4 ms·

This is exciting, although the cost of treatment is very high. Contributing factors to the high cost is that gene therapy is a cutting edge approach with a high

This is exciting, although the cost of treatment is very high. Contributing factors to the high cost is that gene therapy is a cutting edge approach with a high failure rate and thus high development costs, and DMD is a rare disease with a small patient population. In time, as the development of gene therapy treatments become more refined, costs will go down. Successes in gene therapy treatments are exciting, as the success rate of traditional small molecule therapies have stagnated over the last 10-20 years. Moreover, small molecule therapies may never prove effective in treating many genetic diseases.

tptacek 3y ago
The disease itself is incredibly expensive. The price of breakthrough treatments will come down faster than the costs of having the disease.
- BenFranklin100 3y ago
  I don’t know the specifics of the costs of caring for one with DMD, but I strongly agree that in general, new disease therapies are the only sure way to drive down medical costs in the medium and long term. This represents a victory, no doubt.
  - fullburley 3y ago
    It is very expensive. We will likely have $2M USD in insurance claims for medical this year, and our son is currently aged out of the approved label. At a gross cost of $3.2M for this treatment, its expensive but definitely cost effective relatively speaking.
    - arthur2e5 3y ago
      Considering this new treatment is just a single dose[^1] that hopefully lasts for quite a while[^2], it might actually cost a lot less in the long run. [^1] see FDA press release and monograph. addditional doses won't do much because by then the immune system has learned to attack the AAV vector -- a different vector strain would be needed and that would be a new drug to be approved [^2] no idea what the turnover rate is. and honestly, the FDA isn't even sure it helps with anything, hence conditional.
ramraj07 3y ago
I remember over lunch with a person researching custom DNA vaccines and he mentioned to get personalized injectable grade DNA made per person costs a million dollars. My mind was blown.
- throwaway2037 3y ago
  I agree: It is mind-blowing -- the cost, but the science is also mind-blowing. This stuff really is science fiction. When I was a kid, I could never imagine it was possible. Think how primitive computers were 30 years ago. All of these bio-breakthroughs are impossible without staggering amounts of compute and improved laboratory hardware. My thinking: How long for each divide-by-ten step? 1M USD today, 100K USD next, 10K USD later. At 10K USD, for these life threatening diseases, it is a drop in the bucket. Any insurance company would pay for it, because it would easily save many times more in avoided treatments for the illness. Think about the revolution that 3D printers brought. I could see something similar for custom DNA vaccines. They should be targeting wealthy people who want cosmetic effects, like hair regrowth, less wrinkles, hormones like a 30 year old, etc. They can pay, and it will help to mature the industry and production pipeline.
  - 100ideas 3y ago
    In principle, these costs could be 1000x less. Much like minicomputer -> dell price shift.
  - bboygravity 3y ago
    If the rate of price-decline of DNA sequencing is any referece: it took 14 years for the price of that to be devided by 100.000. So 3,5M USD cost now would be 35 USD in 14 years (not taking into account any hyperinflation that I think is likely in that same time-frame).
    - light_hue_1 3y ago
      The cost has nothing to do with the cost of sequencing DNA. DMD is extremely rare and expensive. The gene therapy itself is very cheap. The cost of the drug is entirely due to the value it provides. The company is already talking about upping the price to $12 million.
LifeIsBio 3y ago
It is exciting! As others in the thread are saying, the cost of individuals with severe rare diseases are also very high. Here’s a recent attempt at quantifying the costs across all rare disease: https://chiesirarediseases.com/assets/pdf/chiesiglobalrarediseases.whitepaper-feb.-2022_production-proof.pdf https://chiesirarediseases.com/assets/pdf/chiesiglobalraredi...

tptacek 3y ago

The disease itself is incredibly expensive. The price of breakthrough treatments will come down faster than the costs of having the disease.

BenFranklin100 3y ago
I don’t know the specifics of the costs of caring for one with DMD, but I strongly agree that in general, new disease therapies are the only sure way to drive down medical costs in the medium and long term. This represents a victory, no doubt.
- fullburley 3y ago
  It is very expensive. We will likely have $2M USD in insurance claims for medical this year, and our son is currently aged out of the approved label. At a gross cost of $3.2M for this treatment, its expensive but definitely cost effective relatively speaking.
  - arthur2e5 3y ago
    Considering this new treatment is just a single dose[^1] that hopefully lasts for quite a while[^2], it might actually cost a lot less in the long run. [^1] see FDA press release and monograph. addditional doses won't do much because by then the immune system has learned to attack the AAV vector -- a different vector strain would be needed and that would be a new drug to be approved [^2] no idea what the turnover rate is. and honestly, the FDA isn't even sure it helps with anything, hence conditional.

ramraj07 3y ago

I remember over lunch with a person researching custom DNA vaccines and he mentioned to get personalized injectable grade DNA made per person costs a million dollars. My mind was blown.

throwaway2037 3y ago
I agree: It is mind-blowing -- the cost, but the science is also mind-blowing. This stuff really is science fiction. When I was a kid, I could never imagine it was possible. Think how primitive computers were 30 years ago. All of these bio-breakthroughs are impossible without staggering amounts of compute and improved laboratory hardware. My thinking: How long for each divide-by-ten step? 1M USD today, 100K USD next, 10K USD later. At 10K USD, for these life threatening diseases, it is a drop in the bucket. Any insurance company would pay for it, because it would easily save many times more in avoided treatments for the illness. Think about the revolution that 3D printers brought. I could see something similar for custom DNA vaccines. They should be targeting wealthy people who want cosmetic effects, like hair regrowth, less wrinkles, hormones like a 30 year old, etc. They can pay, and it will help to mature the industry and production pipeline.
- 100ideas 3y ago
  In principle, these costs could be 1000x less. Much like minicomputer -> dell price shift.
- bboygravity 3y ago
  If the rate of price-decline of DNA sequencing is any referece: it took 14 years for the price of that to be devided by 100.000. So 3,5M USD cost now would be 35 USD in 14 years (not taking into account any hyperinflation that I think is likely in that same time-frame).
  - light_hue_1 3y ago
    The cost has nothing to do with the cost of sequencing DNA. DMD is extremely rare and expensive. The gene therapy itself is very cheap. The cost of the drug is entirely due to the value it provides. The company is already talking about upping the price to $12 million.

LifeIsBio 3y ago

It is exciting! As others in the thread are saying, the cost of individuals with severe rare diseases are also very high. Here’s a recent attempt at quantifying the costs across all rare disease: https://chiesirarediseases.com/assets/pdf/chiesiglobalrarediseases.whitepaper-feb.-2022_production-proof.pdf https://chiesirarediseases.com/assets/pdf/chiesiglobalraredi...