8 ms·

Governments and non-profits already funded the development of gene therapy. Letting private companies charge money for it and then blocking people who need it f

by elil17 4y ago

Governments and non-profits already funded the development of gene therapy. Letting private companies charge money for it and then blocking people who need it from getting it is a policy decision.

whiddershins 4y ago
Doing the procedure has a cost. There must be some pressure to reduce the cost, else it will never reduce.
- elil17 4y ago
  I agree there needs to be pressure. Why not regulatory pressure?
  - sn0wf1re 4y ago
    What sort of regulation? Make it cheaper by x% per year or we stop using it? Doesn't sound that dissimilar to "make it cheaper than the total cost of the current treatment or we won't use it".
    - elil17 4y ago
      I’d just change the “or we won’t use it” to “or your patents go away.”
      - pc86 4y ago
        And then instead of reading "that atidarsagene autotemcel not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatment" today, we would have read something like "current regulatory environment precludes research and development expenditures into atidarsagene autotemcel" in some biopharm prospectus a decade ago. You can't have it both ways and preventing people from making money means, not surprisingly, they will be very hesitant to spend money researching those areas.
        elil17 4y ago
        But you can have it both ways because most rare disease research is already funded by governments and nonprofits
        whiddershins 4y ago
        This is something people say over and over, I’m not sure of the source. Even if it were true, turning a discovery into a distributable, replicable, intervention is the opposite of a trivial task. And yes, our lovely regulatory environment makes it enormously expensive to study drugs and treatment and to get them approved. Who knows how many lives have been lost by this. When people talk about for example university research driving technology, I think the practicalities of the real world … aka business aka living in 4dimensional reality … are inaccessible to them. They elide all these pesky details which represent like 98% of what it takes to actually do anything.
      - refurb 4y ago
        Sounds like a great way for people to just not invest in new therapies. "Hey, that's a nice life-saving treatment you spent $500M developing. It would be a shame if we just took it from you."
      - concordDance 4y ago
        The USA really doesn't like it when you ignore their patents. Expect tarrifs, sanctions and trade secrets so you won't be able to reproduce future treatments.
  - whiddershins 4y ago
    because that is Dirigisme, which we reject for good reasons
krona 4y ago
In quality-adjusted life years (QALYs), there is an upper limit to the cost of any intervention because the most you can save from one intervention is one life. If the same money can be used to substantially improve the lives of 100 people with other interventions, then the cost-utility analysis may say a particular intervention is not effective. You might not like the utilitarian approach but this is how the UK measures effectiveness.
- elil17 4y ago
  I do like the utilitarian approach, I just think sometimes you need to look outside the box of do A or don’t do A. If the only options were pay for the treatment or don’t, then Ireland might be making the right call by not paying for it. Ireland could simply let a local company violate the drug patents. A country like the UK could impose conditions such as profit caps on pharmaceutical companies who base their work on publicly funded research. We could reduce the length of drug patents. There are many, many options besides “role over and let pharma companies charge $4 million/treatment when it costs them $1 million.”
  - ericd 4y ago
    From the article: "Libmeldy is approved for use in the European Union and U.K., although the U.K.'s drug price watchdog initially rejected the therapy due to its hefty list price of £2.8 million ($3.4 million at today's exchange rates), BBC News (opens in new tab) reported in 2022. The therapy's manufacturer, Orchard Therapeutics, then offered Libmeldy to the NHS at a significant discount." So it does seem like they've already discounted it heavily.
    - heavyset_go 4y ago
      "Significant discount" doesn't mean much when they're profiteering.
      - ericd 4y ago
        Do you know that they ended up at a “profiteering” price point, or are you just assuming? And you do have to pay the team of researchers to go on a speculative hunt for a cure for a super low incidence disease, if you want them to do it again in the future. If you then turn around and seize the results, you’re not encouraging them to take the risk next time.
BurningFrog 4y ago
This argument ignores the finite resource fact. No policy decision can change that.

by elil17 4y ago

Governments and non-profits already funded the development of gene therapy. Letting private companies charge money for it and then blocking people who need it from getting it is a policy decision.

whiddershins 4y ago

Doing the procedure has a cost. There must be some pressure to reduce the cost, else it will never reduce.

elil17 4y ago
I agree there needs to be pressure. Why not regulatory pressure?
- sn0wf1re 4y ago
  What sort of regulation? Make it cheaper by x% per year or we stop using it? Doesn't sound that dissimilar to "make it cheaper than the total cost of the current treatment or we won't use it".
  - elil17 4y ago
    I’d just change the “or we won’t use it” to “or your patents go away.”
    - pc86 4y ago
      And then instead of reading "that atidarsagene autotemcel not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatment" today, we would have read something like "current regulatory environment precludes research and development expenditures into atidarsagene autotemcel" in some biopharm prospectus a decade ago. You can't have it both ways and preventing people from making money means, not surprisingly, they will be very hesitant to spend money researching those areas.
      - elil17 4y ago
        But you can have it both ways because most rare disease research is already funded by governments and nonprofits
        whiddershins 4y ago
        This is something people say over and over, I’m not sure of the source. Even if it were true, turning a discovery into a distributable, replicable, intervention is the opposite of a trivial task. And yes, our lovely regulatory environment makes it enormously expensive to study drugs and treatment and to get them approved. Who knows how many lives have been lost by this. When people talk about for example university research driving technology, I think the practicalities of the real world … aka business aka living in 4dimensional reality … are inaccessible to them. They elide all these pesky details which represent like 98% of what it takes to actually do anything.
    - refurb 4y ago
      Sounds like a great way for people to just not invest in new therapies. "Hey, that's a nice life-saving treatment you spent $500M developing. It would be a shame if we just took it from you."
    - concordDance 4y ago
      The USA really doesn't like it when you ignore their patents. Expect tarrifs, sanctions and trade secrets so you won't be able to reproduce future treatments.
- whiddershins 4y ago
  because that is Dirigisme, which we reject for good reasons

krona 4y ago

In quality-adjusted life years (QALYs), there is an upper limit to the cost of any intervention because the most you can save from one intervention is one life. If the same money can be used to substantially improve the lives of 100 people with other interventions, then the cost-utility analysis may say a particular intervention is not effective. You might not like the utilitarian approach but this is how the UK measures effectiveness.

elil17 4y ago
I do like the utilitarian approach, I just think sometimes you need to look outside the box of do A or don’t do A. If the only options were pay for the treatment or don’t, then Ireland might be making the right call by not paying for it. Ireland could simply let a local company violate the drug patents. A country like the UK could impose conditions such as profit caps on pharmaceutical companies who base their work on publicly funded research. We could reduce the length of drug patents. There are many, many options besides “role over and let pharma companies charge $4 million/treatment when it costs them $1 million.”
- ericd 4y ago
  From the article: "Libmeldy is approved for use in the European Union and U.K., although the U.K.'s drug price watchdog initially rejected the therapy due to its hefty list price of £2.8 million ($3.4 million at today's exchange rates), BBC News (opens in new tab) reported in 2022. The therapy's manufacturer, Orchard Therapeutics, then offered Libmeldy to the NHS at a significant discount." So it does seem like they've already discounted it heavily.
  - heavyset_go 4y ago
    "Significant discount" doesn't mean much when they're profiteering.
    - ericd 4y ago
      Do you know that they ended up at a “profiteering” price point, or are you just assuming? And you do have to pay the team of researchers to go on a speculative hunt for a cure for a super low incidence disease, if you want them to do it again in the future. If you then turn around and seize the results, you’re not encouraging them to take the risk next time.

BurningFrog 4y ago

This argument ignores the finite resource fact. No policy decision can change that.