MSB to dispute FDA finding in Type A meeting, page-239

Hey, @ddwn.

My sense is that for the first ever approval for an allogeneic stem cell treatment, they want the gold standard of an RCT to be THE standard. I would imagine this is how they justify their Jakafi decision being "different" (i.e., weren't stem cells...though they were clearly VERY defensive about that decision when it was raised--they knew it was going to get thrown in their faces and were ready to deflect).

As I noted before, it's not uncommon to get the lower level bureaucrats being very conservative and hidebound.

We think it's a no brainer - it's been used plenty by Dr Kurtzberg and others, the trial design wasn't great, there were a few failures, but the ODAC saw the results, it's not going to hurt anyone, these kids have no other options, etc., etc., etc.

But they see "1st ever stem cell treatment," and "single-arm trial" and their little brains get all knotted up. So give MSB a CRL, and kick the decision up to the higher ups if the applicant wants to appeal it, making someone else responsible for approving this.

At this point, we are getting into the realms of policy decisions and agency direction. Big issues. Controlled, for now, by small minds that cannot see the forest for the trees.

Hopefully, someone higher in the bureaucracy is able to look past this "no RCT issue" (where it will come post approval for the kids, as committed to by MSB) and see that as a very reasonable and elegant solution, one that make the FDA look like they are going soft on stem cells.