Being that they have approved drugs in the last 5 years that had little to no efficacy or that were dangerous (Aduhelm, Remdesivir, the J&J covid vaccine, and the AstraZeneca covid vaccine, all of which have been pulled from market or found to not work) I would say they don't really need to prove anything to approve drugs as long as they're from a company that pays the FDA bills.
But to your hypothetical, yes, it is possible they may want to prove LL's effect alone. Because the n=5 is so small they need confirmation on a bigger scale and the problem in this scenario is that 5 years ago the trial was for a different kind of cancer. mTNBC expresses PD-L1 on its own more often than mssCRC (which, while rare, it still does sometimes express PD-L1) so that may be a reason they wouldn't allow us to use an ICI in this trial until we've proven that LL does induce PD-L1. Patients can unenroll from the trial and get an ICI (which, if this happens the FDA should absolutely look at the outcomes and give us credit for any perceived benefit) but we would still need to run a Phase 3 trial anyway for approval and at that time the protocol would include an ICI. I'm sure all of this is what is being discussed at the FDA meeting that has either already happened or is happening soon. I trust Dr. JL and Dr. Pestell to convey this information in our best interest.
But to your hypothetical, yes, it is possible they may want to prove LL's effect alone. Because the n=5 is so small they need confirmation on a bigger scale and the problem in this scenario is that 5 years ago the trial was for a different kind of cancer. mTNBC expresses PD-L1 on its own more often than mssCRC (which, while rare, it still does sometimes express PD-L1) so that may be a reason they wouldn't allow us to use an ICI in this trial until we've proven that LL does induce PD-L1. Patients can unenroll from the trial and get an ICI (which, if this happens the FDA should absolutely look at the outcomes and give us credit for any perceived benefit) but we would still need to run a Phase 3 trial anyway for approval and at that time the protocol would include an ICI. I'm sure all of this is what is being discussed at the FDA meeting that has either already happened or is happening soon. I trust Dr. JL and Dr. Pestell to convey this information in our best interest.
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