FDA approves most expensive drug ever, a $3.5 million-per-dose gene therapy for hemophilia B

[ElNono]

FDA approves most expensive drug ever, a $3.5 million-per-dose gene therapy for hemophilia B

Washington — U.S. health regulators on Tuesday approved the first gene therapy for hemophilia, a $3.5 million one-time treatment for the blood-clotting disorder. The Food and Drug Administration cleared Hemgenix, an IV treatment for adults with hemophilia B, the less common form of the genetic disorder which primarily affects men.

Currently, patients receive frequent, expensive IVs of a protein that helps blood clot and prevent bleeding.

Drugmaker CSL Behring, based in Pennsylvania, announced the $3.5 million price tag shortly after the FDA approval, saying its drug would ultimately reduce health care costs because patients would have fewer bleeding incidents and need fewer clotting treatments.

According to a study cited by the National Library of Medicine, the price makes Hemgenix the most expensive medicine in the world, easily topping Novartis' Zolgensma gene therapy for spinal muscular atrophy (SMA), which costs right around $2 million per dose and is also a single-dose medicine.

https://www.cbsnews.com/news/fda-approves-hemgenix-most-expensive-drug-hemophilia-b/

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Probably $250/dose outside the US, tbh

[DMC]

between 30,000 – 33,000 people with hemophilia in the US. All races and ethnic groups are affected. Hemophilia B is four times less common than hemophilia A. -Google result

[ElNono]

between 30,000 – 33,000 people with hemophilia in the US. All races and ethnic groups are affected. Hemophilia B is four times less common than hemophilia A. -Google result

I doubt insurance will even cover this, tbh...

[DMC]

I doubt insurance will even cover this, tbh...

And no hemophilia B forgiveness from POTUS.

[ElNono]

And no hemophilia B forgiveness from POTUS.

Somebody will sue to have Medicare cover it for them... then we're all going to wonder how come we spend so much per capita in healthcare...

[DMC]

Somebody will sue to have Medicare cover it for them... then we're all going to wonder how come we spend so much per capita in healthcare...

At that total (30K) we could support them all for 30% or less of the proposed student loan bailout. But then you're going to have patients with other diseases crying foul because their medication isn't covered. The business minded solution is to attack it from a dilution of cost by having more people with the disease. That way the drug company can still make the same profit but the patient can see a lower per capita cost.

Ib4 pgardn takes that seriously.

[ElNono]

At that total (30K) we could support them all for 30% or less of the proposed student loan bailout. But then you're going to have patients with other diseases crying foul because their medication isn't covered. The business minded solution is to attack it from a dilution of cost by having more people with the disease. That way the drug company can still make the same profit but the patient can see a lower per capita cost.

Ib4 pgardn takes that seriously.

Well, it's not like if you fix the current people, it stops. 1 in 25k people are born with hemophilia B in the US. And basically, unlike people getting a loan, they really can't opt out of it.

[DMC]

Well, it's not like if you fix the current people, it stops. 1 in 25k people are born with hemophilia B in the US. And basically, unlike people getting a loan, they really can't opt out of it.

x-linked so 1 in 25K male births, which is roughly 1 in 50K births.

[Th'Pusher]

x-linked so 1 in 25K male births, which is roughly 1 in 50K births.

Wow! That totally changes the argument!

[Isitjustme?]

Wow! That totally changes the argument!

:lol "well actually" guy, well actuallying per/par

[Blake]

Wow! That totally changes the argument!

:lol

[DMC]

Wow! That totally changes the argument!

It changes the cost by half. What's the argument? Seemed like just a discussion.

[DMC]

Wow! That totally changes the argument!


:lol "well actually" guy, well actuallying per/par


:lol

Three uneducated people third party guffawing a medical discussion they don't understand :lol

[Blake]

Three uneducated people third party guffawing a medical discussion they don't understand :lol

:lol DMC pretending he's medically educated

[DMC]

:lol DMC pretending he's medically educated
This is you admitting you indeed don't understand :lol

[Blake]

between 30,000 – 33,000 people with hemophilia in the US. All races and ethnic groups are affected. Hemophilia B is four times less common than hemophilia A. -Google result

:lol

[Millennial_Messiah]

Somebody will sue to have Medicare cover it for them... then we're all going to wonder how come we spend so much per capita in healthcare...

BigPharma..... and Pfizer is at the top of the culprit list.

Also, private health insurance should be regulated big time.

[ElNono]

BigPharma..... and Pfizer is at the top of the culprit list.

Also, private health insurance should be regulated big time.

I prefer capitalism for almost everything, but healthcare should be one of those things where the government comes in and sets price caps (like most of the rest of the civilized world does).

[Millennial_Messiah]

I prefer capitalism for almost everything, but healthcare should be one of those things where the government comes in and sets price caps (like most of the rest of the civilized world does).
Healthcare yes, education no.

Health care falls under the "life" category of "life, liberty, and the pursuit of happiness". Health care is a right.

Education... is a privilege, not a right.

As per usual the USA has it bass-ackwards

[ElNono]

Healthcare yes, education no.

Health care falls under the "life" category of "life, liberty, and the pursuit of happiness". Health care is a right.

Education... is a privilege, not a right.

As per usual the USA has it bass-ackwards

I'd like to see more trade/vocational schools, tbh... it's just difficult since the scam we have going right now where the government backstops expensive loans is making too many schools too much money.

[Millennial_Messiah]

I'd like to see more trade/vocational schools, tbh... it's just difficult since the scam we have going right now where the government backstops expensive loans is making too many schools too much money.

yes, and the democrats don't want to bite the gnarly greedy hands that feed them. It's corruption.

[ElNono]

yes, and the democrats don't want to bite the gnarly greedy hands that feed them. It's corruption.

I mean, not sure why single out Democrats. After all, Bernie was the one that wanted to close that handout.

[DMC]

I mean, not sure why single out Democrats. After all, Bernie was the one that wanted to close that handout.

I don't really consider Bernie a true democrat, as made evident by how the DNC bent him over in 2016.

[DMC]

I'd like to see more trade/vocational schools, tbh... it's just difficult since the scam we have going right now where the government backstops expensive loans is making too many schools too much money.

I'd like to see trade schools along the border to help those who want to live in the US have a marketable skillset.

[ElNono]

I don't really consider Bernie a true democrat, as made evident by how the DNC bent him over in 2016.

Certainly a lot closer to Dems than GOP...


I'd like to see trade schools along the border to help those who want to live in the US have a marketable skillset.

If you're talking immigrants, those people have jobs. Normally the ones citizens don't want to do, despite not getting an education.

[Th'Pusher]

Three uneducated people third party guffawing a medical discussion they don't understand :lol

:lol medical discussion. This is about the cost of medical care. Do you think insurance is going to cover this @ $1.75M
Per dose? You try so hard to have a retort for everything.

[Millennial_Messiah]

Certainly a lot closer to Dems than GOP...


He's more of a populist than the Chuck Schumer team... he at least says he's against kickbacks and financial corruption and greed unlike the crony capitalist Democrat establishment base.

Heck Trumpism is left wing economics and far right wing socially.

[DMC]

Certainly a lot closer to Dems than GOP...

Different enough though that he's not a good representation of the democratic party.


If you're talking immigrants, those people have jobs. Normally the ones citizens don't want to do, despite not getting an education.
Pretty sure the thousands crossing borders don't have jobs. There would be no reason for an immigrant in San Antonio to travel to the border for training.

I am talking about skillsets, not just manual labor. How many border crossers do you think know how to hang drywall or tape and float it, do framing or electrical and plumbing work? Or drive a forklift or lay brick and mortar or do stone work or tile? How about actual roofing with understanding of code and safety instead of fetching shingles and materials?

If we're going to let people in (and we should) we should give them opportunity to have a marketable skillset. It helps them and the country at the same time. There's a huge deficit of certain skillsets in this country and it's only going to get worse as Americans born here refuse to touch any job that gets their hands dirty.

[DMC]

:lol medical discussion. This is about the cost of medical care. Do you think insurance is going to cover this @ $1.75M
Per dose? You try so hard to have a retort for everything.

:lol

[DMC]

Healthcare yes, education no.

Health care falls under the "life" category of "life, liberty, and the pursuit of happiness". Health care is a right.

Education... is a privilege, not a right.

As per usual the USA has it bass-ackwards

Education is a requirement and should be considered that by governments. Many successful societies know this already and build for it. Considering it a privilege instead of a requirement makes it luxury.

[ElNono]

Different enough though that he's not a good representation of the democratic party.

His voting record says otherwise.


Pretty sure the thousands crossing borders don't have jobs. There would be no reason for an immigrant in San Antonio to travel to the border for training.

I am talking about skillsets, not just manual labor. How many border crossers do you think know how to hang drywall or tape and float it, do framing or electrical and plumbing work? Or drive a forklift or lay brick and mortar or do stone work or tile? How about actual roofing with understanding of code and safety instead of fetching shingles and materials?

If we're going to let people in (and we should) we should give them opportunity to have a marketable skillset. It helps them and the country at the same time. There's a huge deficit of certain skillsets in this country and it's only going to get worse as Americans born here refuse to touch any job that gets their hands dirty.

I'm actually pretty sure thousands crossing the borders do have jobs (ie: seasonal migrants, which are a lot, people that have family here and can offer them a low wage job, etc) and are substantially better at the skillsets that you listed than the average American.

They're not coming here because they don't have skills, they come here because their countries are a political clusterfuck.

But to your point, why not Americans learning those skills? Is it that they don't want to do those jobs? There's still money in them.

[ElNono]

He's more of a populist than the Chuck Schumer team... he at least says he's against kickbacks and financial corruption and greed unlike the crony capitalist Democrat establishment base.

Heck Trumpism is left wing economics and far right wing socially.

But the point is you can't find a single GOP figurehead against that college loan crony capitalism. Singling out (D)s on this is disingenuous at best.

[pgardn]

The ability to “mend or fix” genes was destined to present all kinds of monetary and ethical quandaries.
This particular gene is not the first and it certainly won’t be the last Imo.
Once some of the technological problems are overcome and it becomes cheaper to “fix” a certain group of genes, there will be arguments on why even “fix” the problem. Ie the biological severity of not having a certain functional gene will come under subjective scrutiny.

Also, once we start to fiddle with regulatory genes, side effects will inevitably show up and other subjective decisions will be created.

[DMC]

His voting record says otherwise.



I'm actually pretty sure thousands crossing the borders do have jobs (ie: seasonal migrants, which are a lot, people that have family here and can offer them a low wage job, etc) and are substantially better at the skillsets that you listed than the average American.

They're not coming here because they don't have skills, they come here because their countries are a political clusterfuck.

But to your point, why not Americans learning those skills? Is it that they don't want to do those jobs? There's still money in them.
Americans won't walk from the corner to the store across the street to apply for a job. Some of these immigrants walk a thousand miles in hopes of having an opportunity to get a job. Americans, for the most part, have plenty opportunity and turn it down. This is why there are deficits in skilled labor positions.

Yes there is money in those jobs, lots of money. A good roofer can make 6 figures. Some of it is hard work but a lot of it is also skill based pay. If you know a good mason or painter or anything in the trades, you probably keep that person's information handy. You can go through too many ass clowns just to find one real tradesman. With so many crossing, the ass clown to tradesman ratio will get worse if there isn't a trades school for some of these people to attend.

My comments aren't altruistic. I would like to see better skillsets in the trades. I don't care where they come from.

[ElNono]

Americans won't walk from the corner to the store across the street to apply for a job. Some of these immigrants walk a thousand miles in hopes of having an opportunity to get a job. Americans, for the most part, have plenty opportunity and turn it down. This is why there are deficits in skilled labor positions.

Yes there is money in those jobs, lots of money. A good roofer can make 6 figures. Some of it is hard work but a lot of it is also skill based pay. If you know a good mason or painter or anything in the trades, you probably keep that person's information handy. You can go through too many ass clowns just to find one real tradesman. With so many crossing, the ass clown to tradesman ratio will get worse if there isn't a trades school for some of these people to attend.

My comments aren't altruistic. I would like to see better skillsets in the trades. I don't care where they come from.

Plus they're seemingly jobs that are always in demand and don't require big brains... heck, Andy could probably learn to do half of them...

[DMC]

Plus they're seemingly jobs that are always in demand and don't require big brains... heck, Andy could probably learn to do half of them...

Would you want Andy in your house? Could you imagine having Hobbit tracks on your hardwood floors, urine on your toilet seat and your cat's asshole stretched?

[ElNono]

Would you want Andy in your house? Could you imagine having Hobbit tracks on your hardwood floors, urine on your toilet seat and your cat's asshole stretched?

That's definitely a problem, tbh...

[Blake]

:lol medical discussion. This is about the cost of medical care. Do you think insurance is going to cover this @ $1.75M
Per dose? You try so hard to have a retort for everything.


:lol

:lol you bolded the prepositional phrase instead of the direct object. You're an idiot

[DMC]

:lol you bolded the prepositional phrase instead of the direct object. You're an idiot

Speaking of a stretched asshole :lol

[Blake]

Speaking of a stretched asshole :lol

See, this is ad hominem. Your capitulation is duly noted.