Former hedge fund manager acquires life-saving drug, raises price 5,555% to $750/pill

Tyranny of the seen ...

ETA: There are plenty of drugs/therapies that have already been approved elsewhere in the developed world that have yet to pass muster with the FDA. It takes no great leap of logic to assume that there are others in addition to those.

 

That's the rub. I have no doubt the drug approval is riddled with problems. However, an assertion the FDA leaves a "graveyard" of life-saving drugs behind is something that can be proven, right, or is that speculation based on what could happen if their wasn't a drug-approval regime?

 

All snark aside, I would love to see the data that supports this theory.

 

You're unlikely to see any such data. We can't know: 1) which drugs should have been approved; and therefore 2) how many people would have been better off had those drugs been approved.

The realty is that FDA regulators are confronted with two risks: 1) they could approve a drug that turns out to be dangerous; or 2) they could fail to approve a drug that could have been effective. If the first occurs, there are stories about it in the New York Times. Nobody notices when the second occurs.

We can only get glimpses into the consequences of this dynamic. A few years ago there was a meningitis outbreak at Princeton. A vaccine for the strain implicated was already approved for use in Europe and Australia ... but not here. Alcon developed an intraocular lens (for those with cataracts) that was long approved in Europe and Australia, but was only approved here just last year. While these drugs were awaiting approval here, people here died of (or suffered from) the conditions toward which they were targeted.

These instances merely point to the problem of the delaying of drugs/therapies. They don't speak to instances in which drugs/therapies aren't even brought to the testing stage.

 

To go down the rabbit hole, which part of the "nanny state" are we willing tolerate? I assume the vast majority of us like some form of the "nanny state."

 

Just to suss out one of your examples, the Princeton outbreak was meningitis B, for which there are two vaccines now licensed here. During that outbreak, I'm almost positive public health officials let those vaccines be used as INDs with informed consent.

But look at the other side of the coin: There was a pertussis vaccine that was very effective, but also very reactogenic, on the market back in the 1970s and '80s. These reactions, while they could be painful, were almost all temporary and expected, thus communicated to vaccinees. There were very few, if any, permanent or long-term side effects.

But that vaccine ended up putting more than a dozen drug makers out of business and resulted in hundreds of millions of dollars paid to a class action suit, and the creation of the National Childhood Vaccine Injury Act. It also led to a different, less reactogenic vaccine being manufactured, which is much less effective and has led to a pretty big pertussis outbreak over the last decade.

 

I think it's probably more appropriately framed as "how much" and/or "in what manifestation" rather than "yes/no" ...

 

Yes, Princeton Univ. officials got a waiver from the FDA.

You point to an interesting side to the FDA-as-roadblock view having to do with its definition of "safety." I don't know all the nuances of it, but as I understand it they weigh safety from the perspective of "the average person." For, say, a case of ringworm, that's probably fine. But for, say, pancreatic cancer, that doesn't work all that well; the person with pancreatic cancer's going to have a far lower safety threshold than the ringworm sufferer.

 

Right, which means my vision is different then your', your's is different from Ragu's, Ragu's is different from the person deep off the grid in Montana, etc. America has always had a big "nanny state" component, going back to blue laws. Whose vision takes precedence?

 

Whichever party's HofR nominees wins the most votes nationally, I guess!

Seriously, as a matter of political preference the nanny-state version of the FDA ain't going away. That doesn't necessarily mean that on net it makes us better off.

 

Just need common-sense reforms, plus eliminate fraud and waste.

 

That's all taken into account.

And, there are many products that aren't "licensed" for general, routine use because the benefit doesn't outweigh the risk, which could be practically non-existent on a grand scale.

But, these products are available for use in emergencies, when they would be used in people who would never get it otherwise. They are given on an Emergency Use Authorization, like, for example, when Hart Building staffers were offered anthrax vaccine after the 2001 attacks. There are no absolute restrictions in cases like that.