The lack of published negative results isn't a problem unique to pharma but the drive for profits and the actual life and death outcomes makes it particular severe in this area of science, I think.
I highly recommend the book, it was both hilarious and terrifying.
Both don't always report results. Academia is worse. That doesn't make the industry good in any way.
I'm not going to claim that industry is perfect. But take a look at the graphs. It is a picture of an industry that is generally in compliance (enforcement could perhaps be stepped up), compared to academics and government who appear to believe that compliance does not apply to them.
(Edited some typos)
This is high stakes medicine, yet the current environment has the docs on this floor terrified of doing anything that isn’t in the standard of care runbook. My wife has had elevated blood glucose levels for six months, which is not good when you’re fighting cancer, yet i can’t get anyone to treat it because there’s no clinical evidence that managing glucose levels for her specific type of cancer has any benefit.
Guess what. Nobody’s doing that trial any time soon. Meanwhile there are thousands of diabetics managing their blood glucose while fighting cancer and there is endless material available demonstrating that cancer cells thrive in hyperglycemic environments.
So, i don’t know anything about Juno, they could be sociopaths. But i have no issues with high risk attempts as long as the patients are informed and there are ethical standards for transparency. At least they are trying.
If you're not opposed to eschewing professional recommendations to not complicate treatment, there are two things that may be worth looking into:
1). Metformin: Helps control blood glucose by significantly increasing sensitivity of insulin receptors, i.e more glucose is shuttled out of the blood stream per insulin molecule. Not scheduled and very cheap. Also trials are being done on it for cancer prevention -- exactly because of Warburg's phenomenon (cancer cells and glucose)
2). Ketosis: Hit or miss. By abstaining from carbohydrate consumption, the result should be drastically reduced blood glucose levels. However, in some people (e.g type 2 diabetics) it may elevate blood glucose due to metabolic disfunction. In any case, caloric restriction in general have the effect of both increasing insulin sensitivity and decreasing blood glucose -- regardless of macronutrient profile
I must point out that some CAR-T cell therapy is already approved. I am not sure about Juno's particular candidate, but it isn't obviously insane that it was sold for billions.
These are NIH grants to academia - industry is not involved.
I’m personally invested in seeing something like this happen but if not approached defensively it could be vulnerable to getting shot down and made an example of.
But only studies that are pre-registered should be considered when approving a drug, treatment, or device, and any incomplete studies (that is pre-registered but not reported for any reasons) should be treated as "all the patients died", and all individual researchers on the preregistration should be appropriately tainted for future consideration of studies in which they participate.
The related story in the article, "Gaming the system" does report on this:
> .. a recent study ... examined 19 drugs approved by the FDA in 2015. In only 10 cases were all the trials provided to FDA for its decisions registered on ClinicalTrials.gov.
This is unacceptable.
What if you kept repeating experiments, and only published when you got the results you wanted? That goes against my personal ethics and my understanding of how science is supposed to work.
This is pretty much how it's currently done. Science is far less exploratory than you make it seem. When it comes to publishing you should think of it more like law. You make the strongest possible case for your conclusion.
Even better is really cheap experiments like analysis of existing data. That you can keep trying until you find something, sadly this means publishing datasets is a bad idea.
Except the data shows that it's not the industry who's failing to report results, it's academia and the government who's failing.
Pre-registration is supposed to solve this problem by listing the studies before they are conducted so that it is impossible to completely elide them. A drug approval would then look at all studies pre-registered for the treatment, and only at those studies pre-registered for the treatment -- whether those studies were complete or not.
There are unfortunately many cases that have been decided on the basis of finances rather than clinical justifications. I don’t envy the agency, stuck between the disdain of the public, pressure from industry, and continually having to justify their existence to Congressional committees.
Then the reality is, it wasn't mandatory.
It's not reasonable to expect citizens to trust these entities when their conduct is not trustworthy.
Same should apply for pricing. The fact that prices are completely opaque in the US makes it hard for patients to make good decisions.
He argues that the FDA did more harm than good, and that society would be better off without a governmental supervision for health care in general.
btw, in the end he makes a strong argument for negative income tax. This surprised me, as my model of his world-view was apparently more simplistic that it ought to be.
FDA also love stating that they prevented a drug that causes birth defect a while back unlike Britain.
Another example of government regulation working is cars. Death relating to car crashes have fallen every year with safety regulations.
It's not perfect but there are many cases that it works and there are laws and rules set in place.
The free market is not the panacea for everything.
Things like truck under-ride guards likely wouldn't be on commercial trucks if they weren't regulated. Consumers aren't going to all voluntarily coordinate to buy vehicles which have crash-compatible bumper heights. Antibiotic misuse would be even more out of control if it weren't regulated.
Mexico is a good example for road regulations. They do not have the same crash regulations as the US, and many old model vehicles are still legal for sale there. Things like the 1950's VW Beetle were still sold there into the 2000s. They have the highest death rate per distance traveled in the world.
Friedman and the "Chicago Boys" advised Augusto Pinochet in Chile and used his dicatorial powers to ram through changes into the Chilean economy. I don't think Friedman is someone to admire.
It is willful or reckless ignorance to believe that the absence of the FDA will not have serious consequences. There already exists a segment of the drug industry that is nearly completely unregulated by the FDA, as a matter of law. I am of course speaking of the health or dietary supplements. And the experience here does not bode well: there are a sheer number of "miracle" pills and cures that come out of it, unhelpfully promoted by snake oil salesmen like Dr. Oz. And investigations have found that many companies can't even be bothered to put the active ingredients into their products.
Edit: Just to clarify, I'm not arguing for or against here, I just stated another option - so thanks for that downvote whoever the lazy person was did it.
Libertarian logic I guess...
And this creates multiple points of competition, unlike regulation which is somewhat anticompetitive. Transparency, efficacy, cost, reviews in the age of the internet, all combine as a form of emergent regulation which may outperform explicit agencies in some cases.
Committees Of Concerned Citizens On The State Of Toothpaste will pop up and dissolve like mushrooms, some of them funded by this or that corporate interest, some being incompetent to evaluate state of anything, and the rest drowned in all the noise.
Emergent anything only works if there is an explicit external force pushing things towards a specific organization or a set of laws of nature that produce outcome you happen to find satisfactory.
I guess your point was that current state is worse than that already, and that's a valid point, although I don't think this is the case.
The Libertarian capacity for ignoring Chesterton's Fence is nothing short of astonishing. Free markets require free information - the FDA was created in large part to enforce accurate labeling.
It turns out we can directly compare pre-1962 FDA with the current FDA and find strong harms outweighing any benefit of the current regime.
If all the FDA did was "enforce accurate labelling", they wouldn't be responsible for hundreds of thousands of deaths from delayed approval of drugs. Maybe that's not the part people are complaining about.
I find this kind of motte-and-bailey stuff ubiquitous in discussions with libertarians, as the "philosophy" amounts to anarchy which most people realize is bad.
Anyway, the Milton Friedman talk linked explicitly mentions the same beta-blockers example as my link discusses and cites it as causing tens of thousands of deaths. He says it's been a disaster over "the last 20 years" - the talk was in 1978, so this is roughly the 1962-1978 period. And the "governmental supervision for health care" mentioned is broader than the FDA.
Drugs quality testing could also conceivably be performed by a non-government regulatory agency (e.g. Underwriters Labs)
--Ron Paul, noted libertarian and advocate for the abolishment of the FDA, who is apparently ironically ignorant of the phenomenon of "snake oil".