The sad part is ofcourse the one about companies not being interested in drugs for rare diseases, even more when they're not needed frequently. I really hope we can find a solution to that.
Hopefully insurance companies will find a way to pay a fair amount, since they'll likely pay less for a one-time cure than during duration of coverage for treatment.
As that'll not happen all by itself because there's less money in that, pharmaceutical companies should be legally required to spend a certain amount of their research budget on rare diseases.
A nice follow-up read regarding one-shot cures:
Aren’t we both reading the same article about that happening “by itself”?
Assuming greed, wouldn't they identify the illnesses with the least likely abilities to cure? (or at the least, least likely to affect their profits, though perhaps they could focus on torpedo'ing a competitor in another area that focuses on a condition they don't)
That is an extremely glib view. There are “some” drug companies that rip off people. The problem with pricing in the Rx world is the high cost and unpredictable nature of discovering new drugs.
Profit margin of tech companies is twice that of Rx companies. Would you say that tech companies are ripping people off?
All of Health vs All of Tech - yes, you are right. Tech is about double.
But Pharma, Biotech, Life Sciences show better returns over Tech as a whole and virtually equivalent Software & Services return.
I would argue that there are definitely players in the Tech space that are raking some very hefty profits, but are in highly competitive markets that could see that erode into losses very quickly... I do not think we are quite in the realm of price gouging and the lower barriers to entry in most of tech keeps things from swinging too far off the charts.
Please stay on-topic! Well, Facebook is collecting data and selling it to pharma companies for probably more than advertising, I suppose. Yes, they are part of the problem. I'm not generalizing who's at fault, but there is a problem.
It's not really even an invention, it's a human protein that was probably identified in academic research a long time before the company decided to produce it and see if it could be used as a treatment.
The pharmaceutical company was not directly involved in this.
The experiment, described in a case report today in the New England Journal of Medicine, took place in Germany in 2016 at a clinic that specializes in rare, inherited skin diseases—particular one called XLHED, in which patients are born with fang-like front teeth and without the ability to sweat.
The problem: their bodies don’t produce a specific protein required to make sweat glands.
The treatment exploited the fact that the missing protein is needed only temporarily, between weeks 20 and 30, when the sweat glands form in a developing fetus. Schneider says his team injected it directly into the twins’ amniotic sacs.
“The great thing about this is the critical time points in the intrauterine development of these sweat glands,” says Anna David, director of the Institute for Women’s Health at University College London. “I think it is the first time you are seeing a protein drug used for correction of a genetic disorder before birth.”
Corinna, [the parent], says she thinks the XLHED treatments worked. “Extremely successful,” she says. “The twins can sweat normally.” They still have somewhat unusual facial features and missing teeth.
I wonder if this will become a problem for us moving into an age where we know enough to "fix" ourselves but not completely. Basically just more medical riders, instead of a bum knee, you have a bum gene.
A sound strategy (IMHO) would be for humanity to target the most costly genetic anomalies and iterate from there, using cost savings to accelerate the process.
It is not a "gene disorder" in the traditional sense, but you could certainly make the case that it is.
Poor understanding of the world before science came around lead to lots of folklore and myths (i.e. fake explanations), given the facial features I'd be interested to know of any historical link.
It likely would have been considered a case of https://en.wikipedia.org/wiki/Monstrous_birth
(Did I type Goldman Sachs correctly the first time?)