3 ms·

One reason your proposal isn't happening today might be because we don't want tons of doctors choosing to enroll in these "field" randomized trials, if they mig

One reason your proposal isn't happening today might be because we don't want tons of doctors choosing to enroll in these "field" randomized trials, if they might be experimentally treating people with drugs that aren't safe. We want to know about safety first before unleashing on the world, even in a randomized trial context. And since the FDA bundles verification of safety and efficacy together, so we can't do your proposal without unbundling them. Now, imagine that after safety is verified to a 1 or 2-star level, we don't make the drug approved for general use, but doctors can freely enroll in randomized trials as you proposed? That way we're getting the efficacy data quickly but safely. Maybe a minor point, but we do have to unbundle safety from efficacy in some sense to get off the ground here. The question is, what exactly is allowed to happen after safety is established but efficacy is not yet established?

Brakenshire 5y ago
I agree, that's well put and I think that could work very well. I think the key would be having clear centralized standards for how the trials are conducted, or you could have doctors skirting the system and just using it as de-facto prescription, and patients being put at risk for ineffective treatment without producing any valid evidence.
- civilized 5y ago
  Yeah, if the existing system is built on trust of doctors who run clinical trials, we have to ask whether that trust scales. If the system were set up so that doctors would have a lot to lose if they didn't follow the system, trust could work here. We already trust doctors as professionals on many other things...
ezconnect 5y ago
His proposal is to treat a new disease with approved drug you can buy and not experimental drugs. This will give us more data on therapies that might work and we can do more detailed studies on therapies with high percentage of recoveries.

Brakenshire 5y ago

I agree, that's well put and I think that could work very well. I think the key would be having clear centralized standards for how the trials are conducted, or you could have doctors skirting the system and just using it as de-facto prescription, and patients being put at risk for ineffective treatment without producing any valid evidence.

civilized 5y ago
Yeah, if the existing system is built on trust of doctors who run clinical trials, we have to ask whether that trust scales. If the system were set up so that doctors would have a lot to lose if they didn't follow the system, trust could work here. We already trust doctors as professionals on many other things...

ezconnect 5y ago

His proposal is to treat a new disease with approved drug you can buy and not experimental drugs. This will give us more data on therapies that might work and we can do more detailed studies on therapies with high percentage of recoveries.