In my personal opinion, medicines in the US are far too regulated. I agree with what you're saying about efficacy versus safety, but I'd even go further in my stance that safety is really overstated. There's a huge difference between something like the safety profile of elixir sulfanilamide and most of the drugs on the market today. Many of them could be distributed without a prescription over the counter, or distributed behind counter under the regulation of pharmacists, or distributed with a prescription by a wider range of providers. It's absurd to me that something like alcohol is something you can buy in the grocery, but something like acyclovir requires a prescription.
I know my personal opinions about this are unusual, statistically speaking, but I think the pendulum has swung far too far in the other direction since the 1930s. It's frustrating to me that debates in the US are often framed in terms of two extremes, one resembling what we have, which is broken, and another where there's no safety oversight at all, as if those are the only options.
I personally feel like the FDA should concern itself with regulating the characterization of basic safety profiles of substances, and with product purity and truth in labeling. Outside of that, it seems like you start getting into rent-seeking.
Regulators here generally have a mandate to screen for both safety and efficacy. I think the mandate for efficacy is quite important--if you loosen standards there, you get warped incentives, and you'll end up spending lots of money on things that the producers know don't work. We already have an example of such an industry in the health supplement industry.
I'm directly responding to your (insane) line of argumentation: You're arguing that medications that have not passed regulatory requirements for safety or efficacy should be sold to consumers, and that they can simply rely on retail pharmacists or the like as a stand in for the FDA's approval process.
The general problem is there's not enough basic funding to fund quality safety trials. Which are still run, but by the manufacturers themselves.
What probably should happen is more pushback from agencies with downstream remits like the DEA once statistically durable negative effects are observed on the ground.
Yet somehow countries that regulate drugs merely by safety tend to produce better health outcomes than countries that regulate for safety and efficacy like the US FDA does. You're just asserting that the upsides outweigh the downsides, but every study I've seen that tries to measure things quantitatively comes to the opposite conclusion.
The gullible are already getting snake-oil in the form of herbal remedies and homeopathy.
With the current regulations in place, it's definitely possible that regulated drugs have killed more people than unregulated drugs.
Would that still be the case without any regulation? All the regulations do is ensure that products intended for pharmaceutical use have a certain level of evidence of safety and efficacy.
It doesn't mean that unregulated products are unsafe or do not have a positive effect on health, it just means that one cannot sell them or market them as something that can cure disease.
Keep in mind that even with this regulation in place, people still try to sell things like the MMS [0], which the FDA warns against consuming [1].
While regulation isn't perfect, in this case I'd argue that it's a net benefit, all things considered. It's not perfect, but it's better than having no regulation in place.
My take is that we need to strive for effective regulation (so I'm not coming at this from a 'government is always the problem' perspective, just thinking out loud).
There is some incentive for medical providers and insurers to use safe and effective treatments (good reputation is good business). For products that are not immediately physically dangerous, perhaps retrospectively evaluating their choices would be similarly effective for outcomes as the current approach of banning things until they are approved.
I was not suggesting that we need more regulation necessarily. It's a problem of law in that it's entirely made possible (and incentivised) by laws. Patent law and laws governing the development, marketing, distribution, and crucially import of medicines.
That being said, clearly the current system has flaws. Perhaps the FDA could alter their requirements for sale to approximate "does not cause harm", with additional classes for "provides more benefit than harm", etc. The problem is, how do you get drug companies to pay for effectiveness studies if they don't have to? This would actually disincentivize such studies.
Acetaminophen is a interesting example - it's a very (centuries) old drug that predates the regulatory framework we have today. Some scholars (see ref. 1 for some history) suggest that it would not be approved by today's FDA.
It's thus a poor selection if one wishes to discredit the FDA's efficacy; indeed, some other widely-used therapeutics (e.g., aspirin) came into use before drug efficacy was required for new drug approval.
At the same time, there are numerous examples of drugs either not approved by the FDA or withdrawn from the US market by the FDA. It's not clear how less regulation would achieve this type of nation-wide safety benefit.
I think some regulation is needed though. I can't look at a generic white pill and tell whether it's oxycodone or acetaminophen. Also, I don't want to take your acetaminophen if it hasn't been proven that whatever is in it won't cause other more serious issues or that I've at least been adequately informed of those side-effects on the front-end.
R&D is also in-part expensive because of the success rate of drug trials and the expertise necessary to conduct the experiments. I'm mostly a free-market proponent, but when a corporations own interests collide with the larger market's interests by killing or otherwise removing a large portion of the productive population from the workforce, I believe there should be regulation.
Efficacy is a bigger problem than safety. Think about all the products you see advertised on a typical day (especially if you watch TV). How many of thing actually work? Anti-oxidants, probiotics, shampoo with gobbledygook ingredients in it, homeopathic teething ointment.
For the most part, all this is just a waste of time and money. But for medicine, things that don't work create an opportunity cost--someone uses an anti-oxidant-packed cancer treatment and dies. It's a basic information asymmetry that exists in the market: its too hard to prove that things work, and the wonders of modern marketing ensures that lots of things that don't work still sell like crazy.
Efficacy tests address that problem. But proving efficacy is hard and expensive. But they're probably not "too expensive." That is to say, if you made clinical trials cheaper by just testing for safety, you'd probably suffer a net economic loss due to people spending money and dying from products that don't work than you would on the trials.
The problem isn't "should we have regulation at all," but rather what is the right level of regulation. It is quite possible that the current burden of proof is too high in many cases. If drugs were made more broadly available sooner, it would likely save many lives. But also it would also likely cost some. Do we currently use the correct threshold?
There is certainly evidence that since regulatory bodies get tons of flack for letting through bad drugs, but aren't imposed on that much for slowing down the process, they incline to being risk adverse. We also have evidence of outcomes in Europe where the regulatory regimes differ and seem to approve things faster with outcomes that aren't generally worse than the US.
I highly doubt we have the optimal regulatory solution at present and could do much better. And if anything we have standards that are too high.
Is anyone even reading my comment or any of the comments above me? I'm not arguing against the FDA's safety requirements. The argument is for decoupling safety from efficacy requirements to make a treatment available to acquire. Right now availability is coupled with mandatory coverage by Medicare & insurance, which means treatments with borderline/controversial efficacy results like this Alzheimer one is either totally banned or mandatory for prescription & coverage. You wouldn't be able to sell heroin.
You think the average consumer is going to read (let alone understand) dense clinical trials and review data from NIH and elsewhere to make their own determination on the risks and benefits of a drug? LOL, give me a break.
Efficacy and patient safety matter. That's why the FDA exists.
The FDA has a solid origin story: make sure drugs sold are safe. But the 1960s expansion into regulating efficacy has not been positive, and the organization today is not even clearly positive on balance.
Today if the FDA approves a drug as safe, and effective for any single specific use, doctors are allowed to prescribe it "off label" for any other use. This generally works well, and contributes to pharmaceutical innovation. We should expand this to where doctors can prescribe anything the FDA has classified as safe. Restricting advertising and other carrots to drugs that have convinced that FDA of their efficacy (as we do today for off label uses) is probably still good, though.
This is one of those situations where we want the market to solve it, but we probably aren't prepared for what a totally unencumbered market response would be. We've likely over (or at least poorly) regulated drug development, and since most things are cyclical, it will probably swing the other way at some point. That's probably good, as long as it doesn't swing too far. I want a more efficient system, but not necessarily at the expense of a few million casualties from cascading mistakes.
Unfortunately, since it has to do with people being hurt, or the possibility of people being hurt, I think it's unlikely we'll get legislation or a majority of politicians that are willing to look at it rationally. It's too easy to drum up emotional support for some aspect or another for personal gain.
In some ways, I think the FDA is a victim of its own success. If I'm not mistaken, it was created in part because of the 100+ deaths around Elixir sulfanilamide. But since the FDA has helped prevent things like that from recurring, we underestimate the potential dangers of under-regulating.
Not that the FDA couldn't be more efficient (possibly considerably more so), but it's clear that most products that go into production work, and that can't be discounted.
I actually wish the FDA was far less strict. One of the reasons we have so many ineffective drugs is that the FDA values safety over everything else. The ideal drug to get licensed is a sugar pill that due to statical chance (or fraud) shows a slight effect. No side effects and you can sell it to everyone. Of course it wouldn’t do anything, but it would make you a lot of money.
If the FDA was less strict then drug companies would develop drugs that actually worked, but also had side effects (basically we would tip the balance back in favor of effectiveness over safety). While it would always be ideal to have a drug that worked perfectly without side effects, it is always better to have a drug that works than one that doesn’t. You can always choose to not take a drug because of the side effects, but if the drug is not sold then you don’t have a choice.
I know my personal opinions about this are unusual, statistically speaking, but I think the pendulum has swung far too far in the other direction since the 1930s. It's frustrating to me that debates in the US are often framed in terms of two extremes, one resembling what we have, which is broken, and another where there's no safety oversight at all, as if those are the only options.
I personally feel like the FDA should concern itself with regulating the characterization of basic safety profiles of substances, and with product purity and truth in labeling. Outside of that, it seems like you start getting into rent-seeking.
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