[derlvative]

Picture the situation: someone you love has just been diagnosed with a death sentence disease with no know treatment. The FDA just chose not to outlaw a promising but unproven new treatment for it. Now you get to choose whether to take it or not. And somehow the FDA are evil twisted bad guys!?

[hmottestad]

And your loved one dies of the disease at the exact same time they would otherwise have died of it, but now only after having gone through lots of expensive treatment and suffered a bunch of side effects.

The FDA is definitely the evil and twisted bad guys if they can't stop snake oil sellers from stating that their products cure disease X just because the snake oil guy us friends with someone high up in the FDA.

[otterley]

The FDA, like other regulatory agencies, exists for a reason: to help prevent people from being ripped off, or worse, by snake oil salesmen. I’d rather we not go back to the days when hucksters and charlatans were selling marked-up dyed water or poison to desperate people out of a horse-drawn carriage.

[cubefox]

There is a nice paper about this question:

> One explanation for drug bans is that regulators know more than consumers about product quality. But why not just communicate the information in their ban, perhaps via a “would have banned” label? Because product labeling is cheap-talk, any small market failure tempts regulators to lie about quality, inducing consumers who suspect such lies to not believe everything they are told. In fact, when regulators expect market failures to result in under-consumption of a drug, and so would not ban it for informed consumers, regulators ex ante prefer to commit to not banning this drug for uninformed consumers.

https://philpapers.org/rec/HANWLA

[derlvative]

Completely false comparison because the drug in question did provw safety though the clinical trial, only effectiveness that could not be determined. People should have options that are not certain death.

[partitioned]

What about the obvious middle position, where the FDA continues to do everything it does and labels drugs as safe and effective, but then theres also non-FDA approved stuff that won't be labeled as FDA approved and you can do it if you want.

[jsbg]

> The FDA, like other regulatory agencies, exists for a reason: to help prevent people from being ripped off, or worse, by snake oil salesmen.

This is obviously false given the existing supplements business. The idea that we have a way to prevent people from being ripped off is naive. But even so, the "reason" for the FDA doesn't change its incentive structure: allowing a drug that kills a few people will reflect badly on it, while disallowing a drug that would have saved millions of lives likely would have little consequence for the decision makers.

[doe_eyes]

There is a reasonable argument for a regulatory agency to make sure that the peddlers of medication are not selling you something that needlessly will harm you.

There is a weaker but still defensible argument that they should stop others from selling you flu medication that isn't proven to work. But one could imagine a less restrictive reality where such drugs simply must be labeled clearly and you make your own decisions. In fact, there's already a weird carve-out like that for "natural" substances, so you can for example buy artemisinin as a "supplement" if you want to...

But what's the humane argument for "protecting" people with terminal or otherwise devastating diseases from trying drugs that can plausibly work, and were created by people acting in good faith, and that had some clinical trials done, but the trials weren't as thorough as a committee wanted them to?

"Yeah, he died, but at least we stopped him from potentially wasting some money."

[freedomben]

Personally, I think it's egregious to deny people possible treatments, particularly in cases like that. I think FDA should be very strict on approvals (i.e. giving it that stamp), but should not be gatekeeping people. If people want to take non-FDA approved stuff, I don't see why regulatory and/or law enforcement should care. There's an incredible amount of paternalism inherent in the current system that nobody ever seems to question, and that blows my mind.

Now that said, I have a huge problem with this skipping. FDA rules need to be applied consistently and follow the process. I'm sympathetic to the desired outcome, but the problem is the system is set up as a gatekeeper. Fix the system, don't try to hack around it like this. That just further undermines trust and faith in the system.

[dabraham1248]

> If people want to take non-FDA approved stuff, I don't see why regulatory and/or law enforcement should care.

Well, among other things, if anyone makes money selling colored water, it encourages others to sell colored water. It also encourages companies that are trying to sell actual drugs to skip that expensive "testing" phase. Bad money usually chases out the good (https://en.wikipedia.org/wiki/Gresham's_law) so it's likely that we will devolve into a system where very few drugs are adequately tested.

I'm not saying it never happens, but if a law, policy, or agency reduces big corporation profits, then it's almost always because lots of people were dying.

[michaelmrose]

The argument against it is soaking the health care system for unlimited funds until it collapses or simply becomes unaffordable.

For the horrible things at the edge of treatability and beyond there are always many promising ideas most of them worthless or bad and the price people are willing to pay is either all their money or all everyone's money depending on whether insurance covers it.

If you don't allow insurance to cover it then only a handful of rich folks get soaked but this is politically difficult to maintain.

[tchalla]

What’s the best argument you’ve heard against your position about it people taking non-FDA approved stuff?

[thayne]

That gatekeeping protects desperate people from being scammed into spending tons of money on ineffective, and possibly dangerous treatments.

Is it perfect? No. Could it be improved? Yes. But the rules exist for a reason.

[ooterness]

No, not "promising but unproven" at all. This drug failed its clinical trial. It got its chance, and the evidence indicates it doesn't work.

[goldpizza44]

> Now you get to choose whether to take it or not.

The question is who is paying for these treatments? Drug companies are not shy about charging tens of thousands of dollars a year for a course of treatment that may or may not help. Insurance companies and Medicare partD will probably be reluctant to reimburse. Drug companies have large incentives to get patients to pay out of pocket, and no, they won't refund if the product is not effective.

Perhaps if the FDA were to create a new class of approval that states: Drugs under this class are shown to not cause harm but are also not proven to be helpful. They may be taken, but drug companies may not charge for supplies until efficacy is shown in controlled conditions.

The primary issue here is there are plenty of Snake Oil salesmen ready to tell you anything you want to hear and have no qualms about taking "somebody's" money.

I am sure creating a new class such as this, will also create a host of unintended consequences though.

[devmor]

Your imagined situation relies on the supposition that the new treatment is "promising" - how often do you think this is the case?

A person facing death from disease is desperate, and desperate people are easily taken advantage of. This is part of the reason the FDA exists - to protect desperate people from being ripped off.

The FDA can and should prevent unproven medicines that are not ready for human trials from being used without due diligence.

[Klinky]

There's already Expanded Access / Compassionate Care options for terminal patients. It doesn't require the FDA rushing through unproven medications.

[jostmey]

Except that the FDA frequently denies treatments lacking evidence of benefit except in these two cases. The real problem is that it costs too much to bring new treatments through a clinical trial

[derlvative]

Except that I don't think the FDA should do that? I'm arguing that people should have options that are not certain death.

[Aurornis]

The point was that people are up in arms about either outcome: FDA denies approval, people get angry. FDA allows approval, people get angry.

> The real problem is that it costs too much to bring new treatments through a clinical trial

As opposed to what? Just letting anything go? We basically have that situation in the alternative medicine world and the grifters are out in full force peddling things that we know don’t work the way they claim. Imagine how bad it would be if we waived away clinical trial requirements.

[lavelganzu]

This objection is pure ignorance. "Compassionate use" is already one of the expanded access exceptions, and has been for over 50 years.

[n4r9]

The links from OP are paywalled but the first few visible lines are enough to cast significant doubt for me on whether the treatment is even "promising":

> [Sarepta's] gene therapy for Duchenne muscular dystrophy failed to improve muscle function compared to a placebo in a large clinical trial

https://www.statnews.com/2023/10/30/sareptas-duchenne-gene-t...

> the drug failed a large, Phase 3 trial last year ... three review teams ... wrote that the data Sarepta submitted "cast significant uncertainty regarding the benefits of the treatment".

https://www.statnews.com/2024/06/20/sarepta-duchenne-elevidy...

I'm not sure what makes this treatment "promising" other than the gushings of the CEO. Are you arguing that we should give new treatments a free pass on regulations as long as its for a death-sentence disease and the CEO has connections?

[bedhead]

I bet the treatment costs somewhere around $350,000.

These should be approved on the condition that the patients pay into escrow and get refunded one day if the drug fails trials.

[amluto]

I’d like to see a payment structure more like a bounty for efficacy. If you think you can cure DMD, you know up-front what you’ll get paid, and it depends on how well it works, dosing schedule, side effects, etc. If your drug is safe but barely works, you get paid a tiny bit of money. If DMD patients’ families want the drug and the manufacturer is willing to sell it at the appropriately low price, fine.

IMO the actual failure here is the fact that Sarepta can charge $3.2M for a product without demonstrated efficacy.

[redserk]

I don't think being in that situation with a line of snake oil salesmen promising their unproven supposed-cures will help here.

[pama]

By awarding approval to an ineffective drug, the FDA reduces competition that could otherwise have led to an effective therapy in the future.

[JumpCrisscross]

Is it illegal to prescribe the molecule off label?

[derlvative]

This is a good point. Doctors can prescribe drugs approved for disease X off label to treat disease Y even when effectiveness against Y has not been proven. In this case, the drug has not been approved for any disease so it can't be prescribed. Just goes to show how arbitrary the rules are.

[makmanalp]

A customer base of people with profoundly serious troubles at the edge of desperation who are looking for any glimmer of hope. An industry who's incentivized to charge astronomical amounts to an untapped market to recoup costs. A drug that seems to have failed based on multiple trials and 3 separate expert review teams. Now remove the approval requirements. What could possibly go wrong?

It's easy to yell paternalism but this doesn't look like a case of paper pushers getting in the way of a promising drug to me. Also, history is littered with examples of why "buyer beware" does NOT work, and a vast literature on information asymmetry, adverse selection, "The Market for Lemons", etc etc. Also, the FDA already has mechanisms to speed up new drugs getting to patients when the benefit is clear early on, AFAIK.

[jobs_throwaway]

Oh no, now I must use my own brain and make the cost-benefit analysis for myself. I wish it was in the hands of some people who neither know nor give a shit about me or my loved one

[kaibee]

Desperate parents are well known for their medical expertise and cold logical calculus.