Ibrutinib is neither miraculous, nor is it a cure for anything. Like any cytostatic, it has severe side effects, but it does disrupt a critical signaling path in certain types of B-cell cancers. It's an expensive drug that cancer patients can take to prolong their lives for a comparatively short period. It's not a cure.
Of course, since patients on Ibrutinib require daily doses, Pharmacyclics is golden for now, but only until better cytostatics become available or the patent runs out.
Even from a business perspective, this doesn't seem to be a particularly inspiring story. By rights, this company should have gone under, but then randomly struck gold. However nice this turn-around may be for the founders, there is no special insight, scientific or business-related, to be gained here.
$90 per pill and about $100k per year per patient, life long? I should do some math to understand how many of those patients a country is able to sustain. Private insurances or welfare don't make much difference, somebody has to pay all that money and few people make 100k per year plus what they need to live.
The patent is only for a relatively short period and Cancer is generally not a contagious disease. So, really it's 90$ per pill now, and 10cents a pill for the rest of time.
PS: Don't forget, the company was sold for 21 billion which is chump change for the world.
The quick estimate is that US per capita GDP is ~$50k and the population is ~320m. So you can immediately upper bound it at half the population or 160m patients (two people earn <100k, which pays for <1 person's treatment). This is why health economics tends to use much lower amounts like $50k per QALY. I don't know if the UK's NHS has approved Ibrutinib, but if they have, they must've gotten a big price break from Pharmacyclics because $100k is way above any price they would approve.
The NHS seems to have approved it at ~17% discount (but may be towards a higher risk group because it is only available as a first-line medication if you have a chromosomal anomaly -- I am not sure of the relevance of this but it seems it's not the preferred treatment in other cases):
I know the mods typically prefer to leave titles the same as the article's title, but the article's title is misleading.
There's no "cure," miracle or otherwise. The article is pretty interesting, but the drug doesn't cure or even attempt to cure cancer, it just delays it.
So Pharmacyclics cannot have a miracle cure since they don't attempt to cure.
I've been asked several times before why we don't use more "kill-it-dead-forever" (another way to look at covalent inhibitors like Ibrutinib)
Covalent inhibitors are quite tricky to work with because every drug, no matter how specific has some off target effects. If your non-covalent drug accidentally binds to something it shouldn't, after a little while it'll go on its merry way (assuming the kinetics aren't awful). This means some side-effect regarding that protein it bound, but nothing permanent.
On the other hand if a covalent inhibitor hits something it shouldn't, then that protein it hit is SOL, and gone until a new copy of it can be made. This could take a very long time in certain cases, causing much longer lasting effects.
As a result, side-effects with covalent drugs can be both more severe and more variable. This is one reason that although they are popular in some areas of lab biochemistry, they haven't made as much of an impact in the broader clinic.
In this particular case, the specificity of the drug, the importance of the target to the disease mechanism, and the patient population all combine to make it viable. However, it doesn't mean the next covalent inhibitor that comes along will work as well.
That's a reasonable description of some common concerns with covalent drugs, but to say that they haven't had much impact in the clinic is misleading. See, for example
* Beta lactam antibiotics (e.g. penicillin)
* Aspirin
* Prilosec
And there are plenty of others. Many companies do tend to avoid them, but one could reasonably argue that their reputation is undeserved.
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[ 1.8 ms ] story [ 31.3 ms ] threadOf course, since patients on Ibrutinib require daily doses, Pharmacyclics is golden for now, but only until better cytostatics become available or the patent runs out.
Even from a business perspective, this doesn't seem to be a particularly inspiring story. By rights, this company should have gone under, but then randomly struck gold. However nice this turn-around may be for the founders, there is no special insight, scientific or business-related, to be gained here.
PS: Don't forget, the company was sold for 21 billion which is chump change for the world.
http://www.ukmi.nhs.uk/applications/ndo/record_view_open.asp...
That lists the per-pill price as about 51 gbp or 75 usd instead of 90 usd.
It doesn't cure anything. It just slows down the cancer for some time, so, "life long" isn't that long. It's kind of a race of patient against patent.
There's no "cure," miracle or otherwise. The article is pretty interesting, but the drug doesn't cure or even attempt to cure cancer, it just delays it.
So Pharmacyclics cannot have a miracle cure since they don't attempt to cure.
Covalent inhibitors are quite tricky to work with because every drug, no matter how specific has some off target effects. If your non-covalent drug accidentally binds to something it shouldn't, after a little while it'll go on its merry way (assuming the kinetics aren't awful). This means some side-effect regarding that protein it bound, but nothing permanent.
On the other hand if a covalent inhibitor hits something it shouldn't, then that protein it hit is SOL, and gone until a new copy of it can be made. This could take a very long time in certain cases, causing much longer lasting effects.
As a result, side-effects with covalent drugs can be both more severe and more variable. This is one reason that although they are popular in some areas of lab biochemistry, they haven't made as much of an impact in the broader clinic.
In this particular case, the specificity of the drug, the importance of the target to the disease mechanism, and the patient population all combine to make it viable. However, it doesn't mean the next covalent inhibitor that comes along will work as well.
* Beta lactam antibiotics (e.g. penicillin)
* Aspirin
* Prilosec
And there are plenty of others. Many companies do tend to avoid them, but one could reasonably argue that their reputation is undeserved.
I wish they would not use such language. There is no such things as "miracles"
regards,
science