What’s up with this drug now?
Sounds like it’s patents and IP protection should be up.
I’d imagine you could get a single dose manufactured 1 off for $100k by a lab.
A million-dollar drug
191–200 of 215 posts
Re: A million-dollar drug
#192Earlier quoted context omitted.
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…
according to the article, and a few google searches outside of it, the drug was that good. The article's implicit conclusion was that the drug was priced too high, and offers evidence to back that claim. You seem to be assuming a perfect rational market here that has a rational justification, but real markets don't always work that way.
As someone who works in the industry, the article was a 30,000 ft view and didn’t do a good job of digging into the issue.
Regardless, it looks like the drug does reduce pancreatitis attacks, but only by 50%. So it certainly doesn’t “cure” the patient.
The clinical trials were also quite small, so possibly the national payers said “$1M is reasonable if you do X, but you don’t have the data to convince me you can do X.”
Re: A million-dollar drug
#193Earlier quoted context omitted.
Here is the problem with this statement - it assumes that the drug is worth the maximum amount that they could extract from the customer (insurance companies, patients and governments for it). Nothing says that they couldn't sell it for less, just that they thought they could get this much for it.
A thing is worth what another will pay for it.
healthcare should never be one of those things.
Re: A million-dollar drug
#194> "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?" As terrible as it sounds, this logic seems sound to me. If we think having this type of d…
There another way to read that sentence :
>>> Hundreds of millions of investor money has gone into the company, and if there is no return for those investments,
Considering that there are patients who needs it, you can divide the price by 10, this way you make sure everybody will pay and the only sacrifice you make is your ROI to be on a 10x longer period.
The problem with investors is that they want tons of benefits and in a very short time.
They could as well sell the IP if they don't use it anymore.
I understand that this is business, but here I see at as "if we fail, then everybody will fail".
Re: A million-dollar drug
#195Earlier quoted context omitted.
Nope, and I didn’t have any noticeable immune response. Literally no side effects to speak of, it was all very easy, just a simple injection and overnight stay in the hospital for monitoring. 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 stru…
How was the recovery process? Did you feel better immediately or was it in small amounts and over several weeks?
There was no recovery process to speak of in a traditional sense. Since this is a chronic condition, I’ve just simply gotten used to the aches and pains over the years. I really didn’t start to feel any different until a few weeks ago, when I realized I had forgotten to take my pain and arthritis mediciation, but I wasn’t having any pain that day. I went off it and I haven’t had to go back on it. My joints all feel noticeably better, there’s basically no pain anymore when I walk. I’d have some days like this before but nothing this long sustained before.
It’s too early to tell but my doctors think that with a sustained factor level, my body isn’t under as much stress from the bleeding all the time and can focus on repairing the damage caused from a lifetime of near constant bleeding.
Re: A million-dollar drug
#196Earlier quoted context omitted.
I don't think he understands pricing at all. Market price isn't arrived at via a rational process. It's an inherently social process arrived at by negotiation between producers and consumers. And it's especially weird that he thinks pricing shouldn't be political when his product has a government-granted monopoly and the bulk of its customers will be government-run health-care systems and government-licensed insurers…
> Sure, there's a real question of how to pay for treatments for rare diseases. But the millions in development are a sunk cost. To take his ball and bat and stomp off would be disappointing in any circumstances. But when people will die because of it? If people actually cared about treating patients, they would be crowdfunding that treatment rather than trying to fleece the people who invested man-decades of work in…
Oh, you think people should get together and pay for drug development and medical treatment of people with rare diseases? Good idea. In fact, maybe we should create some sort of system where funds are collected widely and then distributed through systems that judge merit, utility, and cost effectiveness.
Oh, wait! We already did! This literally exists in most countries. In the US the government funds about half of all basic research. In many biomedical fields, that number rises to 80-90%. Treatment too is covered by "crowdfunding" through government-supported health care systems.
The guy's grumble here is that he doesn't like the system. And there are plenty of fair arguments to make there. But his complaint isn't, "We need to do better funding cures over treatments," or "The government should support rare diseases better." It's a confusion of of free-market slogans inside a context that not only has little to do with a free market, but one where he only has something to sell due to extensive government funding and a government-granted monopoly.
Free-market approaches work well when figuring out the price of beans that you buy every week at the market. They are of very narrow utility in exotic, life-critical treatments for rare diseases. I'm still all for applying them where we can. But this guy's gripe sounds more like free-market fundamentalism (or straight greed) than any thoughtful understanding of how to fund public goods.
Re: A million-dollar drug
#197The 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. But it's not working here. The patent holder is effectively saying soci…
It's also such an easy fix too. If a company isn't actually using a license or copyright they should loss all claim to it.
Re: A million-dollar drug
#198Earlier quoted context omitted.
It's not even clear that this therapy is locked up because of IPR. If you read uniQure's filings, their patents appear to be for treatments for other conditions (notably Huntingtons), and their protection for the LPLD therapy is under "orphan drug" protection.
Yeah, seems ripe for someone to pick this up and charge $100k a dose. Insurance companies would look at that as a deal compared to $300k/year for the alternative replacement therapy.
another issue I have is with the claim from the article that they constantly have to produce new batches because the old product expires. but with such a small group, it would seem better to first make a comprehensive list of LPLD sufferers, then make a single batch for all of them, and repeat every say 5 years as new cases are found... yeah on average a patient waits 2.5 years...
If there is a long list of rare diseases, a company could specialise in manufacturing doses for rare diseases outside of the classical "order a dose (from continuous production) as a patient is discovered". So they might make a specific rare drug once every X years, but with enough rare drugs they might fill the rest of those years with preparing for and then making and testing single batches of other rare drugs...
Re: A million-dollar drug
#199Earlier quoted context omitted.
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…
You’re welcome and yes I agree about this being most impactful for children. Hemophilia had a big effect on my life, both physically and psychologically, as well as my family. It’s hard to grow up knowing you’re so different from others. Now, someone born with hemophilia may never even have to know. It changes things dramatically. In a single generation we’ve gone from being able to treat the condition at all (replac…
a next step would be for the virus to fix the cells from which the gametes derive, then your future offspring would be cured as well
Re: A million-dollar drug
#200Earlier quoted context omitted.
The drug didn't cost $100 million to make. It cost $100 million to prove that it works in order to get it approved for sale in Europe . In a world where you could just put a drug on the market (where "the market" is, say, hospitals), the drug would have cost far less, and so the inventor would need to recoup far less. Note that I'm not arguing for complete deregulation of drug manufacturing. Testing for product integ…
The number of drugs that have gotten FDA approval at a billion+ cost only to be later rescinded is pretty massive as well... so it's not like the current system is fool-proof.
There have been a number of FDA and even OTC approved NSAIDs which may have killed off millions... I don't know who profits from the present system, but it ain't patients.