
I'm aware that Vinay Prasad is talking about rare diseases- but the philosophy and direction points to upheaval of standard practices.
Market for adults is triple kids.
All above is pure conjecture, but it's mighty quiet out there, I wonder why?
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