Gene Therapy Was Hailed as a Revolution – Then Came the Bill • Hacker News Board

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

#71

Post by hycaria » Mon, Apr 08, 2019, 1:45 PM UTC

empath75 wrote: post #62

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…

Letting people die is even cheaper.

Yeah I really don't get enabling the outrageously expensive carrying of genetic diseases such as SMA, especially at a time where prenatal screening can be done.

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

#72

Post by celinehh94 » Mon, Apr 08, 2019, 1:52 PM UTC

aaavl2821 wrote: post #11

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 dr…

Quick interesting note on this front - ICER is a US non-profit, but has basically zero influence in the actual pricing of drugs. It's actually illegal to value drugs this way in the US

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

#73

Post by celinehh94 » Mon, Apr 08, 2019, 1:54 PM UTC

cf wrote: post #38

Not to be too snarky but to the question in the article, “The question for health systems: how much are one-time therapies worth?”, you mean like vaccines? Presumably, it should cost a similar amount.

Vaccines are actually super unprofitable for Pharma companies, so this isn't a great model to emulate (although I agree with the general metaphor of gene therapy -> vaccines)

https://www.nber.org/papers/w9833

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

#74

Post by bluGill » Mon, Apr 08, 2019, 1:55 PM UTC

awongh wrote: post #35

One thing that I'd like to know is if the profit margin is the same for one of these one time treatments as a repeat treatment. If we assume that these things must be produced within a market system, it seems like the real moral question isn't what the dollar amount is, (since it logically follows that the cost to produce it must increase with the total revenue it's going to produce) but it would seem troublesome to…

Lower than you might think. Not only do you need to account for all the costs of developing that treatment, you also need to account for the costs to looking at a treatment that after some amount of study turns out not to work. This latter can be very expensive, and worse is an unknown cost, sometimes you are lucky and a few treatments work out, while other times you hit an unlucky streak and try a bunch of treatments that don't work out.

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

#75

Post by Not_a_pizza » Mon, Apr 08, 2019, 1:57 PM UTC

anfilt wrote: post #3

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…

At that point it's probably just cheaper to get a plane ticket to a country that does not patent or enforce a patent for the treatment.

Or a different planet!!!

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

#76

Post by Not_a_pizza » Mon, Apr 08, 2019, 1:59 PM UTC

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…

The expectation I have is that these sky high prices will drive a boom not unlike the computer electronics boom of the 60's. When money of this amount is at stake, people are willing to do almost anything to get in on the action.

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

#77

Post by celinehh94 » Mon, Apr 08, 2019, 2:01 PM UTC

celinehh94 wrote: post #72

aaavl2821 wrote: post #11

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 dr…

Quick interesting note on this front - ICER is a US non-profit, but has basically zero influence in the actual pricing of drugs. It's actually illegal to value drugs this way in the US

Sorry - should say use the valuation of the drug to determine patient access to it in the US. AKA, ICER could determine that drug 1 is massively un-cost-effective in comparison to drug 2, but you cannot restrict access to drug 1 due to this information. Most other countries tie drug price somewhat to health gain delivered by said drug.

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

#78

Post by nradov » Mon, Apr 08, 2019, 2:03 PM UTC

votepaunchy wrote: post #48

aaronbrethorst wrote: post #20

Earlier quoted context omitted.

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

It’s Called “reinsurance” and it’s already implemented in many states for cases such as these (million dollar payouts) since post-ACA an insurer could go bankrupt on a single customer with a pre-existing condition. https://en.m.wikipedia.org/wiki/Reinsurance

It's not possible a single insurer to go bankrupt due to a single expensive customer. State insurance commissioners wouldn't allow such a financially fragile insurance company to sell policies in the first place.

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

#79

Post by rayiner » Mon, Apr 08, 2019, 2:04 PM UTC

Ntrails wrote: post #61

aaronbrethorst wrote: post #20

Earlier quoted context omitted.

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

> Sounds like a great argument for Medicare for All, the NHS, or other nationalized systems. There's a good chance that NICE won't be willing to fund these treatments at all . Which is in no way a criticism of them (QALY is the just the most sensible semi-objective thing to base these decisions on) - but is worth highlighting when you say "free healthcare for all". It's only free for the healthcare considered to be g…

At the end of the day, that's what the calculus comes down to. Many of these gene therapies are for rare diseases, but creating them is still extremely expensive. At some point, the cost of dedicating those societal resources to saving those lives is too high.

That's a hard thing to admit, so people look for a way around it. But at the end of the day somebody has to pay. If you tell the drug companies that the products are too expensive, and ask them to accept lower returns than comparable high-tech, high-risk, talent and capital-intensive industries, you're asking those companies and their shareholders to pay. (Or you're asking them to cross-subsidize those treatments with money from treatments that have a much larger number of patients.) People love that idea because it makes it seem like you're getting cheaper treatments for "free" but all you're doing is hiding the costs. There is a cost, it's now just hidden and unaccounted for. If you told Apple they could only charge $250 for an iPhone, what do you think would happen?

Let the drug companies charge what they want to charge, and let government-funded healthcare programs decide what they're willing to pay to save a particular life. That's the most transparent and ethical thing to do.

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

#80

Post by bluGill » Mon, Apr 08, 2019, 2:07 PM UTC

snarf21 wrote: post #60

Aser wrote: post #52

The cost will come down. 20 years ago, the cost of sequencing one person's genome was over $100 million. Today it's $1000.

Not disagreeing with you but can you get a full genome for $1K? I assumed that was only partial.

Do you need a full genome? If a person has symptoms the doctor doesn't need a full genome sequence, just enough to verify that it is the/a sequence the treatment can fix. The cheaper the better, accuracy is important but but quantity outside the sequence in question is not useful.