5 ms·

We do have to make drug discovery vastly more efficient, but we want to make it more efficient at making useful drugs available, not just making drugs available

We do have to make drug discovery vastly more efficient, but we want to make it more efficient at making useful drugs available, not just making drugs available. If you decouple availability from effectiveness then drug companies will just market unproven drugs to people who are desperate or who don't know any better. Or even if the drug companies do nothing people will just start taking drugs on the basis of recommendations from Facebook. Maybe that means building randomized trials into treatment in a much more regular way (like with the Covid RECOVERY trials, where any doctor could enroll patients with a web form, and source the candidate treatment from the local pharmacy), which vastly reduces cost for treatments already on the market with other indications. And perhaps altering the balance of risk for patients with life-threatening or terminal diseases.

civilized 5y ago
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.
Sanguinaire 5y ago
The problem here is letting drug companies market to the uneducated general public - there is a good reason why that practice is banned in most countries.
- irrational 5y ago
  Is it banned when it isn’t the drug companies doing the marketing to the uneducated general public? It wasn’t the drug companies telling uneducated people to use horse dewormer to combat Covid. That seems just as dangerous (probably more so) as drug companies making claims to the general public.
- wnissen 5y ago
  Banned in every country, except the U.S. and New Zealand.
themacguffinman 5y ago
> If you decouple availability from effectiveness then drug companies will just market unproven drugs to people who are desperate or who don't know any better. Or even if the drug companies do nothing people will just start taking drugs on the basis of recommendations from Facebook. But this already happens for certain treatments. The FDA already regulates a separate category with loose efficacy requirements for dietary supplements and herbal remedies. These medically irrelevant treatments are immensely popular, they're just forbidden from marketing specific medical claims and being actually toxic. People still end up taking FDA approved treatments like Ivermectin for non-FDA-approved purposes based on Facebook recommendations. Why do we always have to make people's choices for them? They're grown adults capable of making their own decisions and their own mistakes. If they want to ignore the science and the credibility of the FDA process, they should be free to do so.
- Brakenshire 5y ago
  One big reason is you're removing the economic incentive to produce good evidence. The herbal remedies industry is a perfect example, there will be valid treatments there but yet the industry is almost entirely medically useless. Some of the treatments might be effective for particular diseases, at particular doses but no one ever knows. These companies have billions in revenue and are vastly profitable but they never look for evidence that treatments work because people buy the remedies anyway, and a company that spent the money to run the trials would be undercut by less scrupulous competition. This is not even a market with asymmetry of knowledge, it's a market where no one knows anything, and the lack of regulation makes sure it continues that way.
  - themacguffinman 5y ago
    There's still an economic incentive for the highest level of FDA approval: Medicare and insurance companies have to cover it and doctors can prescribe it. That ensures a high and consistent level of usage across the whole country, not just among people with more money than sense (usually IMHO; it's possible that they're just smarter & savvier than the notoriously conservative FDA, who knows, this is a big reason why taking away choice is bad on principle). I don't think the lack of credible evidence of efficacy in the supplement market is for lack of trying. If an alternative medicine is actually effective, it just becomes medicine that the majority of people can trust and use.

civilized 5y ago

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.

Sanguinaire 5y ago

The problem here is letting drug companies market to the uneducated general public - there is a good reason why that practice is banned in most countries.

irrational 5y ago
Is it banned when it isn’t the drug companies doing the marketing to the uneducated general public? It wasn’t the drug companies telling uneducated people to use horse dewormer to combat Covid. That seems just as dangerous (probably more so) as drug companies making claims to the general public.
wnissen 5y ago
Banned in every country, except the U.S. and New Zealand.

themacguffinman 5y ago

> If you decouple availability from effectiveness then drug companies will just market unproven drugs to people who are desperate or who don't know any better. Or even if the drug companies do nothing people will just start taking drugs on the basis of recommendations from Facebook. But this already happens for certain treatments. The FDA already regulates a separate category with loose efficacy requirements for dietary supplements and herbal remedies. These medically irrelevant treatments are immensely popular, they're just forbidden from marketing specific medical claims and being actually toxic. People still end up taking FDA approved treatments like Ivermectin for non-FDA-approved purposes based on Facebook recommendations. Why do we always have to make people's choices for them? They're grown adults capable of making their own decisions and their own mistakes. If they want to ignore the science and the credibility of the FDA process, they should be free to do so.

Brakenshire 5y ago
One big reason is you're removing the economic incentive to produce good evidence. The herbal remedies industry is a perfect example, there will be valid treatments there but yet the industry is almost entirely medically useless. Some of the treatments might be effective for particular diseases, at particular doses but no one ever knows. These companies have billions in revenue and are vastly profitable but they never look for evidence that treatments work because people buy the remedies anyway, and a company that spent the money to run the trials would be undercut by less scrupulous competition. This is not even a market with asymmetry of knowledge, it's a market where no one knows anything, and the lack of regulation makes sure it continues that way.
- themacguffinman 5y ago
  There's still an economic incentive for the highest level of FDA approval: Medicare and insurance companies have to cover it and doctors can prescribe it. That ensures a high and consistent level of usage across the whole country, not just among people with more money than sense (usually IMHO; it's possible that they're just smarter & savvier than the notoriously conservative FDA, who knows, this is a big reason why taking away choice is bad on principle). I don't think the lack of credible evidence of efficacy in the supplement market is for lack of trying. If an alternative medicine is actually effective, it just becomes medicine that the majority of people can trust and use.