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A million-dollar drug

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Re: A million-dollar drug

#61

Post by tptacek »

ams6110 wrote: post #29
mabbo wrote: post #24

The point of the patent system is that we are giving inventors a time-limited monopoly on their invention in exchange for them sharing it with the world. We all benefit because we have access to this new invention and soon can make it ourselves, and the inventor has an incentive to promote and sell as much as they can while they own the monopoly. But it's not working here. The patent holder is effectively saying soci…

If a patent holder is not producing the patented thing, and not licensing it to someone else who is, the patent should expire. Kind of like a trademark that is not defended. Seems that would fix the problem?

No, not here.

Re: A million-dollar drug

#62

Post by freeone3000 »

Mvandenbergh wrote: post #37

NICE, which makes value for money decisions for the NHS, uses a threshold of £25k per Quality Adjusted Life Year for assessing cost effectiveness of treatments. At that threshold, to pay for an £800k drug treatment you'd need to give someone 32 extra years of life (or 64 years at double the quality of life and so on). This is enough to pay that kind of amount for life saving gene therapies, especially if given to you…

RAMQ [Quebec Health] does negotiate separately from the rest of Canada... but they don't have the money. They don't have the money for top-line cancer treatments, and they so severely underpaid optometrists that the optometrist union opted out entirely. There is very little chance that even at $250K a dose (which is an imaginary figure! the sellers have said they will not reduce the price at all!) a negotiation could be reached.

Re: A million-dollar drug

#63

Post by ethbro »

tptacek wrote: post #30
mabbo wrote: post #24

The point of the patent system is that we are giving inventors a time-limited monopoly on their invention in exchange for them sharing it with the world. We all benefit because we have access to this new invention and soon can make it ourselves, and the inventor has an incentive to promote and sell as much as they can while they own the monopoly. But it's not working here. The patent holder is effectively saying soci…

If the drug is patented, then by definition the best known mode of implementation has been disclosed. That's what a patent is. The problem with alipogene tiparvovec probably isn't the IPR†. Rather, it's that the treatment is extraordinarily expensive to administer (ironically, because it's something close to a total cure with a long-term impact, the drug seller is required to provide long-term monitoring to patients)…

> is of questionable effectiveness --- it improves quality-of-life metrics but not necessarily clinical ones like blood fat levels

"Although based on a small number of patients and episodes, overall pancreatitis incidence up to 2 years post-alipogene tivarvovec injection decreased by 5-fold"

"However, several signs of clinical efficacy independent of plasma TG were noticed up to 2 years after LPL gene transfection and raised the possibility that TG-rich lipoprotein characteristics, particularly the size, lipid content and kinetics of CMs, rather than plasma TG concentration per se, are the best surrogate markers of pancreatitis risk in LPLD."

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4956470/#!po=37...

From what I read, 2-year follow-ups seem to find continued (post-26 week) activity of the inserted gene.

So it's inaccurate to say there were no clinical improvements.

Re: A million-dollar drug

#64

Post by whydoineedthis »

TBH, it sounds like the govt regulation for the drug to even get approved are what drove the cost up. The product was essentially made before pharma even had to lift a finger, but they made them jump through a lot of hoops to get it to market. That's the $100mm they are trying to recoup. Also, I think a lot of folks are missing that the disease is not necessarily lethal when diet is controlled. Just saying, that's all.

Re: A million-dollar drug

#65

Post by tptacek »

ethbro wrote: post #63
tptacek wrote: post #30

Earlier quoted context omitted.

If the drug is patented, then by definition the best known mode of implementation has been disclosed. That's what a patent is. The problem with alipogene tiparvovec probably isn't the IPR†. Rather, it's that the treatment is extraordinarily expensive to administer (ironically, because it's something close to a total cure with a long-term impact, the drug seller is required to provide long-term monitoring to patients)…

> is of questionable effectiveness --- it improves quality-of-life metrics but not necessarily clinical ones like blood fat levels "Although based on a small number of patients and episodes, overall pancreatitis incidence up to 2 years post-alipogene tivarvovec injection decreased by 5-fold" "However, several signs of clinical efficacy independent of plasma TG were noticed up to 2 years after LPL gene transfection an…

I worried about the word "clinical" here, which might not be what I meant. It is not, however, inaccurate to say that concerns about efficacy were behind EU health system refusal to pay, and behind the therapy being taken off the market.

Re: A million-dollar drug

#67

Post by ethbro »

tptacek wrote: post #65
ethbro wrote: post #63

Earlier quoted context omitted.

> is of questionable effectiveness --- it improves quality-of-life metrics but not necessarily clinical ones like blood fat levels "Although based on a small number of patients and episodes, overall pancreatitis incidence up to 2 years post-alipogene tivarvovec injection decreased by 5-fold" "However, several signs of clinical efficacy independent of plasma TG were noticed up to 2 years after LPL gene transfection an…

I worried about the word "clinical" here, which might not be what I meant. It is not, however, inaccurate to say that concerns about efficacy were behind EU health system refusal to pay, and behind the therapy being taken off the market.

True. The question seems to be correlating the long-term decrease in serious episodes and gene expression (observed, albeit in a low sample size) with a marker (plasma TG did not change, but centrifuged TG composition by weight did).

The injustice here, if it exists, seems to be that the system is poorly configured to treat rare diseases.

Afaik, the FDA (regulatory side, for non-US readers) began granting more permissive experimental waivers for this sort of thing.

But the insurance side seems well within their rights to not pay by saying "This treatment has not been demonstrated effective to our standards." Something which is likely impossible given the cost of running normal trials and target population (small).

Hopefully, in the future, this will be addressed as more gene therapies become available and individualized medicine becomes the norm (similar to the revolution oncology has gone through in the past 30 years).

Re: A million-dollar drug

#68

Post by hakfoo »

charlesism wrote: post #44
lisper wrote: post #20

Earlier quoted context omitted.

The last sentence of the article sums it up pretty well: "If it's not commercially viable to produce a certain therapy, unfortunately, in our Western society, it does not happen." And it is far from clear that this is not as it should be, because it's not just LPLD carriers about whom tear-terking stories can be told. $1M can be deployed in other ways that could potentially save more lives. Instead of "Sorry LPLD car…

You make some good points. I'm not anti- all patents. Let's say, for example, a pharma patent worked more like a trademark: if you aren't going to use it (or maybe, if you abuse it), you lose it. Then we wouldn't have to trust this one company's opinion on whether it is "commercially viable" or not. Maybe that's a bad idea. I'm not a lawyer, and I don't work in medicine. I just doubt what we have now is the best of a…

They complain about the cost of research, development, and approval... so why not migrate those aspects to state entities which are not beholden to a profit motive?

Drug manufacturing is a very different business than drug discovery. We bundle them together hoping that the profits in one will bankroll the massive loss risk in the other.

But it doesn't work-- we end up with situations like this-- drugs that don't cover their upfront R&D costs, and also issues with questionable market-oriented priorities (I tend to think of the galaxy of me-too erectile-dysfunction medications that hit the market almost immediately after Viagra)

If we shoved a bunch of research dollars into public labs and universities, they could manage research, development, and certification, with the goal to produce a non-patented product that the manufacturers can compete to produce at scale as cheaply and reliably as possible. If the up-front science is paid for, maybe that 1,000 patients are economically viable to produce the actual pills.

Re: A million-dollar drug

#69

Post by tptacek »

thomastjeffery wrote: post #26
lisper wrote: post #20

Earlier quoted context omitted.

The last sentence of the article sums it up pretty well: "If it's not commercially viable to produce a certain therapy, unfortunately, in our Western society, it does not happen." And it is far from clear that this is not as it should be, because it's not just LPLD carriers about whom tear-terking stories can be told. $1M can be deployed in other ways that could potentially save more lives. Instead of "Sorry LPLD car…

Except $1 millon is not the entire economy. It's easy to hide resources by equating them to their monetary value. The fact is that there exist many millions of dollars, and the way each dollar is spent does not determine the way another dollar is spent.

This isn't complicated. Say there are 1,000 LPLD patients in North America (a wild overestimate). Then treating them with this therapy costs us $1Bn. If we limit ourselves to health care problems, is $1Bn better spent on these 1,000 LPLD patients, or on increasing penetration of existing therapies for other conditions? It seems likely to be the latter.

To believe LPLD therapy objectively deserves the allocation you're talking about, you have to believe that there aren't other cohorts of patients that are currently underserved. But we know that isn't true.

Re: A million-dollar drug

#70

Post by xxpor »

Mvandenbergh wrote: post #37

NICE, which makes value for money decisions for the NHS, uses a threshold of £25k per Quality Adjusted Life Year for assessing cost effectiveness of treatments. At that threshold, to pay for an £800k drug treatment you'd need to give someone 32 extra years of life (or 64 years at double the quality of life and so on). This is enough to pay that kind of amount for life saving gene therapies, especially if given to you…

The fact that women can't have children without the drug, but apparently can with it adds a new complication to the QALY calculation I think.
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