The recommendation for a randomised control was first made during the rolling BLA. See the ODAC briefing notes:
“FDA recommended a new randomized trial of remestemcel-L versus standard of care for treatment of steroid-refractory acute GvHD, indicating that such a study would likely be feasible in the adult population. A randomized, controlled study in the adult population could potentially also confirm clinical benefit in the pediatric population, depending on the results.’
This recommendation was then repeated in CRL1.
All FDA regulated trials are controlled. The reason the FDA want a randomised controlled trial is simple. A positive rct provides confirmatory evidence that the new “optimised” product is indeed improved and therefore the half a dozen prior failed randomised controlled trials disappear.
The FDAs position for five years has been that the next step is a randomised controlled trial. Perhaps an unfortunate consequence of the 9:1 vote was that it caused SI to think approval for children was possible not only without having to first to do an rct but without having to do any further trial at all.
Anyway, its off to the salt mine for me.
MSB Price at posting:
35.0¢ Sentiment: None Disclosure: Not Held
(20min delay)