Slashdot Mirror
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.
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.
That's kinda like saying the Turing machine was invented at a public university on public dime. So why is Intel charging $350 for a processor.
A medicine to me is a chemical. This is a genetic treatment. A procedure. Cellular surgery. But not a medicine.
University research is literally just that ... the bare basics.
Thinking that's all that goes into drug production completely neglects to think about financing, building a production plant, clinical trials, government approval, zoning, regulation, packaging, distribution, etc, etc....
It's naive to think that because some phd in a university got something working using a benchtop setup and a few generations of lab mice, that we can just jump right on in to production batches that can be sold and administered to actual people.
If they were charging 350/eye I don't think anyone would be complaining. The problem is the drug industry has been raping the US lately. Have HEP-C, 85 grand, and then there was shekal(sp?) who raised priced on old drugs just because he could. Epi-pens also spiked and had no changes to justify the price surge. And then we have the drug industry that has totally abandoned antibiotics. Something almost all of us need from time to time. Just not profitable.
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.
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...
Leftists now misuse the term 'socialism' more than right wingers. News at 11...
John McAfee 'It was like that time I hired that Bangkok prostitute; to do my taxes, while I fucked my accountant'
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
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.
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.
> Not so much
Yes. Pretty much. Even the NIHs own paper on this subject indicates that the bulk of money spend on drug development comes from the private sector.
The money that the public spends only gets the ball rolling. It doesn't finish the process.
Plus this isn't your typical "one size fits" all pill kind of treatment. These kinds of treatments have to be custom made for each patient. The cost of that isn't trivial. It requires the employment of a large state of the art facility and staff that goes with.
Actual production costs are non-trivial here.
A Pirate and a Puritan look the same on a balance sheet.
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.
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.
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.
That's not how antibiotics work. Antibiotics merely make bacteria vulnerable to your auto-immune response which otherwise is unable to fight it off.
Antibiotics are like a pesticide. They will kill bacteria in a petri dish even without an immune system present. Yes, your body is still trying to kill them on their own so they are likely fighting side by side but antibiotics still work even in people with completely compromised immune systems.
Surgery used to be VERY risky before antibiotics due to secondary infection. It doesn't matter how strong your auto-immune system is, if you get a bacterial infection you're not going to get over it on your own.
Yes, surgery used to be risky because your immune system SOMETIMES couldn't fight off the infection on it's own but plenty of people back then and today have no problem getting over bacterial infections on their own. You are literally fighting off bacterial infections every day and in general your body does this just fine without antibiotics. Most people take antibiotics not because they can't fight the infection off themselves but because having your immune system working in tandem with antibiotics speeds up recovery. I once was in a place where I didn't have access to antibiotics and I got a very nasty staph infection. I generally would have went straight to the doctor and got antibiotics. Instead, I drained and cleaned it daily and although I felt like I was going to die and recovery took longer than usual, I recovered just fine. It's also been shown that antibiotics not only create super bugs, it also makes an individual's immune system lazy so that your immune system is not as good at fighting off infections on its own. Most people would be much better off letting their own immune system take care of it first. The recovery might take a little longer but your immune system is more than capable of killing bacteria and making you 100% well on its own 99% of the time. It's only in rare cases where your body gets overwhelmed that you absolutely need antibiotics. There are plenty of people who survive to adulthood without having once taken antibiotics.
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.
Most hearing aids are designed primarily around analog technology. The reason is latency -- digital audio in general, and bluetooth to a HUGE extent -- has WAY more latency than most lower-order analog designs. Latency screws up your ability to "locate" the source of a sound.
The cruel irony of digital audio is that the number of microseconds a digital filter needs to pre-buffer a sample is usually at least double the number of microseconds of lag that a comparable analog filter would introduce (often, 4x or more due to higher-order artifacts). If you're playing pre-recorded audio, it's no big deal... but for realtime signal processing, it's deal-breaking.
Antibiotics are like a pesticide. They will kill bacteria in a petri dish even without an immune system present.
Some antibiotics (like amoxicillin, cipro) work like that. Others, called bacteriostatic antibiotics (like erythromycin, tetracycline, linezolid), don't.
Bacteriostatic antibiotics temporarily halt bacterial replication, allowing the immune system to finish them off.
At least one child died because of the huge increase in the price of epi-pens.
There was no excuse to raise the prices. It took the free market close to a year and a half to recover and address the problem. That's a problem with barriers to entry vs the myth that the free market can handle everything. It can't.
Unlimited capitalism always leads to unlimited fraud. You have to have regulators in there. But then inevitably, the regulators are captured until something goes horrifically wrong and there is reform.
She was like chocolate when she drank... semi-sweet at first and then increasingly bitter.