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Price Tag On Gene Therapy For Rare Form of Blindness: $850K (apnews.com)
A first-of-its kind genetic treatment for blindness will cost $850,000, less than the $1 million price tag that had been expected, but still among the most expensive medicines in the world. Several readers have shared an Associated Press report: Spark Therapeutics said Wednesday it decided on the lower price for Luxturna (Lux-turn-a) after hearing concerns from health insurers about their ability to cover the injectable treatment. Consternation over skyrocketing drug prices, especially in the U.S., has led to intense scrutiny from patients, Congress, insurers and hospitals. "We wanted to balance the value and the affordability concerns with a responsible price that would ensure access to patients," said CEO Jeffrey Marrazzo, in an interview with The Associated Press. Luxturna is still significantly more expensive than nearly every other medicine on the global market, including two other gene therapies approved earlier last year in the U.S. Approved last month, Luxturna, is the nation's first gene therapy for an inherited disease. It can improve the vision of those with a rare form of blindness that is estimated to affect just a few thousand people in the U.S. Luxturna is an injection -- one for each eye -- that replaces a defective gene in the retina, tissue at the back of the eye that converts light into electric signals that produce vision. The therapy will cost $425,000 per injection.
the basic research that underpinned this (e.g. all the hard work) was done at public University on the public dime.
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You'll poke your eye out with that thing
Dude, if CRISPR can done in a home garage lab, you can bet your ass that people will flying to Asia to get this done on the cheap. Now granted, that's a big risk. But...this tech is getting cheaper, and where ever there's red tape, there's nothing a passport and a flight ticket can't fix.
Life is not for the lazy.
See, evil greedy capitalists made a treatment for a rare disease that costs money and is therefore evil!
If we just socialized everything then there wouldn't be a treatment for the disease at all and it would be free! Free dammit!
A medicine to me is a chemical. This is a genetic treatment. A procedure. Cellular surgery. But not a medicine.
Don't worry.... You won't see it coming.
"If you want to improve, be content to be thought foolish and stupid." - Epictetus
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A gene therapy for a disease that has a pool of potential patients in the thousands costs nearly seven figures? Absolutely shocking! If we can put a man on the moon, surely we can defeat the law of supply and demand!
But ironically, if it works, it makes far more sense than a lot of the emotion-driven spending we do via public healthcare programs and private insurance such as spend hundreds of thousands on treating quite possibly terminal disease in people past their gender's life expectancy.
How about another example? We treat funding cancers that mainly impact retirement age women as the highest cancer priorities, but people doing childhood cancer treatment practically have to sell drugs and do bake sales to get any real funding. Fuck you 6 year old sally, we can't have your parent's 65 year old neighbor die of breast cancer because she's a voter and you're not.
I can't see treatments like this coming to market without huge cash dispensing machines like public or private insurance. The market for those needing treatment is just too small to offset the price of R&D.
Perhaps a better financial approach would be for our public institutions to offer a large bounty for cures like this. (And a substantially lower bounty for remedies that are periodic continuously taken treatments.) Then the private market can respond with a price point that makes having medical insurance irrelevant.
Yeah an' an' zombies!
That was the turning point of my life--I went from negative zero to positive zero.
You'd be guessing wrong, more or less. The company has been developing this drug since October 2007, ten years ago. Their 2016 annual report shows they spend about $86 million / year on internal R&D, mostly for this drug in recent years. That's "e.g. all the hard work".
They also booked $10 million in external R&D for this drug in 2016, but that number is going to get bigger. External R&D is the company paying the university (Penn) for the research the school did over ten years ago. Now that the drug has been approved and it's going on the market, the company will have to pay the school another $3.8 million plus about 5% royalty on all sales. 1,000 patients at $850,000 is $850 million. 5% of that is $42 million. So the school will get about $42 million royalty, plus the $3.8 million base, plus the millions they've already received. Figure the school may have spent $200,00-$500,000 on the initial research, they are doing extremely well. Something like $300K spent on research will net the school about $60 million.
http://ir.sparktx.com/static-f...
There used to be this idea of a 'fair' price, but one of my greatest criticisms of the Republican way of thinking is that a 'fair price' is the same as 'the maximum the market will allow'.
I think a better measure would be something like ( and I suspect these number can be determined fairly well).
I've kicked around some kind of consumer reporting law that would at least make real data available on a given product.
Something like
( Total number of expected units sold * ( cost of physical materials involved + manufacturing costs)) + cost of product research and development + cost of marketing + corporate overhead) = projected production cost.
(Total number of expected units sold * sale price ) - projected production cost = total return on investment.
Honestly if a company isn't already doing this kind of math before they start on a product they are likely to go broke.
A person might even consider a law saying something like more then a 200% return on investment is considered unfair, but at least if it is reported people can have the discussion.
I know the typical objection is ( but what about all the failed products they also research , but there is no reason you can't build that into the cost by make cost of research = total research cost / successful number of products to get an average cost for research across the company.
âoeTolerance applies only to persons, but never to truth. Intolerance applies only to truth, but never to persons.
It is very convenient to forget that we, the people, know have a choice which we did not have in the past. All those people who think that it is a very expensive medicine, forget that if not for the scientists and entire product teams, this product would not exist at all. It is somewhat comparable to the 16 and 17 century... Only very affluent people had access to buy glasses to improve their vision. Lens were very very expensive. Nevertheless, advancements in optics allowed creation of multiple products that do contain lens. Using the logic of today's people that innovations are expensive, consider the world where optical properties were never analysed out of the fear that optical products will be expensive.
change master price? what is the real price for this aka the cost that the health insurance will cover?
So how do we fix this?
We have readers who are experts on economics, law, medicine, and game theory(*). What's the solution to this?
There are rare diseases that affect only a handful of people in the US, and there are tons of medical procedures and devices which could be used but aren't.
Two anecdotes: a) I talked to a doctor at Berman-Gund (Boston) who claims to have a cure for a rare inherited disease that affects only 450 people in the US, but has given up because it's too expensive to develop(**). b) My dentist (heavily involved in research at Tufts) mentioned that there's lots of new diagnostic methods available, but insurance companies won't allow them because they're afraid they will turn up undiscovered conditions that are expensive to treat. Essentially, it's cheaper (in the actuarial sense) to let things go until they are untreatable so that the patient dies quickly.
A) What are the characteristics of a system that fixes these problems, and
B) How do we get from where we are to that system?
(*) For a situation with an incentive for better health.
(**) Meaning: With only 450 potential patients, there is no potential profit and no one is willing to pay for development, trials, and certification
If you have a strong immune system, you should never need antibiotics.
Apropos of nothing, where can I get one of those?
This is very informative, thank you. Given those figures, 850k looks depressingly like breakeven for the company (if that 86mil/yr was all for this, it would take them (860mil+10mil+3.8mil)/0.95 = 919.79 mil revenue to break even, or about 920k per for 1k patients). I'm sure they have some profit baked in to the figure, but not as egregious as the summary headline sounds by itself.
Or we could do like other countries do: let the government pay for the research and the treatment.
I don't respond to AC's.
I know it's fun to hate on $INDUSTRY, but lets get down to the technology here, the science, and what is possible today that wasn't possible five years ago. We can argue about the costs later, it fundamentally doesn't matter. No single person will pay $850K for this treatment, it'll be insurance, charity, etc. What has been developed is a virus that introduces DNA into specific regions of the body. It's really mind-blowing! Think of the possibilities this opens into other treatments. We may be able to adapt the science heal lungs in those born with cystic fibrosis, repair hearts of children born with cardiomyopathy, cure Type I/II diabetes, etc. Besides, if Toyota had to build only as many cars, as patients received this treatment, those would cost a million bucks too! The price seems reasonable for the amount of work that was surely required.
But if it blinds him, he may be able to get it cured for one low price of $850K!
the vast majority of pharmaceutical research is done with public funds
I see this come up a lot but I've had a hard time verifying that this is actually the case. Although the public portion is significant, from what I can tell it's not even a majority:
https://www.ncbi.nlm.nih.gov/b...
"In 2004, federal agencies funded roughly one-third of all U.S. biomedical R and D (Moses et al. 2005). "
"Private sector drug, biotechnology, and medical device companies provide the majority of U.S. biomedical R and D funding (about 58 percent)."
If you post as Anonymous Coward, don't expect a reply.
And all we can find to do is bitch about the price? Maslow is indeed a harsh mistress.
This is amazing stuff, folks. If you want more of it, leave the profit motive in place. If you want less of it, do the opposite.
Had one, not fun.
A few years ago I had a bad retinal detachment that was treated first with a gas bubble injected into my eye to push the torn flap back into place, followed up by laser treatments to sorta weld the retina back into place.
Sitting in the chair with the eye surgeon coming at me with a large needle and saying "Don't move"...not fun. Fortunately they did put some numbing drops in the eye first, but I could still see it coming (with the other eye).
> I see this come up a lot but I've had a hard time verifying that this is actually the case.
Of course you did. It's just bullshit "media narrative". These people are like old women passing chain letters amongst themselves.
As much as they whine, you would think that these people had all been denied coverage for very expensive treatments for rare conditions.
When it's actually your butt on the line, it's much more important that these things exist at all. Frankly, I am amazed that the economic incentives actually work out for people with rare diseases.
A Pirate and a Puritan look the same on a balance sheet.
Virtually every new drug, device, or treatment will eventually have a class-action lawsuit that will cost the company billions. Gotta factor that into the equation too. And the class action may be for minor effects, such as the one currently in vogue for a breast-cancer treatment that caused permanent hair loss or for slightly increased risk of suffering from the thing treatment was supposed to support (increased risk of depression among users of anti-depressant drugs).
Which government is that exactly? Name ONE drug or gene therapy treatment that can be had for peanuts in another "socialist" country because the government actually did all the work.
Things don't work like that even in Europe.
It turns out that people don't work for free.
Also, governments are notoriously stingy and taxpayers everywhere complain that they pay too much.
A Pirate and a Puritan look the same on a balance sheet.
I hate to break it to you (really I don't) but there are already common medical procedures that bill out at MORE than this procedure.
Tens of thousands of people see bills like this every year. Considering the alternative, I doubt if it distresses them too much.
A Pirate and a Puritan look the same on a balance sheet.
So, let's look at the equation:
Cost of being blind:
- Limited job opportunities.
- Need for 'translation' of reading materials, screens etc. etc.
- Depression (a very real factor)
- Danger while traveling/navigating
With vision:
- Significant potential for increased income.
- Minimal need of additional social resources.
- Increased safety while traveling/navigating
Net result: Increase ability to earn and, therefore pay taxes.
If the cost were a mortgage, the payout could be in about 30 yrs. 30 Yrs. of productive income, lifestyle and freedom.
It's expensive, true, but, on balance, worth it.
*** Don't be dull.***
I wouldn't be surprised if even at $850k, the cost of this treatment is less than a lifetime of assistive support for blindness. Hopefully those that qualify for this can get it covered.
Or we could do like other countries do: let the government pay for the research and the treatment.
By all means! After all, these "other countries" just magically shit money out of nowhere. It's not like the citizens pay taxes or anything, right?
Oh, wait, they do pay taxes! Which means perfectly healthy people are forced to pay for R&D and treatment of diseases they don't have, won't get, and more or less can never benefit from. Oh, sure, it's all noble to say you're helping your fellow man, but isn't that what charitable contributions are for? By golly, yes, that's exactly what they're for! What an amazing concept! People voluntarily helping others instead of the government taking their money and giving it to whoever they feel is most worthy of getting it. And of course such dispensations are never, ever politically motivated and the government never, ever wastes money through fraud, graft, inefficiency, or laziness, right?
In the end they will lay their freedom at our feet and say to us, Make us your slaves, but feed us. - Fyodor Dostoyevsky
You say this:
I am in this field and there is no reason it cant cost less than $500.
And then you say this:
It costs $2 billion to push a sugar pill through the FDA clinical trials process.
You are aware these two things are related, right? The R&D and approval costs are baked into the final price of the drug. The whole "reason" it has to cost more the $500 right now and for the foreseeable future is those costs have to be covered. If not, there is no money put back into R&D and approval for the next medical breakthrough and healthcare research essentially stops.
In the end they will lay their freedom at our feet and say to us, Make us your slaves, but feed us. - Fyodor Dostoyevsky
Don't forget however, that every dollar they spend in figuring out this sort of treatment can be recouped later when the technology is perfected and they get FDA permission to start treating other more common diseases. This is proof of concept and easing the idea of messing with our DNA into a world that's still dodgy over GMO foods.
there is no potential profit and no one is willing to pay for development, trials, and certification
If time and resources were infinite this wouldn't be an issue. Neither are, hence no matter how noble it would be to "fix" certain things, hard choices occasionally need to be made. Do you spend tens of millions researching a drug that will benefit, say, 500 people or that same money on a drug that will benefit 50 million people?
There are many problems with this view, but I'll focus on the most obvious one.
There is no way for a patient to "exit" this system in order to take control of their situation. They cannot go to the doctor and sign a waiver saying they know the risks but want to do it anyway - that's illegal.
They are condemned to a lifetime of no hope and no recourse. It would be simple and inexpensive to give them a legal way out of the medical system we have now, so that they could at least try things on their own under the supervision of a real doctor.
There's also the humanitarian angle - we're actually *not* researching drugs that benefit 50 million people medically, we're researching boner drugs and hair-growth drugs, and drugs that mask symptoms long-term but don't actually cure anything. Because *those* drugs can affect 50 million people for profit, while curing actual diseases doesn't.
The company may well lose money. The $86 million internal R&D was for 2016 only. They've been working on this treatment for ten years. I know they also had $10 million external R&D for this treatment in 2016; I don't exactly know how much of the $86 million was for this, but it looks like they had four "promising" ones that would account for most of it. So maybe $25 million internal and $10 million external on this treatment on 2016.
How much was spent on *this* one doesn't much matter, though, because most medications aren't approved. They need to R&D many in order to find one that works well, is safe, and gets approved. If they spend $100 million looking at 8 possible treatments and one of those gets approved and generates $80 million revenue, they've lost $20 million overall.
Overall, large pharmaceutical companies made an average 3.7% return on their R&D investment in 2016 and 3.2% in 2017 (Deloitte). Small firms do better on average, but also have a higher chance of bankruptcy if they don't score a hit.
For this specific case, it looks like the method of virus delivery and CRISPR gene modification can be used for a whole host of diseases. This just happens to be the first application.
"We wanted to balance the value and the affordability concerns with a responsible price that would ensure access to patients," said CEO Jeffrey Marrazzo
That's comedy gold, right there!
Unless, of course, you happen to have this form of blindness without being a multimillionaire. Then, it's just an insult.
Well, the question at hand is, are they $850k (including enough margin to survive) non-trivial?
If they are, fine. If not... then there's plenty of room for looking askance at the pricing.
I've fallen off your lawn, and I can't get up.
https://science.slashdot.org/s...
The University of Pennsylvania is a private university.
The company has been working on this for ten years. I looked at a breakdown of the R&D numbers for one of those ten years and found they spend $86 million on internal R&D and $20 million on external. So figure the total R&D cost alone to find and develop this treatment and move it through approvals is maybe $800 million. That doesn't include the carrying cost of the $800 million over ten years, administrative costs, etc.
If 1,000 patients get it, the treatment brings in $850 million retail minus roughly $80 million in corporate subsidies they've announced, minus distribution so maybe $750 million or so. They'll lose roughly $100 million on the US market, but hopefully make that up in Europe. It depends on how many of the 2,000 or so Europeans affected by the disease get the treatment.
The first computers cost millions of dollars.
This is not a mature technology, people. It starts expensive.
This is very informative, thank you. Given those figures, 850k looks depressingly like breakeven for the company (if that 86mil/yr was all for this, it would take them (860mil+10mil+3.8mil)/0.95 = 919.79 mil revenue to break even, or about 920k per for 1k patients). I'm sure they have some profit baked in to the figure, but not as egregious as the summary headline sounds by itself.
Don't forget that only about 1 in 10 drugs pass all three phases of clinical trials, so for companies to get funding they would need to be reasonably sure they could return at least 5-10x the cost of development. So if it takes a billion dollars to break even, they would probably be expecting in the ballpark of $5-$10 billion in profits from this drug (and from licensing of patents related to the research). Too much less than that and it wouldn't make sense to fund the drug research in the first place.
-- All that is necessary for the triumph of evil is that good men do nothing. -- Edmund Burke
.nt
She was like chocolate when she drank... semi-sweet at first and then increasingly bitter.
Yep, it will probably wind up like futurama in the end.
I like to tell people about the 'peel your pupil' part of having my eyes lasered. Gets a reaction.
John McAfee 'It was like that time I hired that Bangkok prostitute; to do my taxes, while I fucked my accountant'
Read a book about the history medicine. Most of modern medicine has been developed by academics on government payroll. In "socialist" Europe, you indeed usually pay nothing as a patient (or a very small co-pay).
Of course, modern drug development is now usually based on commercial companies, this doesn't mean that it couldn't be done directly by the government. In fact, government institutions still do develop a lot. You asked for an example. Here is a relatively recent and important one: NIH researcher developed the hepatitis A vaccine
I doubt this is true. A very large part of the costs of bringing a medical product to market comes from seeking regulatory approval - this is on the order of hundreds of millions of dollars to approve a single drug for a single use, regardless if it'll go on to treat more than 100 million people or less than 100. It takes billions of dollars worth of dedicated capital to get these approvals and pharma companies conveniently have just that - it's almost like it's a barrier to entry that was created by the FDA to protect these companies from competition...
Also understand these universities make out very well from this whole arrangement. In my experience, the universities that develop these drugs are the ones that actually hold the patents and the pharma companies only license them, paying the institution royalties in return. This means the university doesn't have to risk investing in further trials that might fail, while also recouping at least some of the cost of their research even if it does.
Even if we assume this expensive approval process is necessary for patient safety rather than being yet another example of revolving door corporatism, you'd still want such agreements as universities simply are not geared to bring a compound from the lab to the open market. This is called specialization; leave the science to the scientists and industrial production to the industrialists.
Frankly, I think as long as your doctor is sure you understand the risks involved and it is in your best interest, you should be allowed to buy a drug without FDA approval. This would make treatments for rare diseases like this far cheaper and more available as trial data could be collected over time without such a massive outlay. It would also enable smaller companies to take on promising compounds for rare conditions that have been passed over by big pharma. Obviously this means significantly more risk to the patient, but that risk may be worth it to someone with blindness or a terminal illness, and it should be up to them and them alone to make that subjective determination.
But this would never happen as it'd result in more competition for big pharma and less quid pro quo for those in the FDA.
A couple thousand people in the US have the disease. I guesstimated that half of those might pay full price for the treatment (via their insurance company). The pharmacuetical company has already announced their own subsidies, so some will get the treatment at less than full cost. Some won't be good candidates for whatever reason, and some may decide to stay away from this genetic therapy stuff for now.
Which were originally developed to treat high blood pressure and prostate enlargement. (They got used for other things when some of the side effects were noticed.)
Did you know 80 to 90% of the moderators on slashdot wouldn't recognize a troll even if one dragged them under a bridge.
the scientists don't really care who pays them. They're doing the research because they love it. We only need the profit motive in place because we want it there.
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the scientists don't really care who pays them.
Hey, it's funny -- I was just reading an article that explained how the government-funded NSA is bleeding talent because they can make a lot more in the private sector. And there was this dude named rsilvergun who said:
these guys can clear $500k/yr working for Wallstreet. It's no wonder they don't want to settle for $140k/yr working for Uncle Sam.
I know, I know... consistency is the hobgoblin of little minds. But still.
Requiem for the American Dream
Same as 30 European countries though this will be going down to 29 next year. That market is somewhat larger than the US one.
For having my eye sight restored, yeah, I'll fly to India. Whatever chilled concoction is required can stay there.
You know, some of the best tailored made suits are made in HK. Many people will actually schedule a flight to HK just to have one made and purchased.
Life is not for the lazy.
Dude, if CRISPR can done in a home garage lab, you can bet your ass that people will flying to Asia to get this done on the cheap. Now granted, that's a big risk. But...this tech is getting cheaper, and where ever there's red tape, there's nothing a passport and a flight ticket can't fix.
Its not the red tape, but the private sector getting in the way. If you want surgery in Thailand, you'll get a western trained doctor in a hospital that has western levels of service.
The reason Asian nations like Thailand are so much cheaper than many more developed countries is because they compulsorily license many things that the private medical industry hold patients over a barrel for (the holding over a barrel is to allow easier access to your pockets) via the patent and legal systems.
Calling someone a "hater" only means you can not rationally rebut their argument.
I used a pediatric cancer patient as an example. In my example, she was 6 years old. There is no "democracy" out there where a 6 year old can vote. It is the moral imperative of her elders to represent her interests. In modern democracies, there is absolutely epic fail there as the elders are now increasingly in a vampiric mindset of "I'll extract the most resources I can to ensure I get the lifestyle I want."
I'm not going to argue for a duty to die, but the 65 year old voters should have the mentality associated with the Titanic: the children take priority over everyone in getting them to safety. One of the reasons Boomers sometimes get flabbergasted that I don't respect my elders is precisely that so many of them see their interests as of equal importance with that of the youngest generation (Gen Z, not Millennial; I am Millennial). As a Millennial father of two "Gen Z" kids, I take strong exception to my elders wanting respect and consideration when so many of them cannot even consider the traditional sacrifices the elderly have made to protect their grandkids and grandkids' generation as a whole.
Maybe you're in medicine overall, but there's no way you're in gene therapy if you think you can make enough clinical-grade vector for human eyes with $500. It costs my lab several thousand dollars for enough research-grade vector to do a basic mouse experiment.
Examine even your most deeply held beliefs. Nobody is always right.
Previous price: infinity. Not available at any price.
The trend will continue, no doubt. Though that first price decrease will be tough to beat.
There's no time like the present. Well, the past used to be.
In a true communist society it would cost you... nothing. Because a great society cares about everybody. It's not based on who is the most psychopathic=wins.
"It is no measure of health to be well adjusted to a profoundly sick society." - Jiddu Krishnamurti