by refurb on 11/21/21, 8:20 AM
It doesn't make for a very compelling news story, but the real reason why Glybera failed is because it's not that great of a drug and it loses effect over time and you can't redose with the same AAV vector. They also relied a surrogate endpoint versus actual clinical measures (as those were to confounded by the small sample size and patient variability).
It just barely got approved by the EMA and based on feedback the company didn't bother to pursue FDA approval.
by thinkloop on 11/21/21, 9:26 AM
Everyone is blaming the company or the patent system or general greed, but really there is an extremely simple solution: pay the $1M. In Canada and Europe it's medicare, ie taxes, ie the general population that would pay for it, but they refused. In the US it's insurance companies, ie the general population (as insurance is just socialized losses), that refused. If people are uninsured then force the state, ie everyone who is feeling so bad about it, to pay for it. It's odd that being the genius inventors also means that you now also have to be the only ones to pay for it. This isn't a story of exorbitant profits - even at a million a dose they would barely make their money back. It makes no sense to isolate the group of people who are actually working on the thing to be the only ones to pay for it.
By extension, this story demonstrates how we price life. What if the cure was a billion dollars, is that worth society paying? What about a trillion? At one point a limit is reached, and it seems that one million is one of those limits that we all quietly agree upon.
by jagger27 on 11/21/21, 6:19 AM
I can’t imagine how hard it must be for the dozens of people out there suffering from this rare ailment knowing a perfect cure/treatment is possible but not worth it for the shareholders.
by exhilaration on 11/21/21, 5:13 AM
I'm confused, they say it was priced at $1 million because that was what they felt the drug was worth compared to the ongoing therapies needed to keep these patients alive. Ok fine. But once it became clear no one was going to pay that price, wasn't there some lower price that would still earn them a profit?
by flipbrad on 11/21/21, 6:32 AM
"Use it or lose it" / compulsory licensing doesn't seem like a terribly bad idea sometimes.
by WalterGR on 11/20/21, 11:57 AM
FYI, the drug is (trade name) Glybera (generic) alipogene tiparvovec. Wikipedia:
https://en.wikipedia.org/wiki/Alipogene_tiparvovecFrom the submitted article:
"...The first drug ever approved that can fix a faulty gene.
It's called Glybera, and it can treat a painful and potentially deadly genetic disorder with a single dose — a genuine made-in-Canada medical breakthrough..."
by stefan_ on 11/21/21, 6:27 AM
Never mind non-practicing entities for software patents, but surely for patents on medicine keeping them valid when no one is making any is entirely bogus and not deserving of legal protection?
by legulere on 11/21/21, 9:25 AM
by d136o on 11/21/21, 5:58 AM
It seems to me that it’s not just the price of a single treatment that is too high, but also that the patients are fully recovered and don’t have to pay a recurring annual subscription fee to live normal lives. Or is that too cynical?
by theskypirate on 11/22/21, 8:12 AM
Society itself is collectively responsible for this failure, not the patent owners. Funding could have been provided through charity, or through legislation. But this was not done, and instead development of this drug was left up to profit seeking investors.
It isn't the investors' duty to bankrupt themselves to produce this drug. They tried to find a way to get this produced through the private sector, and they found that it was impossible. It should instead be handled through the public sector, and it is the general public who are ultimately responsible.
by at_compile_time on 11/21/21, 6:30 AM
How long does the patent last? It should be made available as a generic drug when the exclusivity period elapses, right?
by DantesKite on 11/21/21, 5:37 AM
I'm surprised how well this essay was written. It was short and terse where it had to be.
by nefitty on 11/21/21, 4:48 AM
"...
Her blood becomes thick and white with fat particles that can destroy her pancreas.
All her life, Turcotte has had to follow a strict diet. She can't eat cheese or chocolate or any food that contains fat. And she can't drink even a drop of alcohol."
Jesus.
by ak39 on 11/21/21, 11:15 AM
Fascinating story and the use of virus to repair genetic anomalies. What other uses can there be of similar conditions? And why aren't we hearing more of it (if the approach is viable)?
by DoreenMichele on 11/21/21, 5:09 AM
I wish I knew why these patients can't have alcohol. I mean the biological mechanism. What the relationship is between misprocessing fat and alcohol being deadly.
by shmerl on 11/21/21, 7:10 AM
Price should be based on cost of production, not on how often the drug is needed. So stupid not to make it available because someone was crazy to price it that high.