“The US has approved a blood clot therapy that costs $3.5million per dose — making it the most expensive drug in the world.

Hemgenix is a one-off intravenous treatment for hemophilia B, a disorder in which people do not produce a protein needed to create blood clots and control bleeding.

The standard treatment currently involves regular trips to hospital for rounds of IVs that replenish levels of the missing or faulty clotting factor.

But the new therapy delivers a gene that can produce the missing blood-clotting factors into the liver.

In trials it was shown to cut the number of bleeding events expected over the course of a year by over half. It also freed 94 per cent of patients from the need for regular infusions to control the condition. …”

DailyMail article:
https://mol.im/a/11466083

In unrelated news, health insurance premiums in 2023 hit a new high.

Just give hemophiliacs mRNA vaccines and they’ll clot up just fine… sorry, couldn’t resist.

Is this medicine aimed only for royalty?

quote:

Hemgenix is a one-off intravenous treatment

^^^^^^^
What is a one off treatment? Is this a one time treatment?? I will predict no one will pay that price. It is just a marketing move.

Bleeding edge technology is expensive; but I imagine like most technologies it will become cheaper overtime. If we can do gene therapy with a single IV treatment… Game changer.

I'm reminded of what I asked my dad when he complained about the cost of his angioplasty. What's your life worth to you?

quote:

Originally posted by architect:
In unrelated news, health insurance premiums in 2023 hit a new high.

2K more a year for us

quote:

2K more a year for us

^^^^^^^^^^^^^
Mine is much more. Consider yourself lucky.

I bet it's a factor of how much it costs to develop, get approved (can be a multiple ten figure expense), and produce. vs how many doses will likely be sold.

On a societal level, do we have to say we may be able to develop technologies that can save people, but it's just too expensive to do so?

quote:

Originally posted by Rey HRH:
Is this medicine aimed only for royalty?

The government has been know to subsidize some of the cost. I would also like to see the research on this particular drug.
Big Pharma plays pricing games with our medicines. A recent medication presribed for me was 830 dollars for thirty days. This is the generic med that has been on the market since 1998. I think we all would agree this sort of thing is ridiculous.

quote:

Originally posted by Gustofer:
I'm reminded of what I asked my dad when he complained about the cost of his angioplasty. What's your life worth to you?

My Father In Law's angioplasty was around $250K

Not the worst deal if it’s a cure. Doesn’t sound like that’s established. FWIW enzyme replacement therapies cost like $300K per year.

quote:

Originally posted by Flash-LB:

quote:

Originally posted by Gustofer:
I'm reminded of what I asked my dad when he complained about the cost of his angioplasty. What's your life worth to you?

My Father In Law's angioplasty was around $250K

So far, mine is up to $170k. Sounds like I got a deal

My X3 CABG in ‘98 was somewhere north of 140k. Two hospitals, bus ride, six day stay.

quote:

Originally posted by BBMW:
I bet it's a factor of how much it costs to develop, get approved (can be a multiple ten figure expense), and produce. vs how many doses will likely be sold.

On a societal level, do we have to say we may be able to develop technologies that can save people, but it's just too expensive to do so?

quote:

Originally posted by Rey HRH:
Is this medicine aimed only for royalty?

I'm going to bet you're wrong. My work was in pharmaceutical and medical device companies. Cost is way down the list of what drives pricing. Before a company even undertakes development of a drug or medical device, they determine the size of the market, their projected market share, and what price people will be willing to pay for what features. The market size, market share, and price point gives them their expected annual sales.

From the annual sales figure and sales per unit, they back out their target margin and indirect costs such as sales, marketing, factory overhead, etc. Then the question is can they make the product equal to or less than the remaining amount. If yes, then they go through other analyses such as break even points.

If it passes the criteria, then it competes with all the other proposals for funding and resource allocation. That's before anyone does any actual work towards development and manufacturing. This is stage 0 for any product in the companies I've worked for.

My quip about royalty is because the treatment is for some form of hemophilia and that disease is associated with European royalty.

Not to dispute the hereditary aspect of haemophilia as that is an indisputable fact, but I worked at fairly high level in conducting a significant number clinical trials (decades ago) of the spectrum of therapeutic blood and plasma fractions, some of which were and still are used to treat the terrible affliction of haemophilia. Additionally, I had a major role in the submission of newly developed and continuously improved special purpose fractions and technologies.

I left that business a year or two before the HIV struck the world's supply of of pooled plasma derived blood fractions used to treat haemophiliacs. Many hundreds of them innocently died in France alone from that contamination(circa 1981-82, IIRC). Subsequently adequate tests for the HIV were deployed and each unit of plasma became well tested for HIV before being 'pooled' with many thousands of other pints. Along the way many hundreds of thousands of litres of pooled plasma were dumped(vats full) due to contamination from a single contaminated contributed pint unit.

My employer of that decade, a huge international pharmaceutical corporation, employed many of the top scientists in the world working in this field. Amongst them I had the good fortune to work closely with one of the world's finest haematologists and I(not being a physician) once made a casual similar 'hereditary' remark while in his company only to be corrected. His comment was that a large fraction of haemophilia cases occur quite spontaneously with no evident hereditary linkage whatsoever. I do not have those numbers available however.