10 ms·

> Only 1,000 to 2,000 people in the United States are thought to have deteriorating vision caused by this errant gene This drug will only work on, let's say 1

> Only 1,000 to 2,000 people in the United States are thought to have deteriorating vision caused by this errant gene 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 ?

superquest 9y ago
No, it doesn't. It would just really suck to be one of those 1000 that have gene and can't afford the drug.
- yazaddaruvala 9y ago
  While not ideal, the drug/delivery procedure will be reverse engineered in a matter of months, and these 1000 people could get on a plane and get almost the same procedure in India/China for $8,500 instead.
- flukus 9y ago
  When does the gene kick in? Most government funded healthcare would probably see this as worthwhile if it affects people young enough, it's cheaper for the country to pay for the treatment and have a productive citizen than it is to have them on welfare for their entire life. If no one is going blind until late in life then the equation changes though.
  - prepend 9y ago
    This is an interesting line of thought. Blind people are not disabled though, they can have careers. It’s a false assumption that blind = lifetime welfare. I’ve worked with awesome programmers who were completely blind from birth. And quite a few who were legally blind. 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)
    - LordKano 9y ago
      >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.
      - maxerickson 9y ago
        Which is bizarre, because we already publicly fund a huge amount of end of life care.
        alistairSH 9y ago
        And because other nations have socialized health care and don't have "death panels." And I'm not aware of any plan that would preclude a person from purchasing their own insurance to cover any care deemed of insufficient value by the national system.
        Amygaz 9y ago
        And these nations even have strong debates about even allowing people the right to choose when to die. Morality is a big part of adopting universal healthcare. 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.
        LordKano 9y ago
        Every system does. They're not called "death panels" but that's the function. 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.
  - msochor 9y ago
    One of the first patients cured in the research of this drug was a child: http://www.chop.edu/news/children-congenital-blindness-can-see-after-gene-therapy http://www.chop.edu/news/children-congenital-blindness-can-s...
- c3534l 9y ago
  They would be in the same position as they were before, only without that system it wouldn't be worth researching the drug to begin with.
  - okasaki 9y ago
    Likewise if the company gave the therapy for free to everyone who couldn't afford to pay $850k, they would be in the same position as they were before.
    - TomMarius 9y ago
      No, everyone would try to get it for free and there is a chance that the poor people become rich.
    - imcoconut 9y ago
      Before the reaearch endeavor, the company had x more dollars in cash. They bore the cost with the expectation that it would generate cash flows in the future (to hopefully more than offset the cost). The dollar cost was likely quite large. So the company certainly would not be in the same position had they not decided to develop the drug. There was no cost the affected genetically blind, however. They only now have the option of potentially being able to get a new treatment.
  - woofyman 9y ago
    >They would be in the same position as they were before 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.
    - TeMPOraL 9y ago
      > 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.
sjg007 9y ago
Medicaid will pay as blindness is a qualifying disability. It’s a second payer though which is why some people will go through insurance first.
- jopsen 9y ago
  Will Medicaid be around much longer? Given the upcoming deficit created by the tax "reform"..
  - sjg007 9y ago
    Yes, most likely. It is also a 50:50 funded partnership between the States and Feds. Also as we've seen things can change quickly in politics.
  - hackeraccount 9y ago
    Look, if you want to be snide then it's just as easy to say that Medicaid won't be around because expanding it to non-poor people will cause it to go broke. Either that or the desire to cut health care costs from both sides of the spectrum will cause the program to go broke.
doesnt_know 9y ago
> 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...
- hycaria 9y ago
  Why ?!
  - ianai 9y ago
    Revenue seeks after profit. There are many diseases too rare to be feasible to research and develop on a cost/benefit revenue basis.
    - hycaria 9y ago
      So we should create a black hole of funds for a few people ? 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.
      - tincholio 9y ago
        I wonder if you'd think the same if it was your kid (or you yourself) in that position. It's easy to take the pragmatic asshole position when it's not your skin in the game. It boggles the mind that people think like this in this day and age.
        hycaria 9y ago
        It's been published a few times over the past few days that healthcare professionnals often have lower expectancies towards medicine especially during end of life etc. So I guess I would actually think the same indeed.
PeterStuer 9y ago
Your whole reasoning is based on marble counting. A better question is were the people capable of doing the research working on the right problem, or should their talents have been applied to different problems? Once there is a 'cure', of course it should be handed out to everyone that can benefit from it.
coldtea 9y ago
>The number generally thrown around for getting a new drug researched and onto the market is $200 million. 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?
andygates 9y ago
It only accounts for patients in the US. Expand that market (or, if it's regulatorily fiddly, pursue those patients).
adrianN 9y ago
For a drug like this that uses a completely novel technique, gene therapy, I'd say that the R&D investment can also help in the discovery of other drugs. So you'd have to adjust the figures a bit.
zimpenfish 9y ago
> $200 million Doesn't seem to stand up to scrutiny, e.g. http://blogs.sciencemag.org/pipeline/archives/2017/10/18/drug-development-costs-revisited http://blogs.sciencemag.org/pipeline/archives/2017/10/18/dru... quotes $2bn as a more reasoned number.
- Amygaz 9y ago
  Except that 1st. Sparks' stuff only cost a fraction of that to develop (closer to the $200M), and 2nd lot of the early expenses were fronted with tax payer's money.
  - sitkack 9y ago
    And it my understanding that, soon gene therapy will be selecting which bits to flip and flipping them. It still needs testing, feedback, etc, but it isn't the same as going through a full development cycle. Am I wrong?
conanbatt 9y ago
Whats unreasonable are the research costs. I would look into that.
hackeraccount 9y ago
As is reported down thread it might be reasonable or not but some of the assumptions are open to question. It's not unreasonable to suspect that Medicaid will cover the benefit. Also The number of people would just include the U.S. presumably the drug would be sold world wide.
- testvox 9y ago
  But the rest of the world doesn't recognize FDA approval (and the FDA doesn't recognize any other country's approval process). So the cost of certification has to be paid again for every country its sold in.

superquest 9y ago

No, it doesn't. It would just really suck to be one of those 1000 that have gene and can't afford the drug.

yazaddaruvala 9y ago
While not ideal, the drug/delivery procedure will be reverse engineered in a matter of months, and these 1000 people could get on a plane and get almost the same procedure in India/China for $8,500 instead.
flukus 9y ago
When does the gene kick in? Most government funded healthcare would probably see this as worthwhile if it affects people young enough, it's cheaper for the country to pay for the treatment and have a productive citizen than it is to have them on welfare for their entire life. If no one is going blind until late in life then the equation changes though.
- prepend 9y ago
  This is an interesting line of thought. Blind people are not disabled though, they can have careers. It’s a false assumption that blind = lifetime welfare. I’ve worked with awesome programmers who were completely blind from birth. And quite a few who were legally blind. 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)
  - LordKano 9y ago
    >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.
    - maxerickson 9y ago
      Which is bizarre, because we already publicly fund a huge amount of end of life care.
      - alistairSH 9y ago
        And because other nations have socialized health care and don't have "death panels." And I'm not aware of any plan that would preclude a person from purchasing their own insurance to cover any care deemed of insufficient value by the national system.
        Amygaz 9y ago
        And these nations even have strong debates about even allowing people the right to choose when to die. Morality is a big part of adopting universal healthcare. 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.
        LordKano 9y ago
        Every system does. They're not called "death panels" but that's the function. 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.
- msochor 9y ago
  One of the first patients cured in the research of this drug was a child: http://www.chop.edu/news/children-congenital-blindness-can-see-after-gene-therapy http://www.chop.edu/news/children-congenital-blindness-can-s...
c3534l 9y ago
They would be in the same position as they were before, only without that system it wouldn't be worth researching the drug to begin with.
- okasaki 9y ago
  Likewise if the company gave the therapy for free to everyone who couldn't afford to pay $850k, they would be in the same position as they were before.
  - TomMarius 9y ago
    No, everyone would try to get it for free and there is a chance that the poor people become rich.
  - imcoconut 9y ago
    Before the reaearch endeavor, the company had x more dollars in cash. They bore the cost with the expectation that it would generate cash flows in the future (to hopefully more than offset the cost). The dollar cost was likely quite large. So the company certainly would not be in the same position had they not decided to develop the drug. There was no cost the affected genetically blind, however. They only now have the option of potentially being able to get a new treatment.
- woofyman 9y ago
  >They would be in the same position as they were before 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.
  - TeMPOraL 9y ago
    > 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.

sjg007 9y ago

Medicaid will pay as blindness is a qualifying disability. It’s a second payer though which is why some people will go through insurance first.

jopsen 9y ago
Will Medicaid be around much longer? Given the upcoming deficit created by the tax "reform"..
- sjg007 9y ago
  Yes, most likely. It is also a 50:50 funded partnership between the States and Feds. Also as we've seen things can change quickly in politics.
- hackeraccount 9y ago
  Look, if you want to be snide then it's just as easy to say that Medicaid won't be around because expanding it to non-poor people will cause it to go broke. Either that or the desire to cut health care costs from both sides of the spectrum will cause the program to go broke.

doesnt_know 9y ago

> 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...

hycaria 9y ago
Why ?!
- ianai 9y ago
  Revenue seeks after profit. There are many diseases too rare to be feasible to research and develop on a cost/benefit revenue basis.
  - hycaria 9y ago
    So we should create a black hole of funds for a few people ? 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.
    - tincholio 9y ago
      I wonder if you'd think the same if it was your kid (or you yourself) in that position. It's easy to take the pragmatic asshole position when it's not your skin in the game. It boggles the mind that people think like this in this day and age.
      - hycaria 9y ago
        It's been published a few times over the past few days that healthcare professionnals often have lower expectancies towards medicine especially during end of life etc. So I guess I would actually think the same indeed.

PeterStuer 9y ago

Your whole reasoning is based on marble counting. A better question is were the people capable of doing the research working on the right problem, or should their talents have been applied to different problems? Once there is a 'cure', of course it should be handed out to everyone that can benefit from it.

coldtea 9y ago

>The number generally thrown around for getting a new drug researched and onto the market is $200 million. 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?

andygates 9y ago

It only accounts for patients in the US. Expand that market (or, if it's regulatorily fiddly, pursue those patients).

adrianN 9y ago

For a drug like this that uses a completely novel technique, gene therapy, I'd say that the R&D investment can also help in the discovery of other drugs. So you'd have to adjust the figures a bit.

zimpenfish 9y ago

> $200 million Doesn't seem to stand up to scrutiny, e.g. http://blogs.sciencemag.org/pipeline/archives/2017/10/18/drug-development-costs-revisited http://blogs.sciencemag.org/pipeline/archives/2017/10/18/dru... quotes $2bn as a more reasoned number.

Amygaz 9y ago
Except that 1st. Sparks' stuff only cost a fraction of that to develop (closer to the $200M), and 2nd lot of the early expenses were fronted with tax payer's money.
- sitkack 9y ago
  And it my understanding that, soon gene therapy will be selecting which bits to flip and flipping them. It still needs testing, feedback, etc, but it isn't the same as going through a full development cycle. Am I wrong?

conanbatt 9y ago

Whats unreasonable are the research costs. I would look into that.

hackeraccount 9y ago

As is reported down thread it might be reasonable or not but some of the assumptions are open to question. It's not unreasonable to suspect that Medicaid will cover the benefit. Also The number of people would just include the U.S. presumably the drug would be sold world wide.

testvox 9y ago
But the rest of the world doesn't recognize FDA approval (and the FDA doesn't recognize any other country's approval process). So the cost of certification has to be paid again for every country its sold in.