Comments
ogpokemontrainer t1_ixusfq7 wrote
Approved at $3.5 million a dose
GratefulOctopus t1_ixuxtu1 wrote
Can anyone explain why it costs so much? Is it just trying to offset the research investment? Like I have a hard time imagining the actual drug is that expensive to make, maybe how it's administered requires some fancy tech? Looking for sciencey/technical reasons. But can accept that it's just pharmicutical business mark up
[deleted] t1_ixuzf92 wrote
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Sariel007 OP t1_ixuzrk7 wrote
In an analysis of the drug development costs for 98 companies over a decade, the average cost per drug developed and approved by a single-drug company was $350 million.[3] But for companies that approved between eight and 13 drugs over 10 years, the cost per drug went as high as $5.5 billion.
GratefulOctopus t1_ixv04rl wrote
Ok so it is mostly just to offset research cost? It's not how much it is to make/administer the drug?
Plastic-Bluebird-625 t1_ixv1486 wrote
Correct. It's not about the cost of making it. It's the years of research with tons of doctors is what makes the cost.
demonmariner t1_ixv22ml wrote
Not quite so uplifting.
FUCKYOUINYOURFACE t1_ixv3d53 wrote
It’s also how many can benefit from the treatment. They have to charge more when the prevalence of the disease is lower.
sfdragonboy t1_ixv4gz6 wrote
This would be awesome if cost effective since current Factor medications or treatments are very expensive.
Sariel007 OP t1_ixv5kr0 wrote
One dose cure instead of "...patients will undergo regular infusions of their missing clotting factor. Though effective at stopping spontaneous bleeding, these infusions are not a cure and in the most severe cases, people may need to get them every two to three days—a costly and time-consuming measure. "
StupidPointless t1_ixv6yyq wrote
So, com that it's approved, but it's anyone actually going to be able to get it? Will insurance help with it at all? Or is this completely out of reach for regular people?
[deleted] t1_ixvdece wrote
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[deleted] t1_ixvdns8 wrote
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cygnoids t1_ixvgx98 wrote
This is the case but it’s also expensive to develop the cells used for AAV based drugs because the cells are killedwhen the virus is made. This, you can’t reuse the same set of cells throughout the process.
Colonelfudgenustard t1_ixvgyb3 wrote
They've got to pay back all those volunteers in the studies. Wait a second . . . the volunteers don't get paid!
nocloudno t1_ixvgz1r wrote
It just took the prize for being the most expensive drug in the world.
KindlyContribution54 t1_ixvi25z wrote
I saw a bunch of scientists weighing in on this when it was posted elsewhere and it does actually have high ongoing costs custom tailoring it to each patient and expensive materials.
[deleted] t1_ixvjjb2 wrote
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CdnAevyn t1_ixvkamn wrote
It depends on the drug, however while I can’t find the article again with a quick search at work, a few days ago I read this specific drug takes an entire team/lab months to create just around a dozen doses.
Until we’re able to make this more efficient, producing each dose will probably be a large part of the cost when taking into account the salaries/costs of all the staff involved. The fact that hemophilia b is quite rare (I think 1 in 25,000 people) will also often lead to a higher cost.
Regardless, this is an amazing step forward in the fight against a horrible blood disorder.
hobopwnzor t1_ixvkpsv wrote
The new generation of medicines is insanely complex biological medicines. So fancy delivery of very complex difficult to manufacture and test drugs that directly alter your bodies mechanisms. Were kind moving beyond the days when you could just make a chemical and expect it to work better than those already on the market.
fireisveryfun t1_ixvl45v wrote
It's a single dose, manufactured in incredibly small amounts - a few dozen a year. From what I've gathered it looks like the price is intended to recoup research costs. That said, the burden to pay really should be covered or at the very least VERY heavily subsidized by the government.
[deleted] t1_ixvna51 wrote
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scarlet_sage t1_ixvnm9y wrote
It did not appear to be a cure, though it doesn't explain why.
>It also appeared to lower the rate of bleeding problems by 54% in one trial, and it reduced the need for regular infusions of clotting factors....
>it should reduce the frequency of expensive transfusions; they also say that the drug’s effectiveness is expected to last for several years at least.
AinsiSera t1_ixvnosp wrote
I read elsewhere that those treatments easily add up to $100k/month, so honestly $3.5 mil is pretty cost effective competitively.
YATr_2003 t1_ixvogvs wrote
There are two reasons the article briefly mentioned, but not other commenters said, so I thought I should highlight them.
The first is that Haemophilia is a rare disease, and as such there are less people to distribute the development cost between, so the cost per patient is high. This is true for any Haemophilia drug, and treatment for some (very) severe cases can be millions of dollars per year. This leads to the second reason, which is that this treatment can be cheaper than the treatment given today. If the government or the insurance companies are willing to pay for it that much money, why not charge them? Yeah, this is immoral and shitty, but since when does that bother pharma companies?
ComradeBlyat308 t1_ixvoxvn wrote
Given the subsidies these places get, wouldn't be surprised if it already WAS paid for by the government, and the research costs they're trying to recoup were ones they never paid in the first place.
RO489 t1_ixvt06y wrote
Depending if your insurance/ employer agree. Since insurance is mostly tied to employment in the US, they aren't firing the math of a lifetime cost. I'd imagine single payer would be a no brainer, but if you are an employer, it'll increase everyone's cost.
Another argument in favor of single payer.
CharlisonX t1_ixvtpel wrote
Does these gene therapies include targeting the reproductive organs? if not, the number of diseased can multiply very quickly.
whiskeyknitting t1_ixvv8ul wrote
I had a coworker whose only son was a hemophiliac and he died after a fall down the stairs in college. No a huge fall for the rest of us, but he bled to death.
It wrecked his parents, whom we were pretty sure were related by marriage. They had an arranged marriage and she kinda lost her mind after her son died.
[deleted] t1_ixvwdcj wrote
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IAMSHADOWBANKINGGUY t1_ixvwg75 wrote
It is permanent? OP said its a one dose cure.
Edit: it isn't.
NoDownsideToOutside t1_ixw1ihm wrote
People fail to realize the grants these labs are given. These companies are definitely double dipping. I’m not saying stuff like this isn’t hard to produce, but I doubt $3.5 mil/dose worth of labor, and research was done, even without grants/subsidies/tax breaks the company received.
Also a lot of these things are developed and then the company buys the patents solely for distribution profits.
TheBearWillBeFine t1_ixw3f6f wrote
I work doing safety testing for drugs just like this. Setting aside all the money it takes to develop one of these viral gene therapy drugs, the cost to manufacture a single dose on a lot of them is somewhere around 50-90,000 dollars. The viruses need to be grown, then emptied of their regular genetic material, have the new, specially designed genetics put in, then replicated up to a level that can be infused into a person. Every single one of those steps requires highly skilled people to ensure it happens correctly. Growth of viruses need to happen in cell tissue culture, which also costs a lot for all the materials to do correctly, then all the supplies needed to purify out the virus from all the other crud in manufacturing. Then, once the product is made, it still has to be quality tested, to ensure things like dosage is correct, and that there isn’t anything left that’s contaminating. And all this doesn’t even count the years and years of research and development that goes into getting a virus with the correct components to be effective at treatment but otherwise non damaging to a patient. Drug product patents have to be filed pretty much as soon as a compound is discovered, so well before clinical trials even begin. They’re often only good for 20-25 years. So, if you figure 7-10 years of base research and development, another 3-5 years of clinical trials, getting FDA approval, etc, on average a company only has 3-5 years to make back ALL the money they put into a new drug, plus some profit to keep the doors open, lights on and investors interested in funding the next project.
[deleted] t1_ixw400p wrote
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FlareHeart t1_ixw428e wrote
Tell me you didn't read the article without telling me you didn't read the article:
>...they also say that the drug’s effectiveness is expected to last for several years at least.
WulfricTheSwift t1_ixw44ym wrote
And here I thought this was to feed vampires at first lol. My apologies for lack of knowledge here. 😕
TheBearWillBeFine t1_ixw48vh wrote
It depends on the therapy. In this case, no, it’s not likely to target sperm or egg cells specifically. This sort of treatment is far more about reducing or eliminating symptoms in someone already affected than stopping transmission to offspring. That sort of discussion can get very dicey very quickly. The word “eugenics” comes to mind
[deleted] t1_ixw4ppl wrote
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IAMSHADOWBANKINGGUY t1_ixw4yyx wrote
Yeah I didn't. My bad. OP said it was, looks like they were mistaken then.
Although it looks like even the makers of the drug aren't sure.
TheLyz t1_ixw5vw4 wrote
The clotting factors they get infused with aren't much cheaper. One bleeding incident could result in needing $250,000 (probably more now, this was 10 years ago) of Factor-7 to stop it. It isn't cheap to be a hemophiliac.
velifer t1_ixw66p5 wrote
>It's a single dose,
That current evidence is showing will likely need to be re-administered every five years. That's not in their reports to the shareholders.
​
>manufactured in incredibly small amounts
They will make every single dose they can sell. This is not a supply issue. This is a patent and greed issue.
​
>From what I've gathered it looks like the price is intended to recoup research costs.
The price is set to maximize what an insurance company will pay. It's set a just slightly less than the price of clotting factors the patient will need if they don't use this therapy.
​
>That said, the burden to pay really should be covered or at the very least VERY heavily subsidized by the government.
The government already paid for it, much of the work was done with public dollars and grants.
picking_a_name_ t1_ixw8odn wrote
It's the cost of the research for this drug, plus paying for the fact that only 1 in a 1,000 drug potentials actually make it to market. So they recoup those costs too. Plus using the costs of other medicines you would have had to buy instead.
uski t1_ixwejj9 wrote
I think something is missing here... WHY did it cost so much? I think some people were gouging others and now everyone is like "meh, that's how much it costs"
itwasquiteawhileago t1_ixwemjx wrote
This is where orphan drugs come in. The government (US, anyway, but I'm assuming others have similar programs) can help provide some funding and allowances for private companies to work on drugs for conditions that have a low enough prevalence as to make it economically non-viable. Should someone that has a one in a million disease be left to suffer because Big Pharma can't turn a profit? This is a primary example of why and how governments should function: to ensure people aren't left behind. And, who knows what other things may spawn off such research, so it's not like everyone else might not benefit, too.
giceman715 t1_ixwevlr wrote
Praying their numbers are off the Venezuelan Bolivar currency
ComprehensiveBuyer65 t1_ixwg2pb wrote
It takes bio tech companies years and years to develop treatments and medications. It costs them billions of dollars: they have to pay many types of scientists, the equipment they use is very exacting and expensive. They use enormous amounts and variety’s of PPE. Then after all that they only have a set amount of years to recoup the money they spent making their drug/treatment before other companies can start making generic brands.
InformationHorder t1_ixwg8ei wrote
So we can get the generic brand for half, right?
Basquests t1_ixwgkks wrote
Haemophilia is an X linked disease. Ie its on your X chromosome. Women have 2Xs, men have XY. If a man gives a child his X, they have a daughter (mums always give Xs), if a Y, the child is XY (son).
Men are XY, so if they have a daughter, the daughter will definitely get a faulty X from dad. Theres one X, and its faulty wrt haemophilia. But most likely a healthy X from Mom. 1 healthy X is sufficient to be very normal. 2 bad Xs normally don't happen - pregnancy often doesn't reach term
My Mum's father had it , of his 4 kids, the 3 girls are all guaranteed to be carriers (1 good X 1 bad).
The brother of my grandpa is a man, got the Y from grandpa..i.e a haemophiliac male has no chance of giving it to his son. But all the daughter's are carriers.
So haemophilia ended for most of my wider family - my uncle and his kids can't have it, one auntie never married, 1 auntie was a carrier but had 1 daughter (almost 0% chance, but 50% of being a carrier...but she ended up not even being that), and 1 son (flip a coin on getting the good or bad X).
In my case, my mum had 2 sons...50% chance for each of us having it. We both do.
It's really not that pervasive as it skips a generation every time.
None of any kids i have, will have haemophilia. If i have only sons, Haemophilia ends for any of my future generations. If i have daughters, each will certainly be a carrier, but only have a 50% chance of making their own children a carrier (girl) or a haemophiliac (boy).
[deleted] t1_ixwlb7d wrote
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GuidoOfCanada t1_ixwmps7 wrote
This is really awesome, however my excitement is tempered by an awareness that this will likely bolster the monarchy in Europe... I feel like we should just let nature take its course for those folks
Terrorzwergi t1_ixwnjd4 wrote
> I work doing safety testing for drugs just like this. Setting aside all the money it takes to develop one of these viral gene therapy drugs, the cost to manufacture a single dose on a lot of them is somewhere around 50-90,000 dollars. The viruses need to be grown, then emptied of their regular genetic material, have the new, specially designed genetics put in, then replicated up to a level that can be infused into a person. Every single one of those steps requires highly skilled people to ensure it happens correctly. Growth of viruses need to happen in cell tissue culture, which also costs a lot for all the materials to do correctly, then all the supplies needed to purify out the virus from all the other crud in manufacturing. Then, once the product is made, it still has to be quality tested, to ensure things like dosage is correct, and that there isn’t anything left that’s contaminating. And all this doesn’t even count the years and years of research and development that goes into getting a virus with the correct components to be effective at treatment but otherwise non damaging to a patient. Drug product patents have to be filed pretty much as soon as a compound is discovered, so well before clinical trials even begin. They’re often only good for 20-25 years. So, if you figure 7-10 years of base research and development, another 3-5 years of clinical trials, getting FDA approval, etc, on average a company only has 3-5 years to make back ALL the money they put into a new drug, plus some profit to keep the doors open, lights on and investors interested in funding the next project.
Astavri t1_ixwrjyp wrote
Besides research or development, there is the manufacturing fixed costs. But research and development has their own fixed costs in process development to get it to the commercial level. Clinical trials are not cheap either.
Basically think of it this way. A machine is going to cost 100 million and it only makes 100 doses per year because that's how many people need it. This is just an abstract number to show the concept.
On the plus side, the alternatives are very expensive as well and you need to take them repeatedly, whereas this is a possibly a cure IIRC.
[deleted] t1_ixwshsd wrote
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[deleted] t1_ixwt8c3 wrote
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ClassyRedandGlassy t1_ixwyuk7 wrote
Like seriously? Uplifting news for who? The wealthy?? Yet again
[deleted] t1_ixwz7tj wrote
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Replicator666 t1_ixxulb8 wrote
Part of it is probably also that it is supposed to be a cure (like permanent after one dose)
There's something called humera (?) to treat hep c. I think one treatment which is a couple doses runs about 0.5 million
SilverNicktail t1_iy05irm wrote
People who can be cured of a rare genetic disease? "New cutting edge medical technology costs a bloody fortune" is so unsurprising to me as to be completely mundane. The improvement right now is that holy shit, we can cure hemophilia, and the improvement in future will be doing it better, more efficiently, cheaper. Plus, the cost of this treatment compared to all the other care they will require over their lifetimes if not treated - this might well be cheaper.
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