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Gene Therapy Was Hailed as a Revolution – Then Came the Bill

bloomberg.com

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Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#11

Post by aaavl2821 »

As mentioned in the article, a prominent cost-effectiveness research group, ICER, suggests that the treatment may be cost effective at prices of up to $1.5M, but with a different set of the assumptions, the cost-effectiveness threshold could be as low as $310K.

Other studies suggest CAR-T drugs are cost-effective at current prices, but they are not widely covered by payers. In these cases, it is less clear whether drug companies or payers are to blame for access to treatment

The article also does not mention what the gene therapy in question actually does. In small studies, it has essentially cured a disease that would otherwise kill babies before they are two. It's a pretty amazing product. And there are many companies hot on their heels, some of which are developing small molecule drugs -- much cheaper to develop and manufacture than gene therapy -- that actually seem to be potentially nearly as effective

The detailed ICER review is a good read if you're looking to understand how drugs are valued: https://icer-review.org/wp-content/uploads/2018/07/ICER_SMA_...

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#12

Post by devoply »

In capitalism no one sheds any tears if you can't make a business work. If you won't someone else will. Probably in China or India. They'll happily read all your papers, develop a copy-cat cure and sell it for 100k maybe even 10k in the name of Humanism. No one will shed a tear for all the profit investors lost out on.

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#13

Post by refurb »

tuxxy wrote: post #8
refurb wrote: post #4

Earlier quoted context omitted.

Unlike small molecules, gene therapy isn't something that somebody can just whip up in a lab. Similar to biologics, there are often some trade secrets involved in the manufacturing that require a lot of time and money to figure out.

How can anyone think this is ethical for lifesaving medical care?

Not sure what you mean. Are you saying it’s unethical that it’s hard to make new therapies?

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#14

Post by smacke »

tuxxy wrote: post #8
refurb wrote: post #4

Earlier quoted context omitted.

Unlike small molecules, gene therapy isn't something that somebody can just whip up in a lab. Similar to biologics, there are often some trade secrets involved in the manufacturing that require a lot of time and money to figure out.

How can anyone think this is ethical for lifesaving medical care?

Playing devil's advocate: there may be an argument that such care would not have been developed without the free-market monetary incentive. (Maybe not; maybe it would have been developed thanks to some government-funded grant. The point is: it's worth understanding what's possible and impossible in our current system.)

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#15

Post by Proven »

refurb wrote: post #2

What this article misses is that even with a price tag of more than $1M, many of these therapies will save the system money . They kind of gloss over it when talking about Zolgensma, but the current treatment, Spinraza, costs ~$750K in the first year and $375K every year after. So even by year 2, you've already spent one million and you pay that indefinitely . So if Zolgensma shows 5 years of efficacy and Zolgensma c…

You assume the cost of new therapies is workable simply because it’s more efficient than existing.

In reality many existing therapies are all pushing the system into debt and unworkable (for example it overcharges the healthy in order to subsidize the ill).

The reality is $2 million price tag may be acceptable to a multi-millionaire but unworkable for a 40 year old who makes $50K/yr.

After certain age and cost level, the math just doesn’t work out. It’s tough, but once you realize you don’t have the natural right to free or subsidized healthcare, it’s easier to accept.

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#16

Post by refurb »

sytelus wrote: post #9
refurb wrote: post #2

What this article misses is that even with a price tag of more than $1M, many of these therapies will save the system money . They kind of gloss over it when talking about Zolgensma, but the current treatment, Spinraza, costs ~$750K in the first year and $375K every year after. So even by year 2, you've already spent one million and you pay that indefinitely . So if Zolgensma shows 5 years of efficacy and Zolgensma c…

I don't think if the question is whether large bills can be handled by distributing $1M payments over N years. The question is, should this really be costing $1M? I understand if there is a billion dollar investment by big pharma that benefits 1000 people and so there is no other way but to charge $1M. However, it appears that fundamental techniques are all same and it gets reused for hundreds or thousands different…

For many of these diseases, yes there are only a a few thousand patients in the world.

And although gene therapy might be a fundamental technique, creating a gene therapy for a different disease is really hard because human biology is really hard.

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#17

Post by Reelin »

refurb wrote: post #13
tuxxy wrote: post #8

Earlier quoted context omitted.

How can anyone think this is ethical for lifesaving medical care?

Not sure what you mean. Are you saying it’s unethical that it’s hard to make new therapies?

Parent is asking how it can be ethical to place such a large price tag on a lifesaving therapy if the materials themselves are cheap. It's one thing for AMD or Intel to recoup engineering costs with outlandish per-chip costs as compared to the cost of manufacturing itself. However, serious ethical questions about profit margins and recouping R&D appear once someone's life is directly tied to usage.

That being said, obviously we have to fund development somehow.

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#18

Post by skwb »

refurb wrote: post #2

What this article misses is that even with a price tag of more than $1M, many of these therapies will save the system money . They kind of gloss over it when talking about Zolgensma, but the current treatment, Spinraza, costs ~$750K in the first year and $375K every year after. So even by year 2, you've already spent one million and you pay that indefinitely . So if Zolgensma shows 5 years of efficacy and Zolgensma c…

But due to insurance churn, you don’t want to be footing the bill for your competitor down the road. As an insurance manager, your unit of analysis is not healthcare system or society, but your quarterly bottom line.

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#19

Post by tuxxy »

Reelin wrote: post #17
refurb wrote: post #13

Earlier quoted context omitted.

Not sure what you mean. Are you saying it’s unethical that it’s hard to make new therapies?

Parent is asking how it can be ethical to place such a large price tag on a lifesaving therapy if the materials themselves are cheap. It's one thing for AMD or Intel to recoup engineering costs with outlandish per-chip costs as compared to the cost of manufacturing itself. However, serious ethical questions about profit margins and recouping R&D appear once someone's life is directly tied to usage. That being said, o…

This is a great comment, but I have to say my comment was rather rushed. I was implying more that the idea of trade secrets on life-saving treatment was unethical. The question of funding is an important discussion, but I was trying to discuss the trade secret details and I didn't really get my point across in the comment.

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#20

Post by aaronbrethorst »

skwb wrote: post #18
refurb wrote: post #2

What this article misses is that even with a price tag of more than $1M, many of these therapies will save the system money . They kind of gloss over it when talking about Zolgensma, but the current treatment, Spinraza, costs ~$750K in the first year and $375K every year after. So even by year 2, you've already spent one million and you pay that indefinitely . So if Zolgensma shows 5 years of efficacy and Zolgensma c…

But due to insurance churn, you don’t want to be footing the bill for your competitor down the road. As an insurance manager, your unit of analysis is not healthcare system or society, but your quarterly bottom line.

Sounds like a great argument for Medicare for All, the NHS, or other nationalized systems.
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