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

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

#81
post #56

Earlier quoted context omitted.

CRISPR's not that hard, this guy did it in his garage. Delivering it to cells is a bit harder, but not that hard unless you're talking brain cells, then the blood-brain barrier causes trouble. https://www.theguardian.com/science/2017/dec/24/josiah-zayne...

anybody with a decent level of biochemical knowledge can do these things in a garage (I built a biolab in my garage and work at a lab that does CRISPR). However, the person you cited is clueless and is just kind of injecting things into his body without any really thought to efficacy or safety. There is a big difference between injecting reagents into a single human, and doing actual medicine.

[deleted]

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

#82
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.

CRISPR's not that hard, this guy did it in his garage. Delivering it to cells is a bit harder, but not that hard unless you're talking brain cells, then the blood-brain barrier causes trouble. https://www.theguardian.com/science/2017/dec/24/josiah-zayne...

Delivering it to cells is a bit harder

That's a bit of an understatement. The difference between doing CRISPR on a cell and a petri dish and doing it in a human is huge. That's why no one has done it yet.

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

#83

Earlier quoted context omitted.

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

Privatize the gains, socialize the losses!

Reinsurance is entirely private, not sure what you mean by "socialize the loss".

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

#84

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

I wouldn't say it has zero influence. Their assessments definitely impact the public perception of pricing in the US, so companies pay attention. But yes, it's not like NICE in the UK where the decisions have a direct impact on how much insurers pay for drugs.

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

#85
post #28

I'm ashamed when I see how much they are trying to charge. Glybera was a gene therapy that nobody used basically because it cost too much. This is a clear example that a company will never reduce the price of such a therapy. The actions of Mylan and the scumbag Shkreli also highlights the thought processes of pharma companies. As such, we shouldn't waste our time. The government should be allowed to negotiate pricing…

One of the reasons Glybera failed is that it just wasn't that good of a drug. It did reduce the symptoms of the disease, but they had zero evidence it actually reduce medical costs associated with it (hospitalization, etc).

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

#86
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.

Pricing it like a vaccine entirely ignores the number of patients treated. Unlike vaccines, you'd never give gene therapy to most of the population. It all likelihood, you give gene therapy to a few thousand patients.

Hard to even break even if you're charging $100 and treating 2000 patients.

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

#87
post #12

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.

Are you arguing that Chinese or Indian drug companies don't make a profit? If so, that wouldn't be accurate at all.

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

#88
post #74
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 treatment…

i theorize it isn’t lower than i think because the article hints that companies not selling the product at what the market will bear, and instead would rather price it to a specific margin. to me this is a special industry in that, if your gamble of profitability doesn’t pay off for something that already has sunk cost, don’t take it off the market, just sell it for whatever price the market will bear. that’s your moral responsibility.... not sure what the argument against that would be

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

#89
post #88
post #74

Earlier quoted context omitted.

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 treatment…

i theorize it isn’t lower than i think because the article hints that companies not selling the product at what the market will bear, and instead would rather price it to a specific margin. to me this is a special industry in that, if your gamble of profitability doesn’t pay off for something that already has sunk cost, don’t take it off the market, just sell it for whatever price the market will bear. that’s your mo…

I've read your reply 3 times, I can't figure out what you mean.

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

#90

Earlier quoted context omitted.

Or for insurance companies to form an association that comes up with policies that would benefit all of them if they all did them, and then requires all member companies to implement them. Not sure exactly what carrot or stick they could use to ensure that any remaining insurance companies have an incentive to join the association rather than trying to free-ride off what they're doing, but one might be able to come u…

This Wikipedia page might offer some ideas: https://en.wikipedia.org/wiki/Cartel

The page does not list enforcement mechanisms for cartels, and in fact says: "In general, cartel agreements are economically unstable in that there is an incentive for members to cheat by selling at below the agreed price or selling more than the production quotas set by the cartel (see also game theory). This has caused many cartels that attempt to set product prices to be unsuccessful in the long term. Empirical studies of 20th century cartels have determined that the mean duration of discovered cartels is from 5 to 8 years."

Come to think of it, the general idea is "If you, as an insurance company, foot the bill for these treatments with long-term benefits for your clients, this will cost $X, but save you $Y and other nearby insurance companies $Z in the future. Therefore, we the association will pay you $z $X, so there should exist values of $z that make this profitable for both parties (anything between $X-$Y and $Z)." The carrot is the payment of $z.

In terms of whether this association could become a price-fixing cartel... It's probably easier to verify compliance when compliance consists of performing a positive behavior (i.e. paying healthcare providers to do certain treatments; whatever the details of the payment, the important thing is whether the treatment was performed) instead of a negative behavior (i.e. not selling extra product at a lower price on the side). It doesn't seem obvious that this would make price-fixing much easier than it currently is.

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