People are fixated on the price for this drug, but the article itself is pretty clear on the rationale for the price tag: current alternative therapies have a price tag of up to $300k/yr, indefinitely. This drug is a one-time therapy. Presumably, any health system would jump at the chance to replace a $300k/yr recurring charge with a 1-time $1MM fee.<p>But they don't. Health systems in Europe apparently refuse to pay for this therapy. The reason for that, from reading other articles, appears to be that it is of questionable efficacy. Patients report fewer pancreatitis attacks, but clinical indicators like blood fat levels are apparently unchanged. The tiny market and efficacy concerns might explain why the therapy is no longer available at any 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.<p>It is amazing to me that we can manufacture and program viral particles to target specific cells. In my case, my treatment is a non-nucleonic technique that didn't repair the actual chromosomal hereditary defect but inserted a working gene, additional and freely available genetic material into my liver cells. It's amazing that the body can just recognize this extra bit of code and just start producing the factor. The engineer in me is also impressed they can make so many copies of the engineered virus.<p>It appears to be working. My factor levels have steadily increased and I’m now no longer a severe or even moderate hemophiliac. I’m their first patient to have achieved these results for hemophilia A severe and it puts me well into the therapeutic range. It really feels like I have been given a new body, it’s indescribable. For the first time in my life, I’m pain free.<p>It’s a weird feeling. Pain, mostly from bleeds into my joints, has been my constant companion. I was never able to do sports as a kid and had to avoid many other activities that would put me at risk. I have mixed feelings about all of this. On one hand I’m extraordinarily grateful, and the other side I see what a huge disadvantage I’ve been at compared to others for all of my life. I thought this day would never come, I had resigned myself to my limitations but now they are gone in a matter of weeks through the miracle of a medical experiment.<p>My old medication, a replacement clotting factor that was synthetically produced in a lab, worked but not nearly as well as this. Not to mention the fact it cost over $100,000 per month and I had to give myself an IV every other day. It's truly been a life changing experience for me, and I hope that gene therapy becomes widely available to others.<p>Also, the thing that was a milestone in this case was that the Hemophilia A factor VIII gene has been notoriously difficult to create a genetic therapy for because of the complexity of the actual gene itself. On the scale of what the body produces, its one of the more complicated proteins. So it basically means many other genetic conditions will be able to be treated with and addressed with similar technology.<p>Such an exciting time to be alive, and yes there are amazing things happening right now despite all the bad news you may hear!
<i>Glybera was never sold in North America and was available in Europe for just two years, beginning in 2015</i><p>The patent will expire within a few years, setting the stage for generic manufacturers to step in, but the article doesn't mention it.<p>In the US, patent protection extends for 20 years after the earliest filing date for the application on which the grant is based. There can be adjustments to the term if the grant was delayed.<p><a href="https://en.wikipedia.org/wiki/Term_of_patent" rel="nofollow">https://en.wikipedia.org/wiki/Term_of_patent</a><p>The article is fuzzy about the timeline of events, bu has this quote:<p><i>The stunning results of the mouse experiments were featured on the cover of the journal Human Gene Therapy in September 2004. The cover image showed how, week by week, the mouse blood changed from milky white to a clear, transparent red, illustrating just how effectively the gene therapy was working.</i><p>So it seems like a good first guess to put the patent filing at or before 2004.<p>If so, then the Glybera patent would expire sometime after 2024. At that point, generic manufacturers can step in to sell their version of the product, assuming regulatory approval.
This seems bizarre to me:<p><i>Van Deventer says the company never considered lowering the price. "Why would we? Pricing shouldn't be a political decision. It should be a rational decision based on merits and value."</i><p>If no one will pay $1 million, your revenue is $0. Assuming the drug costs much less than $1 million to make, surely any revenue number is better than $0?
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.<p>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 young children but not enough to treat something that can be largely managed through diet control.<p>Since the majority of the world's patients are in Quebec, I don't understand why the provincial government doesn't cut a deal for this drug. I'm sure they'd rather sell it $250k a dose to a nice big patient population than at $1m to nobody. Quebec has a lot of pricing power here as the only large potential buyer.
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.<p>But it's not working here. The patent holder is effectively saying society isn't allowed to have the invention. They're using a legal means meant to share the knowledge with the world to instead horde it away from the rest of us.<p>When the system doesn't work, the system must be improved. How can we incentivize inventors to not do this?
Getting a drug approved for use is an expensive proposition, but that doesn't mean that it is impossible for a nonprofit organization to step in and get the job done when the target treatment group is too small or too poor to make it worthwhile for those with a profit motive.<p>The Drugs for Neglected Diseases Initiative, a nonprofit which grew out of Doctors Without Borders, just received approval for a pill that successfully treats African Sleeping Sickness.<p>>Ultimately, the drive for approval cost $63 million and involved clinical trials including 750 patients in Congo and the Central African Republic. Two million villagers were screened.<p>The costs were paid by seven European countries, the Bill and Melinda Gates Foundation, Doctors Without Borders and other donors.<p><a href="https://www.nytimes.com/2018/11/16/health/sleeping-sickness-africa-cure.html" rel="nofollow">https://www.nytimes.com/2018/11/16/health/sleeping-sickness-...</a>
in society, we have another large, one-time purchase people often make in which there are special financial arrangements and rules: buying a home. it seems to me that gene therapies (high priced, one-time use drugs) are going to warrant alternative financial instruments. there's absolutely no reason this drug should have failed when the amortized cost over 10 years is less than an inferior product. regardless of the other questions this is a pure market failure by the financial industry.<p>edit: the downvotes probably assume I mean the patient is going to pay out of pocket. of course not. the insurance companies would be paying. the only inferiority between gene therapies and non-cures is their pricing structure. we invented a solution to this problem thousands of years ago: debt.
> "Why would we? 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?"<p>As terrible as it sounds, this logic seems sound to me. If we think having this type of drug is worth the > $100MM it took to develop, then health systems should either fund the $100MM research, or they should let private companies do it and pay them fairly for their work. $1MM for something so expensive to develop with so few possible users doesn't seem crazy to me.<p>If the $100MM is in fact <i>not</i> worth it, then, well, you get the situation we have now.<p>I'm sure a lot of people will read this article and think the owners/funders of the drugs are the bad guys. But nobody can be expected to work for free.
"Well, one pharma company has the patent, and we need to let them charge what they think the market will bear. Sorry LPLD carriers, you have to die now." Apparently, this is the best the world can manage in 2018.
To me, this is akin to a game company raising the price of a game. The costs are already sunk. The only thing that counts is setting a price on copies of that game. Those who pirate copies of the game incur to loss of revenue because those people would've never been able to pay for it. It becomes a question of projecting the maker's morals onto those who did not pitch to the coffer instead enjoying the game like a dirty thief. This is crazy, the only loss to the manufacturer is the uncomfortable idea people are enjoying or benefiting from your creation for free. Yet there are developers who happily contribute to open source projects and games with little to no recognition or reward.<p>Likewise with drug manufacturing, it's the same idea. It's illegal to copy a pharmaceutical drug and take it yourself and others without the approval of the patent holder. Yet, it is the patent holding party that determines how many licensed copies it is willing to sell. Thus, they are able to take the moral high ground in pricing this drug, but it's the same principle as above-pony up for my idea, shame on you for benefitting from it and not contributing to my wealth.<p>I really do think this company is out of touch with reality. All in all, I feel like this only earns them bad press, especially after Martin Shrekli debacle. There's also a potential for a lawsuit, based on human rights I'm sure.
>"You need to maintain the factory, you need to do the paperwork, you need to test the product, you need to make new product batches all the time because product expires," he said.<p>I'm pretty confident LPLD sufferers around the world would be prepared to move to the LPLD hot spot in Quebec, and work part time in the factory under proper supervision?
So I was expecting another story about some IP troll buying the rights to a drug with a monopoly and then jacking up the price (like the whole EpiPen fiasco) but this wasn't that.<p>There's an old Chris Rock bit about Big Pharma where he says there'll never be another cure for HIV like there was for polio because there's no money in that. The money is in getting you to the next stop.<p>It's poignant because this isn't a theoretical scenario. Gilead recently was downgraded on declining revenues because they're wiping out the disease (Hepatitis C IIRC?).<p>So, back to Glybera. There are plenty of low-incidence diseases that are treated by expensive drugs to manage them. This is a lifelong commitment. Covering such drugs in company health plans can significantly increase the per-member costs.<p>If you have a drug that essentially cures the drug in one dose shouldn't that be weighed against the lifelong cost of covering a regime to manage the disease that is inferior? Multiply that by the disease being quite rare and sure, you end up with a $1 million price tag.<p>As further evidence for how screwed up the US health insurance system is: companies enroll in plans for their members typically for a period of a year or maybe a few years. Let's say your drug's price of $1m compares favourably to $100k/year for 40 years to manage the disease. How can a company who might only be covering the employees for 1-3 years be expected to cover that higher cost?<p>To be clear, this is further evidence of how stupid the US model is. In a single payer model this particular concern goes away.<p>How many rare genetic disorders are out there where $1 million per patient for an essentially complete cure isn't a bargain compared to the cost of managing the disease? Probably a lot. Is it fair to decry such expensive drugs just on their price tag without looking at the facts? Probably not but I bet you it will happen.
This article brings up a thought experiment I've been pondering for a good while now...<p>So what would you charge for a drug that guaranteed you would live 30 extra years after you spent 20 million to develop it?<p>For an added twist, let's say it's NOT FDA approved but you have solid clinical evidence that it's efficacy was solid and can your "street cred" (whatever that means in biotech) allows you access to the world's most successful (ie richest) people.<p>I have my answer...what's yours?
> But it's the only way scientific discoveries ever get to patients, because universities don't make drugs.<p>Why not?<p>If there are so very few patients, a lab could easily produce enough for them.
Video for article <a href="https://www.youtube.com/watch?v=rBSv0deIVzM" rel="nofollow">https://www.youtube.com/watch?v=rBSv0deIVzM</a>
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.
If it works but it's not economically-viable: the moral and ethical choice is to open-source it or give it to a non-profit medical foundation. It sucks to have a rare disease because there isn't enough profit to recoup r&d; this is where non-profits, NGOs and GSEs should advance medicine where big pharma trades lives for profits.
Will other scientific or technological advances (like gene therapy drugs for more common disorders) cause the price of this drug to come down in the future?<p>Say, if gene therapy became common, there wouldn't be as much red tape for approving this particular one, and there might be machines or research that could be shared with other drugs to amortize the cost?
> Pricing shouldn't be a political decision. It should be a rational decision based on merits and values," he said.<p>The money quote. A rational decision? Maybe. Exactly what merits? Exactly which values?<p>"Hundreds of millions of investor money has gone into the company"<p>But none of that led to the development of the drug.
That's probably the greatest example of the issue that Open Longevity was going to solve by combining patient organization with ICO funding to conduct clinical trials without profit driven big pharma. Unfortunately, that didn't happen…
> <i>The problem was the price.</i><p>I thought that drug patents were basically illegal in India. It seems there’s a huge market there for this kind of thing. An abandoned drug with a huge price tag.<p>Also I’m not terribly worried. The patent will wear off eventually and then anyone can make it.
As sad as this story is, the silver lining is that patents expire, and 20 years from the patent filing point (probably 5-10 years from now?), patients will be able to be affordably cured. It sucks but is better than nothing.
What’s up with this drug now?<p>Sounds like it’s patents and IP protection should be up.<p>I’d imagine you could get a single dose manufactured 1 off for $100k by a lab.
This is kind of a common issue with genetic disorders. When I was active on cystic fibrosis lists, sometimes articles were posted that said stuff like "It costs an average of $100k annually to treat CF" and parents would discuss it and go "That sounds low to me" and some older patient would chime in and say "Last year, my drug prescriptions alone were $100k. That's not counting the cost of doctor's visits and hospitalizations."<p>(Estimates in articles vary wildly. I searched briefly and just trying to find something that makes sense for like two minutes just makes me tired. So if you want to jump up and dispute that with some googled up article, I don't feel like arguing with you about it. I've already told you actual people living with the condition read those articles and go "Where on earth are they getting these ridiculous low ball figures???")<p>So then they come out with some very expensive drug that costs like a quarter million or more annually and it only treats around five percent or so of patients with CF. The price on Wikipedia [1] is currently listed at over $300k annually.<p>[1] <a href="https://en.wikipedia.org/wiki/Ivacaftor" rel="nofollow">https://en.wikipedia.org/wiki/Ivacaftor</a><p>And then who can afford that? If you have been sick your whole life, you probably don't have a lot of savings or a lot of ability to come up with big bucks, etc. You are probably deeply in debt and not making much money.<p>I don't know the answer. Conventional medicine doesn't really work for people with genetic disorders. It tends to be crazy expensive and also merely mitigates things somewhat. The standard expectation is that you will suffer a bit less and maybe live a bit longer, but you won't ever really be well.<p>So such people understandably want <i>a cure.</i> That's the holy grail for folks coping with genetic disorders.<p>I think there are potentially other avenues for some portion of people with genetic disorders. But I don't currently have the words for that and I get tired of being ganged up on by boatloads of internet strangers who want to inform me that I'm imagining things and my entire life experience is a hallucination and I don't actually know nothing about genetic anything. So let's just leave it at "I have this opinion and maybe it provides a way out of this trap and maybe it doesn't."<p>Because the current approach of inventing incredibly expensive drugs that might provide a cure is essentially failing as a method. And all other approaches are so awful it's why such things get labeled "dread diseases" -- because what it does to your life is so terrible that if a doctor were not prescribing it, it would be in violation of the Geneva Convention. It's just not humane.