Posted on 01/30/2024 2:14:08 PM PST by nickcarraway
Libmeldy, the world’s most expensive drug which costs €2.8 million, will be available in Ireland, Belgium and the Netherlands.
Health services in the three countries have approved the groundbreaking gene therapy Atidarsagene autotemcel – sold as Libmeldy – Ireland’s Health minister Stephen Donnelly announced.
With a price tag of €2.8 million per one-off dose, the drug developed by Orchard Therapeutics treats metachromatic leukodystrophy (MLD), a rare and life-threatening inherited disease that affects the metabolic system in children.
Libmeldy took 20 years to develop, with a 10-year interval between the first human trials and approval from the European Medicines Agency which arrived in 2020.
Ireland, Belgium and the Netherlands have now negotiated a lower price through the Beneluxa initiative, which includes Luxembourg and Austria and was created to access medicines by talking directly to pharmaceutical companies.
The Beneluxa talks started in December 2022 but ended after initially failing to reach agreement with the manufacturer.
The final cost has not been disclosed but estimates vary from a reduction of between 25 and 65 per cent in some cases, according to Irish broadcaster RTE.
Three children in Ireland are due to be treated over a five-year period and will travel to Spain for the procedure, which would originally have cost around €10 million, RTE said.
“The drug will make a significant positive impact on the lives of the children with the condition,” Donnelly said.
“”have now negotiated a lower price””
From - To?
That’s a bargain, right? Don’t those three countries have the same healthcare system that the UK has? Wait - wait - and wait....?? It’s all incredible. I don’t think any pharmaceutical company in the world is going broke so why the gouge?
mRNA comes with a steep price tag — I say no.
20 years to develop before they made a dime, that’s why the high price tag.
...AND it a “rare” condition.
We are a billion into development and we sell 3 doses a year...lets see...mhmmm...carry the one...we should have our money back in...
Tried to find out where Orchard is and ran into this - dated 1/24/24. Saw on their website they started up in 2015 - so how could they have been working on a drug for 20 years? I didn’t find out where they were based....
Headquarters in London and Boston
Like BoT said, thats what Ive been thinking about.
Why would someone intentionally develop this? Maybe it was an accident on the way to inventing something else?
It doesnt make sense under usual circumstances.
When WHO accidentally and absolutely was not threatening everyone with Marburg we were having this conversation. Marburg, terrible thing, we should “just cure it.” Well why doesnt anyone just do that? Because there are only a small number of cases, often in difficult to get to places so the patient is dead shortly after anyone knows, and then who is going to pay for it? Dead people with no insurance cant pay for doses to reimburse development.
So why would any drug company intentionally put their time and money in this drug?
“”Orchard was founded in 2015—but our roots run deeper, going back to some of the first research and clinical development involving hematopoietic stem cell, or HSC, gene therapy.””
Roots run deeper - meaning what?
...of course it is.
Did you happen to watch the series “The Expanse”?
This whole story we are living in seems like someone developed the proto-molecule and Im aware of it but Im not important enough of a character to be able to do anything about.
That coupled with the fact that it is a rare disease, I suspect is the main reason. All drugs take a long to to develop & then test. But being a rare disease means that it will not be in great demand.
“”Did you happen to watch the series “The Expanse”?””
No, I didn’t - haven’t heard of it. For a minute, I thought you meant “Fringe.” LOL As a matter of fact, I think we’re living in “Fringe” now...Crazy times!
“”But being a rare disease means that it will not be in great demand.””
Then wouldn’t that cause someone/anyone to say - “we can’t do it - it would be too expensive”!!!!!
Or was the thought - we’ll get governments like the NIH to pay for it?
Who pays? Is it like liver transplants to ghetto rats? Everyone who has worked pays via taxes and increased healthcare costs and the recipients get it free? Or are the recipients only those children whose relatives can come up with the money?
That’s a valid question, and one would assume that many said it, but as to why this company decided to do it is anyone’s guess. Speculation runs the gambit but the only one that can answer that is the owner of the company that decided to develop Like you, I have no clue it. If the article hadn’t stated that it was a rare disease, I wouldn’t have even known that fact. 😋
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