[sofon]

> One proposal, developed by American economist Bartley Madden, is "free-to-choose medicine." Once drugs have passed Phase I trials demonstrating safety, doctors would be able to prescribe them... > > More radically, it might be possible to repeal the 1962 Kefauver-Harris amendment to the Federal Food, Drug, and Cosmetic Act, a provision that requires drug developers to prove a medication's efficacy (rather than just its safety) before it can receive FDA approval.

Personally I'd be in favour of letting seriously ill people try whatever they want. However I would not be in favour of allowing companies to charge for drugs which had not yet been proved effective.

Providing drugs for free might give companies more data to help develop (or potentially prove safety, efficacy) but generating revenue here is too open to abuse.

[jcranmer]

A lot of these kind of proposals share the same basic misconception: they assume that once a company identifies a drug candidate, then it's because it works, and the regulatory hurdles it needs to clear to be approved are just bureaucratic paperwork. But the actual evidence of the drug approval process is that said model is completely the opposite of reality. Most drug candidates--around 90%--ultimately fail. Half of all candidates advanced to Phase III (the main efficacy trial) fail. Any serious proposal needs to start from the basis that a drug probably won't work, until proven otherwise.

There is, as it turns out, an existing market segment for drugs that are safety-only-no-efficacy-required approval status. It's the herbal supplement industry, and it makes Big Pharma look like saints in comparison. There's been plenty of exposes showing that many supplement companies can't even be bothered putting the active ingredients in their products.

[candiodari]

> Any serious proposal needs to start from the basis that a drug probably won't work, until proven otherwise.

Also any proposal needs to take into account that drug trials kill something like 1-2% of patients and inflict lifelong damage on a further 5-10% of them.

Yes, even the ones "proven" to be safe (in the very short term) to use on normal healthy people.

We've had "free to choose" medicine before, and time and time again there is a screwup where 50 or so people (sometimes more) get subjected to incredible damage or killed because of a confluence of factors (pregnancy is a factor that is particularly famous for messing with drugs in a thousand different ways, as is alcohol, parasites, and certain types of bacteria. But it is very hard to know beforehand, there's even a few drugs that have different effects on different races)

[valuearb]

So we ban drugs that save thousands because they killed dozens?

[sithadmin]

>Providing drugs for free might give companies more data to help develop (or potentially prove safety, efficacy)

It's a nice thought, but you generally can't have patients and doctors opting-in for unproven treatments and expect the resulting data to stand up to any sort of scrutiny. To glean useful data, one would require a really, really large-n study population and clear impacts from use of the drug that are so pronounced they drown out nearly all other confounding factors (e.g. studies on the health impact of cigarettes) -- and expecting an unproven, last-resort type drug to attract a study population this large is just unrealistic.

[candiodari]

It's not just that experiments won't work like that, it's the consequences of experimenting on live human beings ... that is where the real problem lies. These companies don't want the freedom to experiment, they already have that. The technical way to state what they want is permission to do stage 2 trials ... and not be responsible for patient outcomes. If their liver gets reabsorbed (which is one of the more common "oops"es for drug testing), they don't want to pay for 50 years of dialysis plus constant treatments. That's what they're asking for.

[CryptoPunk]

>>It's a nice thought, but you generally can't have patients and doctors opting-in for unproven treatments and expect the resulting data to stand up to any sort of scrutiny.

It can give much earlier indications of drug efficacy, even if it doesn't provide the proof of Phase 3 compliant studies.

The history of cancer research shows how earlier human experimentation can lead to a much shorter time between a drug being discovered, and it being proven effective enough to become a part of the standard treatmemt protocol. This shorter time to incorporation of an effective drug in standard treatments means survival for hundreds of thousands of people that would have otherwise died.

That the motivations of the parties that would be affected by the loosening of the rules are not all altruistic or even ethical is worth taking into consideration, but it can't be the only priority when crafting regulatory strategy.

[dithering]

That's.... extremely sensible. A lot like not allowing a market for human organs.

[nybble41]

There is nothing sensible about not allowing a market for human organs, unless your goal is to ensure a shortage of organs.

[dithering]

You should look up where most of our medical teaching skeletons come from.

[HarryHirsch]

One proposal, developed by American economist Bartley Madden, is "free-to-choose medicine."

Physicians are extremely serious about medical ethics, and lawyers are very conscientious about conflicts of interest. What is wrong with the economic profession, that they talk and talk without any real-world considerations. We saw why Theranos tried to sidestep FDA authority, and with that experience fresh in mind that fellow wants to abolish efficicacy testing. Here, have some homeopathic globuli!