A gene therapy to treat a rare, inherited form of blindness will cost $850K
washingtonpost.com
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This drug will only work on, let's say 1500 people. Of those, let's optimistically say that 1/3 have insurance that will cover expensive treatment. So 500 patients can be expected to be treated by this drug per generation.
So this drug will optimistically get a revenue of $500 million per 2 decades or so, or $25 million per year.
The number generally thrown around for getting a new drug researched and onto the market is $200 million. So it will take 8 years just to break even (although likely front-loaded once it becomes known that this drug exists), and total revenue for this drug for the forseeable future is about 500 million.
Does this seem spectacularly unreasonable ?
Like everything I guess it depends on your political views. Personally I think it's fine if medical care and research is a massive black hole of funds that the tax payer foots the bill for...
I don't see where the gain for society is there. Not everyone can be saved/helped, and medicines/drugs are probably too greatly overestimated now in my opinion.
Also it's sad but some organisms are just not fit for some tasks (or sometimes even for life), and I don't think throwing money without any supervision or afterthought about it is a very wise answer as a society. Like, maybe we can just have blind people.
I don’t know what the solution to medical funding should be. It’s clear to me that the current situation (medicine for profit) does not work. It’s also clear that the current economic system is prone to waste and inefficiencies.
For all I know, maybe people should be able to get a grant for certain situations. The normal economic forces would then work to incentivize the grant holders to exchange grant funds for goods/services. The funds would still funnel the way they currently do, but grant holders would have gained some measurable benefit along the way. Presumably the grant holders would have made their choices based on perceived needs. (Ie if they have 2 chronic illnesses but only one is life threatening then the life threatening illness will be allocated most resources.) this is just a thought experiment.
Edit- another idea: change the funding for the DHSS to include funding for basic medical research and treatment with a set % of the GDP. I’m thinking that ratio should be based on disease rates.
I'm not advocating for unlimited healthcare funding - just against expecting healthcare to fund itself.
If you care about saving lives, you want to allocate budget in the most cost-effective way, which means putting money where a marginal life saved costs the least.
So do you want the extreme rare condition that some important congresscritter's daughter has consume 80% of the medical budget ? This is the way to go.
> So do you want the extreme rare condition that some important congresscritter's daughter has consume 80% of the medical budget ? This is the way to go.
People would scream bloody murder if that happened.
As futile as it would be, before I had a child, I thought that in the event of a end-of-humanity catastrophe, I would get as close to the source as possible and admire the end. Now with a kid, I am going to hold the fort has long as possible, cook my own hand and serve it, just it case this isn't the end.
Doesn't seem to stand up to scrutiny, e.g. http://blogs.sciencemag.org/pipeline/archives/2017/10/18/dru... quotes $2bn as a more reasoned number.
It would just really suck to be one of those 1000 that have gene and can't afford the drug.
No they wouldn’t. They’d have the knowledge that there’s a cure and they can’t afford it. I can’t imagine how psychologically damaging that would be.
Probably very little, especially with reasonable hope that the treatment will become affordable over the next years. Also, it changes the situation from an impossible one to merely a practical problem of convincing other people to fund your treatment.
Regardless, curing some people and giving hope to others is strictly better than not curing anyone at all.
Now if we can only remember that detail when it inevitably makes the newspapers.
The comparison could be what the reduction in lifetime earnings due to being blind vs the $850k.
But I think that having a utilitarian view toward delivering healthcare is frequently unethical once you start rationing care (eg, I’m 75 and not working at all, living off social security, $300k heart surgery is detrimental to society’s bottom line since if I die I stop collecting social security, etc etc)
This is what so many people fear about the possibility of single payer in the US. Eventually, people will start to be pressured to see their duty to die for the benefit of society.
There's a reason why a 97 year old with terminal cancer isn't going to receive a liver transplant ahead of a 12 year old with no other health issues.
Under the ACA, we have the "Independent Payment Advisory Board". They decide which treatments will and won't be covered.
Private insurance has the equivalent. Most insurance companies don't cover "Experimental" treatments.
I'm unfamiliar with the details of the systems in Europe but they all have to have a body with a similar function. No system could survive with a policy of paying all of the money you want for every treatment you can find.
This means that dialysis (very very basic treatment, but if you need it, you'll die without) ends somewhere in your 70s. For instance if you have a compromised liver. When they stop it, you will die in a matter of months at best, in some cases painfully.
What I don't get is why there isn't more noise about this in the Netherlands. After all, this system is so generic that you can of course make the argument that something like 30% or so of people will be confronted with a "no" that will end their life.
True. As noted, in our current system, it's just the insurance companies making the call instead of a bureaucrat. And it's usually done at a general level, not individually (except for rare, expensive, edge cases).
But, politicians in the US who oppose change would have us believe death panels are going to single out Grandma as not worthy of care.
Lots of fear-mongering about this.
If a private entity sets their criteria for treatment in a way that people don't like, it's possible to switch to a different insurer.
When the government does, people are just stuck.
Canadians end up going to the US if they want more than what the public system offers.
Also, I don't see how the current private and public insurance systems in the US would prevent insurers or government to decide, in the future, that certain groups of people do not deserve treatments or covereage anymore.
http://www.chop.edu/news/children-congenital-blindness-can-s...
How much of the number is:
1) due to BS (as opposed to useful, e.g. for safety) bureaucracy
2) padding from the pharmaceutical, just because they can?
The virus bit might be hard to do, and maybe editing the header of it. I'm not sure. I guess that probably is quite hard.
But manufacturing DNA and injecting it is fairly trivial, as in this guy with a PhD and a small lab was able to do it for about $400. There are biotech companies who will just make the DNA for you. https://www.theguardian.com/science/2017/dec/24/josiah-zayne... ... this was using CRISPR which is like a DNA find/replace, so in some ways more sophisticated than the way Luxturna works.
I'm not saying anything about the price of this treatment per se, obviously a load of R+D and regulatory ballet had to be done here, but just saying the basic building blocks of some gene therapies are quite a bit cheaper than I imagined.
e.g. https://freethoughtblogs.com/pharyngula/2017/10/14/i-could-u...
Still I'm not going to defend Zayner, he probably is a bit of a dick.
But the point is that you can modify the DNA of at least a small number of your own cells in a rough untargeted way for about $400. That's quite surprising. Makes you wonder what might be going on behind closed doors elsewhere.
edit: Here's Zayner's write up of his stunt: http://www.ifyoudontknownowyaknow.com/2017/10/the-first-huma... (possibly dodgy ads, adblock recommended)
https://en.m.wikipedia.org/wiki/Voretigene_neparvovec
It’s a gene therapy delivered in a viral vector. They inject it (into your eye which much be fun). It appears to be one of the first approved gene therapies.
Not necessarily. For example, consider that neurons in our auditory cortex aren't different from neurons in our visual cortex!
What is different about them (and the "subsequent" perceptual/conscious level) is that they receive different inputs, and have learned the systematicity of how these inputs behave in relation to the body's own actions.
This is related to the "Sensorimotor Contigency" theory of consciousness [1], which would predict that it should in fact be possible to process entirely different types of sensory input, given long enough exposure and a clear systematicity of the input signal.
[1] http://www.scholarpedia.org/article/Sensorimotor_theory_of_c...
https://www.wired.com/2007/04/esp/
and it seems the experiment has been updated:
https://www.theguardian.com/technology/2017/jan/06/first-hum...
“As we walk down the street there’s radiation, X-rays, infrared and ultraviolet, as well as the electromagnetic field of the planet. So we want to create new senses to become aware of our environment.”
and
Harbisson, who is colourblind, has an ‘eyeborg’ that allows him to “hear” the light spectrum (including infrared and ultraviolet), while Ribas has a sensor in her elbow that vibrates when an earthquake occurs anywhere in the world.
It just doesn’t seem to be commercially viable (colorblind-ness therapy was never persued because risk/reward/commercial aspects don’t work out IIRC).
I suppose, more generally, even in the absence of an $850k fix, if you have genetic blindness, is it unethical to have children?
Looking at the last decades, it's no longer unreasonable to think we might cure things like blindness or deafness, or paralysis, in the next 100 years. But that can only happen if our technological civilization survives uninterrupted. If it spirals out of control and self-destructs, we'll be stuck in a pre-industrial age for thousands (if not hundreds of thousands) of years, until the Earth replenishes some of the easily accessible high-density energy sources we pretty much entirely mined out.
The part where it doesn't: AAVs do not integrate their DNA into the host genome, not by themselves at least. They form latent infections, which will persist until the cell divides or die.
However, the part where they could: AAVs can infect any type of cells, including germline cells. While they persist, if the cell becomes infected with an Adeno or Herpes virus, which can also infect a broad range of cells, then they could integrate into the host genome, including germline cells.
I can’t imagine the price I’d put on a sense; nice to know you can literally get half of the price for a single eye treated.
It is their corporate presentation; lots of data on the drug starting around slide 10. these are data from the clinical studies required for FDA approval. Investor presentations (for public companies) tend to have good summaries of the key data on a drug; for more info, you can read scientific publications on the drug in academic journals
Besides, there's nothing humanitarian about spending other peoples' money. That requires no humanitarianism, just political access to taxpayer coffers.
The best way for more medical innovation like this is for it to be expensive at first. Over time, competition and demand drives prices down. Pretty much all the medical technology we now take for granted was only accessible to the rich at first.
That's a bold assertion, and you haven't supported it with an argument.
What about diseases of poverty in the third world? Is the best way of getting medical innovation here by making the cures expensive? The trouble with this kind of thinking is that it (a) conflates the value of an intervention with the price it can command, which is most problematic when it (b) requires large upfront payments and cannot efficiently aggregate or amortize costs over time.
Here's some food for thought for a different approach: prizes. If there were funds made available to pay a prize for working cures, drugs, etc. for medical problems, it could replace patents as a reward mechanism and permit market behaviour to increase the efficiency via generics and widespread application directly, rather than needing exclusivity and high prices for a long period. See e.g. https://en.wikipedia.org/wiki/Prizes_as_an_alternative_to_pa...
> What about diseases of poverty in the third world?
To be clear, I'm talking about new medical innovation, not the proliferation of existing medical solutions which obviously can be paid for by governments and nonprofits without harming the development of new methods. That said, there are quite a few 'last mile' solutions getting medicine to poor parts of the world that organizations like the Gates Foundation have identified.[5]
> Here's some food for thought for a different approach: prizes
I like the prize method as a way of incentivizing innovation--and it should certainly be tested. But I would be hesitant to endorse it as a replacement for the patent system unless it was proven to work more effectively in creating more cost effective innovations. It could, of course, be tested by allocating a percentage of the NCI's budget for prizes. But I think you'll find that the appetite for that sort of risk is sorely wanting on the governmental level, as it should be. We want investors to be taking these risks with their own money (and get rewarded when they pay off), not gambling with our money and losing it when they don't. Remember that, like startups, only a small fraction of drug ventures make it to the market and we only hear about the ones that do.
[1]http://www.nytimes.com/1989/08/28/opinion/azt-s-inhuman-cost... [2]https://www.healthline.com/health/hiv-aids/cost-of-treatment [3]https://www.forbes.com/sites/larahoffmans/2012/03/23/they-un... [4]https://www.lasik.com/category/cost-of-lasik/ [5]https://www.youtube.com/watch?v=GlUS6KE67Vs
Really? Do you know how expensive supporting braille is? Accessibility requirements for software? Fitting a blind person's house out with accessible features? Providing public services and transit for the blind? Income support?
All of these things are costs absorbed by society, which you cover one way or another (e.g. taxes, additional costs in products & services etc.).
Being blind is a major disability, the lifetime cost to society of a single blind person is many multiples of 425k per eye.
> The best way for more medical innovation like this is for it to be expensive at first. Over time, competition and demand drives prices down. Pretty much all the medical technology we now take for granted was only accessible to the rich at first.
What?? What if there are no rich blind people competing for this care? How is the cost going to come down then?
Plus, it's not as if every person will even get the procedure if it is free. There are complications. There are religious objections. There are personal preferences.
Higher prices don't come down because rich people buy them. They come down because it creates incentives for new innovators to produce cheaper and more efficient methods.
That argument aside, I am of the opinion that all healthcare should (eventually) be free so that arguments like these become moot.
The conversation is often about how do some EU countries have free healthcare and US doesn't. The answer is higher taxes, longer waiting lists, lower quality, no support for extreme cases and low accountability.
"Free healthcare" countries often cannot afford these modern cutting edge treatments so people often end up leaving for US to pay for the treatment themselves. If US didn't have such a brutal healthcare system then who would do the research since the "free healthcare" countries cannot afford it?
Of course the alternative is to leave people with health conditions out on the street and just ignore them, which does seem to be a common occurrence in the US
“The demand for healthcare is effectively infinite.”
There really is no limit to a person’s healthcare needs. It’s a moving target. The only way to make all healthcare free would be to start by defining exactly what is and isn’t healthcare. That’s exactly what is upsetting about the concept of government provided services. At some point someone will have to say “enough.”
Who would decide that, and how?
Currently experienced three health systems: Polish, US and German; first is woefully underfunded and public, second is ultra-private, third is a properly funded public system.
In Poland, I used both public and private, using private whenever I could (think dentist, blood test etc.); in US I never went to the doctor because I had to pay extra; in Germany I feel comfortable and happy with the fact that I get public healthcare for my taxes. Actually planning a string of visits to the dentist ;)
"a partial refund if the drug does not work in the first three months"
So is it $850,000 every four years ?
The disease effects a small number of people (1 in 4000), and doesn’t cause complete blindness in all those effected.
It’s delivered in a viral vector, kind of not so easy to do yourselves I’d guess. Probably the easiest way would be to get hold of some and culture the virus yourself? Then just make as much as you want.
Still injecting something made without strict QC into your eye might not be so appealing.
You want to be putting enterprise-grade things into your eye. The only way you'd be able to guarantee that is to source the treatment through traditional channels.
The kit looks fun, but I don’t think it as (easily) be used to replicate this work, it’s just too different.
The issues mentioned in the article also apply.
I remember an old psychological test where you were presented with a scenario where a member of your family is sick and while there exists a drug that can cure them, you cannot afford it. You're given the option to break into the local pharmacy and steal the drug- and asked whether you would do it. The way you answer the question is an indication of your moral development stage. If I remember correctly, the idea is that the most morally developed individuals will decide that stealing the drug is not moral, that if everyone stole what they needed without regard to the interests of the original proprietor, there would be anarchy and society would collapse. So on the whole, the moral decision is to not steal the drug, even when your relative needs it.
Well, if society allows a life-saving drug (or, indeed, a blindness-curing treatment) to be unaffordable to those who need it, then that's already a society that is not serving its members' interests. What the hell do I care if society can support science advanced enough that it can cure blindness, if it won't cure my blindness? I'm expected to do the moral thing and sit nicely and not rock the boat- but others are not expected to help me, in my need? Bugger that for a game of soldiers!
A society that leaves people blind when it can cure them is just as useless as one that burns. So let it burn.
I'd steal that drug in a heartbeat.
Oh btw- in another article about this miracle cure, I read the ridiculous claim that blindness will cost a person about $1million in their lifetime, so the $850k price tag is justified. I don't see this claim in this article, but it just goes to show what some may consider "fairness": "blindness robbed you of $1million, now we're gonna take another".
It's always like this and it's perfectly natural part of the process. You will not get the benefit of the newest invention today. But you, and many others like you, are interested, and so people will work to bring the costs down, and in 5, 10, 20 years, suddenly you can afford it.
As heartless as it may sound - suck it up, wait for your turn. It will come.
> Well, if society allows a life-saving drug (or, indeed, a blindness-curing treatment) to be unaffordable to those who need it, then that's already a society that is not serving its members' interests. What the hell do I care if society can support science advanced enough that it can cure blindness, if it won't cure my blindness? I'm expected to do the moral thing and sit nicely and not rock the boat- but others are not expected to help me, in my need? Bugger that for a game of soldiers!
It's a fucking fresh discovery. Have a little patience!
Stealing a drug in your example is not a moral thing to do because if everyone did that, there would be no drug, and no pharmacies. Unfortunately, in this world we're constrained by limits of physics and economics (i.e. basically how good individuals are at coordinating to do something together on large scales). Things take time.
Also, frankly, you yourself have contributed exactly nothing to the development of this drug, so I don't see why you should blame society for not having it available to you immediately. Blame God, if you believe in one, for creating a shitty universe in which blindness and death are a thing, and in which we're left alone to solve them to the best of our capacity.
And then be thankful for the good people who will make sure this drug will, eventually, get to you.
Well, I'm not blind but I'm revolted at the idea that a blind person should wait for a quarter of their life to go by, just so somebody can recoup the costs of research that should have been supported by public funds in the first place.
>> Also, frankly, you yourself have contributed exactly nothing to the development of this drug, so I don't see why you should blame society for not having it available to you immediately.
The people who developed that drug were able to do it because they grew up in a society that allowed them to become scientists, by freely sharing the work contributed by others and the knowledge discovered by others (because science). And that, on top of every other benefit of living in a human society and not having to invent and construct every bit of science, technology and infrastructure from the ground up, on their own.
That is the secret sauce of how societies work: solidarity. If you take this away, you take everything else away with it too, including the benefits of having science and technological progress in the first place.
But, you say- all the technology and science will become free in the long run. Just wait. Well, some people are blind now. Why let them wait, when we can cure them already?
And if the blind men can wait patiently for 20 years to see the light, why can't the sellers of the drug wait their turn also? Let's set up a fund to collect $850k for each blind person who is cured, thanks to this new treatment. And let the creators of the drug get that money in twenty years from now. They can wait patiently too, can't they?
No, that was the "generic you".
> That is the secret sauce of how societies work: solidarity. If you take this away, you take everything else away with it too, including the benefits of having science and technological progress in the first place.
No. That's how families work. Sometimes.
Society at large works on trade. You don't get your shoes from the shoemaker for free, because he knows you need them and feels solidarity with his fellow human being. You trade money for the shoes, so that he can trade this money with a farmer for food, in order to eat. This same thing scales up to every human need, including developing drugs.
Don't get me wrong - I wish societies were built on solidarity. Things would be so much better. But in the real world, they aren't - and this is the world we have to deal with.
(INB4 open source - this is an exception which happens because there's essentially no marginal cost to software beyond time, and barriers of entry are low; pretty much nothing else works like this on larger scales.)
> But, you say- all the technology and science will become free in the long run. Just wait. Well, some people are blind now. Why let them wait, when we can cure them already?
Because we can't cure them yet without breaking other things. It costs too much to do it now, but we have a reasonable expectation that the price will drop, so it makes sense to wait a bit longer. It's kind of like asking why the country won't put 100% of its GDP towards healthcare. It technically could, but it would destroy itself in the process.
> Let's set up a fund to collect $850k for each blind person who is cured, thanks to this new treatment. And let the creators of the drug get that money in twenty years from now. They can wait patiently too, can't they?
That's... an interesting thought and maybe someone should bring it up with the company. Still, someone will have to bankroll that $850k x number of relevant blind people, and that someone may say it might be worth waiting a year or ten, so that the price is $85k per person.
(Also, in a way, this is how credit system works.)
OK. Sorry if I sounded a bit rude, above.
>> No. That's how families work. Sometimes.
That trade exists and people exchange goods and services for money, does not mean there is no solidarity, or that there can be none. People -in capitalist economies- give to charity. They help each other with personal problems. When there is a natural catastrophe, they help each other. Public money is spent to help those in need, even if they themselves cannot contribut to the communal pot (e.g. with disability allowances and unemployment benefits). Public health and public education are also prime examples of this.
I am prefectly aware that these benefits that come with simply being born into a given society are not universal. For example, I know that in the US, neither healthcare nor (higher) education are free. However, these are examples of erosion of solidarity, that have been strongly criticised and should be strongly criticised.
>> Because we can't cure them yet without breaking other things.
I don't see how this is the case. Where should public money be better spent than in healthcare?
Also, btw, I'm really not convinced by the claim that "good things come to those who wait" (in this case anyway). There are plenty of examples of drug firms resisting the release of generic, cheap, alternatives to their proprietary, expensive drugs. There is Martin Shkrelli, who bought Daraprim, first in 1953, and hiked its price from $13.50 to $750 (per tablet). There is the Mylan, who hiked the price of EpiPen from $94, in 2007, to $608, in 2016 (Epinephrine was first approved for marketing in 1987).
There are also examples of technologies becoming cheaper, but I'm particularly interested in medical technologies, not computers. I don't care if computer become cheaper and available to everybody. People can live without computers. They can't live without, well, without drugs that keep them alive.
> I don't see how this is the case. Where should public money be better spent than in healthcare?
R&D to make healthcare cheaper? It's an exploration/exploitation problem.
Also, policing, education, administration, infrastructure, defense.
Healthcare is a practically infinite money sink, so there have to be limits somewhere. Especially that beyond some point, it becomes cheaper to save an additional life through spending on e.g. policing than healthcare.
Don't get me wrong - I'm not saying everything is perfect in the way our society allocates resources - just that it's a bit too much to condemn it entirely for not providing a quality-of-life improvements (blindness is tough, but not life-threatening) for free, to everyone, just as they come out of the lab.
That would be a start. Public healthcare, in the European model, would be ideal. It's true that it's a huge money sink, but that can be managed somewhat- by cost/benefit calculations, which sounds harsh and inhumane but is I think is justifiable.
Which is fine by me, to be perfectly clear. If people can access the drugs they need, then fine, let the companies make a profit, why not. I mean, within limits and all.
The Heinz Dilemma is not evaluated based on your decision but (as you correctly note in passing) based on your reasoning.
for those who can afford it. The rest can go get stuffed.
Originally, computers were huge, laughably simple, and could only be afforded by governments. Thanks to the investments of those governments, then rich corporations - computers improved exponentially while costs decreased exponentially. The rest weren't getting "stuffed". The rest are the beneficiaries of progress predicated by those initial computers.
So on the whole, the moral decision is to not steal the drug, even when your relative needs it.
This is a good example of where you have the ethical lesson at your fingertips.
then that's already a society that is not serving its members' interests
But then in the next sentence you throw that lesson away without solving the problem that the reasoning solved in the first place. The lesson is that you can't view the ethics of a situation in isolation. You have to consider the wider consequences of destroying the incentive for some to use their free time to research drugs and create inventions.
I'm sorry, but the incentive is right there: curing a person's blindness. Curing cancer. Curing HIV. Helping people live longer, healthier, happier, more productive lives. These are worthy goals and most scientists will at the very least recognise them as such. Most anyone will recognise them as such.
But you're talking about monetary incentives. And that's the higher moral ground, is it?
The price tag isn't so high because someone wants to get really rich off other people's suffering. It's so high, because to develop a drug, we need to employ ridiculous amounts of resources of our civilization, in labour, knowledge and material.
Until we hit post-scarcity, where all the basic needs can be fulfilled pretty much for free, money will be driving things. Those are the constraints under which we work today.
Of course, people need to pay their bills. And I need to get the life-saving drug that's too expensive for me to buy. So, if it's OK for you to price that life-saving drug so high that I can't afford it, because you have bills to pay, then it's certainly OK for me to steal it from you, because I have a life to live.
If you don't help me to stay alive (or to cure my blindness etc), why should I help you pay your bills?
But ultimately, it doesn't generalize well. Society will forgive you a lot in emergencies, but if a behaviour like this becomes common, it really would threaten the ability to provide drugs at all, and it would end up condemning to death much more people in the near future.
So is that what you spend all your time on? If not, why not?
I was a little sad for my plumber friend, but he isn't. He said, that at first this was a DYI build-you-own-house kind of thing, so customers he never had. Now, he is dealing with people who bought those houses, and even if it is quick and easy to fix, they don't want to or don't know how to deal with that. In addition, it takes him no time at all to do those repairs he can actually do more jobs during a day. So he is not losing any income, or sleep.
And in this case, does it boil do to publishing the dna fragment? Is this another 'patenting a number' discussion?
Isn't this a given?. Which product are you buying that costs the same as cost " to create and deliver"