Posted on 05/25/2019 5:24:36 AM PDT by SMGFan
US regulators have approved the most expensive medicine ever, for a disorder that destroys a baby’s muscle control and kills nearly all of those with the most common variant of the disease within a couple years.
The treatment is priced at $2.125 million. Out-of-pocket costs for patients will vary based on insurance coverage.
The gene therapy, developed by Novartis, treats a rare condition called spinal muscular atrophy, which strikes about 400 babies born in the US each year.
The Food and Drug Administration on Friday approved the drug, called Zolgensma, for all children under age 2 who are confirmed by a genetic test to have any of the four types of the disease.
(Excerpt) Read more at nypost.com ...
Gold , platinum, Brown Lipped Abalone, Almas Caviar. Wagyu Beef , Italian white Alba truffle , /s
https://en.wikipedia.org/wiki/Onasemnogene_abeparvovec
AVXS-101 is a biologic drug consisting of self-complementary AAV9 virus capsids that contain a SMN1 transgene along with promoters.
Insurance companies won’t cover this. Cost is back-breaking. This is one of those ‘orphan drug’ things. Yes it is heart breaking for a family with the child but the cost is beyond reasonable.
The cost to make nearly all drugs is irrelevant. Most cost nearly nothing to make, compared with the cost of development. The cost of development is driven totally by government regulations, and is usually more than a couple of billion dollars. This cost must be divided by the number of sales over the remaining decade long life of the drug patent. Development takes around a decade away from the 20 year patent protection. The patent requires publication of sufficient information that any competent drug company can duplicate the drug (at usually next to zero production cost).
Given $2 billion cost divided by 10 years at say, 100 patients per year, gives you the $2 million price.
“but the cost is beyond reasonable.”
Most likely the cost to produce is very high. Also, there is the development cost and the approval process costs. Over a decade ago the estimated cost for the approval process was over one billion dollars. That’s just the FDA approval costs, not including developing the drug. If we had the full expenses you would find the drug developer was already in the hole for bushel baskets of money.
As an example of where medicine is going, my sister has eyes so dry she is at risk for a number of diseases. She just paid $400 for eye drops. They take her blood and process it. The drops are good only for her. They can’t be used by anybody else. And, they work. What is the yearly cost? It’s going to be in the thousands of dollars. For eye drops.
It is thought that a single dose of the drug will have a lasting effect throughout the patient’s lifetime.
Not many will be able to afford even the single dose, but at least it’s not a daily pill.
Thank you, in how many years will it be before a family in need will have this drug?
Pharm companies gotta make their money some how.
What better way than taking advantage of desperate people?
I am sure that to some degree some drugs are more difficult to manufacture thus cost more, but the pharm industry has already set the precedent itself for the price gouging they do.
In light of that, it becomes almost impossible to believe this cost is justified.
Why on earth would a drug company continue to develope a drug that NO ONE on God’s green earth could afford, I mean what are the odds of extremely wealthy parents having a child born with this disease?? Knowing the cost of developing this drug and how few people could actually EVER afford it WHY on earth continue the research and development!!! There is NO WAY in hell any insurance company would cover this!!!
How much do you charge for the spinal muscular atrophy treatment that YOU developed?
Because research leads to more research.
A success here might have impacts on other things, where they will continue to research and hopefully make other in roads.
AVXS-101 is a biologic drug consisting of self-complementary AAV9 virus capsids that contain a SMN1 transgene along with promoters.
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It is the recombining microbionic binuclesic stabilizing compound inherent in the virus capsids which make this work.
Two decades of research, development, and testing by several dozen PhD chemists, biologists, and physicians. A larger number of highly trained laboratory technicians. Several billion dollars worth of laboratory equipment whose function you wouldn't understand even if it was explained to you.
THAT's what it costs to produce.
Here's the catch 22.
Imagine the wailing and gnashing of teeth, if the same company DIDN'T develop the drug, but could have, but refused because of costs.
Pick your poison.
1-They don't develop the drug, because of cost.
2-They develop the drug, but nobody can afford the cost.
“Why on earth would a drug company continue to develop a drug that NO ONE on God’s green earth could afford,”
This is just a guess. The importance to the drug company was not the drug itself. It was the technology used to develop the drug. This was not just mixing likely ingredients together to produce a number of compounds to try. This undoubtedly put the scientific method and gene research through its paces. If we could look into the back story we would probably find a significant investment in highly advanced machinery that will automate the process of future drug development.
Typically, the first of anything is expensive compared to the final manufactured product. The difference between this and, say, a prototype aircraft, is the emotion involved in the final use; medicine for children. In my manufacturing career we sometimes ran short runs of related products on our main line, thus reducing their costs. I think that once the new medical production lines are up and working the overall cost of a short run of a little used medication will become a loss leader and public relations issue paid for from the advertising or public relations budgets. So, hang in there. This development is probably a lot better than the article makes it seem.
As far as the “cost” I suspect the company put the full development cost into this drug so they can write it off as a tax loss. It’s for accounting purposes. Can you imagine that the people running the drug company don’t know it’s unaffordable to anyone? They know. They know no insurance will pay for it, but they developed it anyway. That alone should give us hope that they already have a plan, after taxes, to make it available at a cost that people can afford. They didn’t do this just to prove how out of touch with reality they are.
To me, that is not the question. The question for me is "How many family members will die because government regulations and confiscation of profits prevent development of new and better drugs?"
I never knew either of my grandfathers, because they died before I could know them. The drug that would have cured them is now given away free at my local drug store as a loss leader. Drugs that might cure my cancer, or other's diseases, will only be developed if drug companies make enough profits to cover these development costs. If not, far, far more will die from what could be curable diseases if drug company profits are confiscated by government control.
I’m sure they’ll justify it.
Having been screwed over myself by pharm companies with unjustified drug costs when the reps walk into the dr’s office in clothes that probably could probably feed us for a month, driving the best car in the lot, and buying the whole office lunch, I have little sympathy for the whining of drug companies that gripe about the costs of R&D.
Need to compare the flip side. Some of these kids could go thru well over $2N in special disability care without TX. How much of that might this save? The cheap option would be diagnose, then late- or post-term abortion. Probably not popular here.
Phase 3 clinical trials typically requires some company/compaies to pay ALL the medical costs of each of the THOUSAND people taking the drug, maybe for 4 or more years.
Otherwise there’s no “scientific” evidence the drug works or not, right...? It’s not optional. It must all be documented.
Sometimes there’s even Phase 4, if the FDA feels like requiring that.
From the time you start preClinicals through Phase 4, it can be maybe 14 years.
Typically that’s $1.3 - $2 billion dollars, yes for each and every drug approved by the FDA. It used to be Everests of paperwork, now it’s stacks of DVD’s (in addition to those same mountains of paperwork).
That is why so much of the outside world simply takes as valid wholesale, whatever the US FDA says:
Cuz going through that whole shebang in, say, Guatemala, is just not feasible. That’s also why more medical testing is being doing in various countries in Africa.
In 1915, before the legal requirement for lots of human testing before marketing the stuff to the public, there was no FDA:
Can’t remember what old medicine it was but this medicine was soluble in almost nothing. A chemist started playing around with looots of different things and he found a liquid —the medicine dissolved nicely in there. Plus, it was sweet..!
The liquid was ethylene glycol. Anyone out there recognize it..? Yes, it’s the majority component of ANTIFREEZE.
WHICH KILLS YOU. It kills your liver very easily, which is why there are so many warnings against draining your radiator onto your driveway or whatever, where animals will trot over and lap it up.
But they were selling it —I think it was a cough syrup— already and it gradually killed hundreds of kids.
That chemist killed himself and it led to the establishment of the FDA.
Even in Europe not so long ago there was the whole flapper arm THALIDOMIDE tradegy, right?
It didn’t happen here cuz of the FDA, cuz of all the costs and headaches of regulation.
People want stuff, they just want it to be cheap and also totally safe.
SORRY..!!
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