NorthWest Biotherapeutics Inc (NWBO)

[Pyrrhonian]

I'm sorry but I saw no "regulation." My mistake was not whether there need be a randomized control group or not, but in saying "active comparator" which necessitates they are taking SOC. But it should have been obvious what I meant. A control group that only gets a placebo can't be called an "active comparator" but is still a randomized control. But either way the fact remains, without a randomized control group enrolled in the same trial with the same criteria as experimental tx group, PFS and OS data cannot be relied upon for regulatory approval. That simple.

Btw the "dramatic enough" statement is so obviously talking about ORR.. You have to put things said in context. The appropriate context for what that official said is EVERYTHING ELSE written in FDA guidance, as well as the plethora of evidence of examples in modern clinical trial design.

If NWBO wants to use SD for approval (which I've never seen) or more appropriately PFS or OS, then they must enroll a control group along side these that either is monitored with no tx given (hard to get patients to agree to this) or better yet (for data validity sake) give them a placebo, and have tx assignments blinded. Bingo, you've got a study whose results can now be used to petition for approval.

If you want to seek approval with a single arm study (like Opdivo and Keytruda recently did) you absolutely must have ORR. That means by RECIST criteria you must show partial responses (PR). Heck, don't even show a CR, but you must show some PRs. It looks like a mere 15% might even get it through, as amazing as that is.

But the Direct Ph I is obviously seeing none at this point. They may have a shot in Ph II. But if not, the only thing they can do to get approval from there is enroll a new trial with a randomized control group and compare survival data (PFS or OS).