You've proposed a nonsensical epistemic framework. Knowing isn't binary.
You should have a probability distribution over possibilities, based on your experience with similar drugs, expert hunches and animal trials. You then use that to estimate risks and benefits. Then compare this risk-reward profile with the risks of doing nothing -- in this case, near certain death from brain cancer.
This "can't presume to know" framework is just sophistry. And I think deep down you know that, if you had a death sentence from brain cancer you'd be begging them to let you in the trial even if they "couldn't presume to know".
Maybe the person calling the FDA "nonsensical" should be someone who has actually researched, manufactured, and sold a pharmaceutical product, not some rando on HN. People who dismiss patient safety cannot be taken seriously in my worldview - which thankfully every developed country's regulatory body shares.
You’re missing that we have treatments with known risk/benefits vs research treatment X with undefined risk and reward.
It’s extremely challenging if not impossible to obtain informed consent in this situation.
We’ve been through this before where a fancy new treatment with promising early/lab results usurped conventional therapy only to later be found inferior.
Earlier TKIs and NSCLC are a recent example that comes to mind.
It's not undefined, though. That's the point I'm getting at. There is no binary of known and unknown. A tentative picture exists based on animal trials and expert judgment. That tentative picture informs an estimation of the risk to reward profile which is then the basis for an informed decision.
This is decision making under uncertainty. It's bad practice to say that uncertainty always means "don't do it".
Its absolutely ridiculous the level of arrogance to presume you can apply some debate-bro logic and talk your way out of NOT HAVING ANY DATA. NOT HAVING ANY DATA means your product fails. End of story, thanks for playing, nice try, go back to level one and try again. NOT HAVING ANY DATA means we deny you authorization to inject our babies with untested vaccines.
You should have a probability distribution over possibilities, based on your experience with similar drugs, expert hunches and animal trials. You then use that to estimate risks and benefits. Then compare this risk-reward profile with the risks of doing nothing -- in this case, near certain death from brain cancer.
This "can't presume to know" framework is just sophistry. And I think deep down you know that, if you had a death sentence from brain cancer you'd be begging them to let you in the trial even if they "couldn't presume to know".