in the 1950s, synthesizing steroids was expensive, so doctors used the pig-derived acthar (which was actually developed by armour & co, the chicago meat packing company). then cheap generic synthetic steroids gained use and acthar use declined, as they basically had the same effects.
except that acthar worked better than corticosteroids to treat a rare form of epilepsy in infants called infantile spasms, even though the drug was not FDA approved for this disease. however, there were too few patients for the drug to be profitable in that indication, so the manufacturer of acthar decided to take it off the market. physicians and patients revolted, as this was the only drug that worked for a serious disease, so the manufacturer kept selling it though they lost millions a year on it
enter questcor pharmaceuticals, which bought the rights to the drug for $100K. they cobbled together some existing publications and got FDA approval for infantile spasms. they increased the price of the drug from $30 / vial to $30K / vial, and then started marketing it in other indications like multiple sclerosis. there was no real evidence it worked in these indications, but because it was approved in the 1950s before modern FDA standards, it retained many approved indications
questcor was eventually acquired for $6B, and to this day there is no competition for acthar because there is no FDA pathway for making a generic version of a pig-derived drug
there is little backwards compatibility for FDA regulations
If you want to use it, though, you have to leave the US or have a veterinarian as a friend. An acquaintance of mine gets DMSO gel used on horses to treat inflammation from a vet and uses it to reduce swelling in his back (has had multiple major spinal surgeries).
The common conspiracy theory is that DMSO is too cheap and treats too broad a category of ailments, so drug companies are lobbying against it with the FDA any time it comes up, as it would jeopardize the revenue streams from steroidal anti-inflammatory drugs, painkillers, etc.
https://en.wikipedia.org/wiki/Dimethyl_sulfoxide#Medicine http://www.dmso.org/articles/information/muir.htm
Why? New drugs have been getting less effective over time, even though the scientific requirements to get them approved have been becoming stricter. The majority of our most effective drugs were approved before there were any requirements for demonstrating safety and/or efficacy.
Also, from that pool of drugs you're referring to, we've eliminated a number of things that had strong effects but whose side-effects were later found after careful study to not outweigh the benefits.
There is a similar effect from carefully studying devices/surgical interventions--not so much a raft of "new innovations" but rather the culling of a lot of things that were overused or actually harmful. Huge cost savings, big improvements in population health & lots of gored oxen.
I personally think FDA approval is dubious in cases like the OP mentioned.
We know from centuries of use that the plant is safe. Whether it helps their gout or not is irrelevant. Even if it's a placebo effect, who cares? Trying it out won't kill them, and if it doesn't work they can move on to something else.
Why waste tax payer money "proving" something like this?
I love the protections the FDA gives, but we need to use common sense, too.
We've known (for various confidence levels) that lithium salts are effective for some mood disorders since at least the 1870s (though there's various stories and one-off doctor recommendations that survive about lithium salts or natural water sources with high lithium levels being good for melancholy much older than that), then the FDA banned it in the 1940s due to the nasty discovery of lithium poisoning fatalities, and now we're using it again since 1970, with recurring blood tests to be sure you're not accumulating too much.
We used lead regularly in all sorts of things until, surprise, lots of people often exposed to lead turned up with nasty health issues.
We used asbestos all over the place until, decades after we started using it, we realized "hey, maybe all these easily-broken-off fibers that turn up in the shredded lungs of people might be related".
We used syrup of ipecac for hundreds of years, until we concluded "I _think_ the side effects from the ipecac are probably worse than the thing we wanted them to expel".
What do you do about all the flawed and weakly powered clinical trials of the past then? Do you consider them invalid at some point? If not, why have double standards then?
Wouldn't that get SOMEBODY to take the nigh guaranteed investment? I think what rubs people the wrong way is the limitless profits that are guaranteed by government sanctioned monopoly for things that save people's lives. It's a strange perversion of the free market that benefits the wrong people disproportionately.
As it currently stands, 60%+ of the new drugs that are developed in the world these days are for cancer or rare disease. Because for most other diseases, drug r&d is not profitable. Neuro is too risky because the biology is complex. Cardiovascular and metabolic disease is too costly because of regulatory and reimbursement hurdles. Infectious disease is a field where generics dominate, and the regulatory system is failing to support efforts to fight drug resistant strains
For-profit companies fund 3x what governments fund. We don't have the federal budgets to support a public drug industry. And we don't have the political will at least in the US to empower federal funding increases of that magnitude
It certainly isn't ideal that profit driven companies control which drugs get approved. But if profit driven companies didn't fund new drug development, no one else would
If it's so straight forward to prove, the government should just fund the studies itself. There's no reason to grant any private company anything in that case.
The top ~15 pharma companies spend over $70B a year on r&d, and VC funds $15B in the US alone
Even assuming government operates as efficiently as private industry, we are talking almost triple the NIH budget to accomplish that
So increase the budget? If we assume the government would operate as efficiently as private industry, then it would be cheaper overall. The other option is for private industry to recoup the costs plus profits from society at large.
Obviously increasing the budget is politically difficult, but let's not pretend the current method makes any real economic sense.
The Unapproved Drugs Initiative sounds much worse.
This right here is the problem. The US government could easily have put a 1 cent tax on every pill and used it to have the FDA contract out the safety research. But instead it chose to grant a private venture yet another monopoly.
Without the FDA's slow-moving approval process, the harm done by Thalidomide's side-effects would have been enormously worse than they were.
Here's an FDA blog (apparently these exist - who knew?) on exactly this:
https://blogs.fda.gov/fdavoice/index.php/2012/02/50-years-af...
To date, it is still not fully understood how paracetamol/acetaminophen works. Due to the fact that it was sold long before understanding the mechanism of action was required by the FDA, and there are little adverse effects in the history of the drug, it's permitted to be sold today.
I wonder if there is some frustrated economist at the FDA that always mumbles about incentives and gets told to shut up.
However, nobody ever actually did modern science on it. We all just assumed it was safe and effective, because it had been so for thousands of years.
So, in 2006 the US FDA started the Unapproved Drugs Initiative. The US government said they would give an exclusive right to the drug to anyone who did the science. So, URL Pharma did a tiny bit of research to prove what generations of physicians already knew, and the US Government let them have the exclusive right to sell Colchicine. The price immediately went from pennies a pill to $3 and up.
While I applaud the goal, it is far too easy to turn these well-meaning initiatives into cynical IP land-grabs that are distinctly anti-consumer.
https://www.jwatch.org/jw201006100000001/2010/06/10/spotligh...