171–180 of 215 posts
I was recently one of the first recipients of gene therapy for my condition. I have Hemophilia A, which is a bleeding disorder that results in bleeding into joints, muscle and soft tissue due to lack of a naturally occurring clotting factor that my body can't produce due to a defective gene. I received my dose of 30 billion viral particles about 13 weeks ago as part of a phase II clinical trial at UCSF. It is amazing…
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Except as a result of the price tag, they priced themselves out of the market and any possibility of revenue. That is not a rational. And efficacy isn't really in question: Perfect cure or not, it has a vast increase in quality of life including the ability to have children, avoid pancreatitis, and enjoy alcoholic beverages to name a few. If insurance companies are covering replacement therapy to the tune of $300,000…
I don’t think they priced themselves out of a market. Uniqure set the list price at $1M. In Europe each country negotiates their own drug price, so the real price would have been even lower than $1M. I’m going to speculate that the drug just wasn’t that good, so the demand wasn’t there and the program folded. I know Germany pays close to $1M for a specific hemophilia therapy and they pay that annually. The price itse…
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Actually, single payers in the EU are much better equipped to pay $1M now to save $300k/yr in perpetuity. All medical spending comes from one big pile. In the US, not people don’t stay with the same health plan for more than 2-3 years. So why would an insurance company pay $1M when they get 2-3 years of pay back, then the next insurer gets a free ride? I know there is an MIT prof who is pushing the idea of something…
Yeah, I'm not saying it doesn't make sense or that the EU wouldn't do it. I'm saying they aren't doing it, because the treatment is of unclear efficacy.
I was recently one of the first recipients of gene therapy for my condition. I have Hemophilia A, which is a bleeding disorder that results in bleeding into joints, muscle and soft tissue due to lack of a naturally occurring clotting factor that my body can't produce due to a defective gene. I received my dose of 30 billion viral particles about 13 weeks ago as part of a phase II clinical trial at UCSF. It is amazing…
I was recently one of the first recipients of gene therapy for my condition. I have Hemophilia A, which is a bleeding disorder that results in bleeding into joints, muscle and soft tissue due to lack of a naturally occurring clotting factor that my body can't produce due to a defective gene. I received my dose of 30 billion viral particles about 13 weeks ago as part of a phase II clinical trial at UCSF. It is amazing…
Did you need immunosuppressive treatment?
They tested me before for immunity to the virus family they engineered, to make sure I didn’t already have immunity.
Other than that they are monitoring my liver closely but there hasn’t been any elevation in blood tests or structural changes (observed via MRI).
I was recently one of the first recipients of gene therapy for my condition. I have Hemophilia A, which is a bleeding disorder that results in bleeding into joints, muscle and soft tissue due to lack of a naturally occurring clotting factor that my body can't produce due to a defective gene. I received my dose of 30 billion viral particles about 13 weeks ago as part of a phase II clinical trial at UCSF. It is amazing…
Thank you for sharing. My first undergrad studies were a double major in Science (Chemistry/Biology) 12 years ago and yet I had zero clue that we were at the stage currently to do what you described. To hear what you've just described is absolute music to my ears and it makes me very happy. While you may have lived a number of years dealing with your issues it means that we are likely approaching a time when children…
In a single generation we’ve gone from being able to treat the condition at all (replacement factor was just becoming available when I was born) to being able to completely cure it.
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Also the 100 million is used to distinguish between real medicine and snake oil. Also, is this treatment better than the current treatment? How can you be sure without a clinical trial?
You cant even do a proper clinical trial if there are only 300 patients in the whole of Europe who have the disease. You are missing the point.
An RCT comparing fecal transplant vs. vancomycin for recurrent C. difficile infection, for example, was halted after recruiting only 43 patients because it was judged no longer ethical to keep people in the vancomycin arm.
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A thing is worth what another will pay for it.
And no one was willing to pay this much for the drug, but the inexplicably decided no revenue was better than lowering the price.
I was recently one of the first recipients of gene therapy for my condition. I have Hemophilia A, which is a bleeding disorder that results in bleeding into joints, muscle and soft tissue due to lack of a naturally occurring clotting factor that my body can't produce due to a defective gene. I received my dose of 30 billion viral particles about 13 weeks ago as part of a phase II clinical trial at UCSF. It is amazing…
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It's bad to analyze this issue through the medium of a long-form article, which wraps the story up in emotional narrative. Here is a better write-up in the MIT Technology review: https://www.technologyreview.com/s/601165/the-worlds-most-ex... . The gist of the problem is this: > The Amsterdam company spent more than $100 million testing the drug and carving a path through Europe’s medical rules and regulations, which…
Thank you for raising this. I think the reason this particular drug didn't make economic sense was touched on in the article but not highlighted: the drug approval process is designed for drugs, not new technology. The price of a new gene therapy technology applied to a small pool of people can't justify it's cost of bring to market. There are plenty of cases where drug company's inflating their margins using phantas…