The million-dollar drug (2018)
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The million-dollar drug (2018)
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Re: The million-dollar drug (2018)
#2FYI, the drug is (trade name) Glybera (generic) alipogene tiparvovec. Wikipedia: https://en.wikipedia.org/wiki/Alipogene_tiparvovec
From 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..."
Re: The million-dollar drug (2018)
#3"...
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.
Re: The million-dollar drug (2018)
#4I 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.
Re: The million-dollar drug (2018)
#5I'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?
Re: The million-dollar drug (2018)
#6"... 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.
Better than not knowing what's killing you.
Re: The million-dollar drug (2018)
#7I'm surprised how well this essay was written. It was short and terse where it had to be.
Re: The million-dollar drug (2018)
#8I 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.
Not sure exactly, but considering alcohol messes with hormonal signaling I'm guessing it throws something out of balance
Edit: Looked up the wiki article, here is their explanation;
>Additional measures are avoidance of agents known to increase endogenous triglyceride levels, such as alcohol
Re: The million-dollar drug (2018)
#9I 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.
Not sure exactly, but considering alcohol messes with hormonal signaling I'm guessing it throws something out of balance Edit: Looked up the wiki article, here is their explanation; >Additional measures are avoidance of agents known to increase endogenous triglyceride levels, such as alcohol
Thx.
https://pubmed.ncbi.nlm.nih.gov/23511381/
Probably more pertinent:
Alcohol changes the structure of the liver cells and slows down the liver’s ability to process fats. This is the second way that triglycerides begin to accumulate and the levels rise. This is also the beginning of fatty liver disease.
https://findpulse.com/what-liquor-raises-your-triglycerides-...
Re: The million-dollar drug (2018)
#10I'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?
It's a matter of principle. Perverse principle but principle: "Why would we? [lower the price] Pricing shouldn't be a political decision. It should be a rational decision based on merits and values," he said. "Hundreds of millions of investor money has gone into the company, and if there is no return for those investments, there will be no new drugs because nobody's going to do that in the future, right?""
Which is to say that you have an investor group that expects to either be paid or they'll take their medicine away and the public be damned.
Or perhaps a perverse process. The system is how you get new, actually amazing drugs. The system is also how actual health care has become really terrible in the US in particular.