Here is an NPR article on this.
This seems like a really big deal. The obvious downside is the cost but hopefully that will come down over time.
this post was submitted on 09 Dec 2023
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From what I saw elsewhere, the cost of the CRISPR treatment is roughly 2 million dollars and another way to implement the cure is via a modified flu virus. That version is roughly 3 million.
If only we knew what the real costs of treatment are, not the bullshit prices the industry decides they'll say it is and then negotiate a barely more realistic real cost with insurance companies.
Guess we'll have to wait until this is approved in other countries for a real answer.
Guess we'll have to wait until this is approved in other countries for a real answer.
Hard to know the price in other countries when it's free, eh?
Just because they don't issue a bill doesn't mean they don't track costs. They track labor, labor rates, and consumables.
That said, this particular treatment is very involved. They harvest cells over multiple periods, send them to a lab to be modified, and when they are ready they do chemotherapy to kill your immune system, then do a bone marrow transplant to introduce the modified cells, and then you have to be in isolation in a hospital until your immune system comes back. Even the best facilities are saying they can only do 5-10 of these per year.
Pretty crazy.
It's also wild that the first step of the treatment is chemotherapy.
It's actually not expensive just because. They don't manufacture this stuff in a pill packing plant with an automated machine that just churns this out.
Cell therapy takes blood from a patient and manufacturers with it to make the drug. It's made manually by a team of people for a specific patient. The material costs alone are a quarter of the price in most cases.
Cell therapy ain't cheap.
It's tricky because the money, time and opportunity cost gone into development, testing and the approval process are also priced into this. Plus the fact that this needs to not only break even but make some money plus the fact that this won't be relevant for a huge market I think (not sure how prevalent SCD is). So it's an outrageous price but probably not just plucked out of thin air
This is why pharma research should be publicly funded, and the results go directly into public domain. We will save so much money and lives in the long run that way.
A LOT of pharma research gets significant public funding. They then patent it and privatize the profits. Then spending millions on advertising.
Then they try and justify pricing from the total cost of not only development, but also advertising budgets, while avoiding any mention of where the actual development funding came from in the first place.
That's not for everything, but it's a large enough number of drugs and treatments that the entire industry is based on bullshit.
The bigger one is to decouple development from manufacturing.
Development should be done on a bounty type system. Both countries and individual groups can put money into bounties.
Once the bounty is claimed, then the drug is effectively free for all to produce. This lets us leverage capitalism to push prices down.
This would reshape drug development from max money, to most needed.
I like this - but would companies that fail (in being second) not get credit for their work? You could imagine the second place actually having a more effective product at the end.
You don't have a yes/no payout. You have a graded payout. E.g. you might have a 1 shot cure pay out the full amount, but a sustained treatment only pay a smaller %. This lets you encourage development of the most effective treatment, not the most profitable. It's currently better to make a condition chronic, and so need treatment for a lifetime, than develop a cure.
You also don't pay out all at once. By spreading it out over sat 10 years. It means it can be adjusted if the company's claims are... less than accurate.
wow this is huge for anyone with sickle cell that lives in a country that has universal healthcare. Other countries I guess it's great for super rich people.
I wonder why it's so expensive.
Emergent tech always starts out very expensive.
Consider that the phone you probably posted this on is significantly more powerful than the first computers, which were several orders of magnitude larger and more expensive.
Yeah, great points. Economies of scale have not kicked in, and I'm sure there are plenty of inefficiencies with the technologies they're currently using.
It would be neat to know precisely what parts are expensive. Broad generalizations about how prices on goods go up and down aren’t quite as interesting. 😅
CliniMACS plus/prodigy, LOVO, G-Rrex and other expensive instruments.
Grade B suites with grade A BSC's and all the trained personnel to work on it.
Shit costs a lot of money.
Manufacturing costs alone are insane. I work in biopharma, specifically manufacturing.
Most of these drugs you see on headlines are made from the blood of the patient. You can't mass produce that.
You can't mass produce that.
Challenge accepted.
I'm wondering how such a treatment will be handled in countries with a high malaria prevalence
There's a new, more effective malaria vaccine coming out right now:
Sweet
This is the best summary I could come up with:
The Food and Drug Administration on Friday approved a powerful treatment for sickle cell disease, a devastating illness that affects more than 100,000 Americans, the majority of whom are Black.
In April 2021, Morning joined the clinical trial at Sarah Cannon Research Institute and HCA Healthcare’s The Children’s Hospital at TriStar Centennial in Nashville, Tennessee, a decision she initially regretted.
Dr. Haydar Frangoul, medical director of pediatric hematology-oncology for the Sarah Cannon Research Institute, said he is hopeful the therapy will provide relief to more patients.
Dr. Nicole Verdun, director of the Office of Therapeutic Products in the FDA's Center for Biologics Evaluation and Research, said Lyfgenia was given the warning after two patients who got the therapy in a clinical trial died from a form of leukemia.
It's unclear whether the gene therapy itself or another part of the treatment process, such as the chemotherapy, caused the cancer, but Verdun said the agency thought the deaths "rose to the level of a black-box warning."
Christopher Vega, 31, from Allentown, Pennsylvania, said the possibility of long-term effects aren’t a concern for him; he is happy to be living a life free of chronic pain.
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