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A million-dollar drug

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Re: A million-dollar drug

#141

Post by Nasrudith »

rileymat2 wrote: post #119
JamesBarney wrote: post #56

Earlier quoted context omitted.

But of that 100 million dollars the bulk was safety testing due to regulation. So you're right it doesn't make economics sense but for a specific reason. As a society we have to strike a balance between safety and efficiency. And I don't think we've struck the right balance. Many times erroring too much on the side of safety, especially with rarer and more fatal diseases without good current treatments.

"But of that 100 million dollars the bulk was safety testing due to regulation." How do you differentiate the cost of safety testing that should be done and the cost of safety testing due to regulation?

I think the answer is risk tolerance related for how much they would be sued without the testing. Many companies do risk analysis above and beyond what the minimal regulatory requirement if they feel it is more cost effective. That would be the number used in comparison.

That said the regulatory premium may still be the right thing to do given horrifying human costs and potential to leave everybody else holding the bag when they go bankrupt with expenses far beyond what they can pay. Even if you seize assets of the entire board in a criminal proceeding it is still possible to do more harm that can be repaired by the courts and thus make regulation the rational thing to do in addition to the right thing for a circumstance. Or the regulations could do more harm than good. In general regulations themselves aren't inherently good or evil but circumstantial.

Re: A million-dollar drug

#142

Post by mirimir »

> But it's the only way scientific discoveries ever get to patients, because universities don't make drugs.

Why not?

If there are so very few patients, a lab could easily produce enough for them.

Re: A million-dollar drug

#143

Post by kentosi »

quotemstr wrote: post #59
wpietri wrote: post #39

Earlier 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…

> 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. Are you suggesting that prices don't have anything to do with scarcity and are just set arbitrarily by social forces? That doesn't seem quite right.

Keep in mind the scarce few people that are actually affected by this disease. No doubt that this factored heavily into the high price.

Re: A million-dollar drug

#144

Post by ineedasername »

tptacek wrote: post #35

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. But they don't. Health systems in Europe apparently refuse to pay f…

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 $300,000k/year, that would seem a pretty good sweet spot pricing that insurance companies would clamor to approve due to the savings over annual costs.

Re: A million-dollar drug

#145

Post by donarb »

School-Cotton wrote: post #108
torpfactory wrote: post #32

Earlier quoted context omitted.

Especially for lifesaving medical patents. If the drug isn’t being produced, or the price is too high (I don’t know how this would be defined), governments should be empowered to seize the IP and solicit bids to produce the invention. We move heaven and earth to save lives in natural disasters, why do we move so slowly with certain medical conditions?

Then nobody would ever bother investing in new drug development. A better model would be for the state to fund this research and have the results be public domain.

Unfortunately, this happens all the time, but not for the public good. Research universities use patent portfolios to increase their revenue by licensing inventions to companies that can manufacture the product.

For example in 2014 NYU earned $214M in IP licensing and nationally led the period from 2004-2010.

Re: A million-dollar drug

#146

Post by ineedasername »

ctdonath wrote: post #136
InTheArena wrote: post #131

Earlier 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.

And no one was willing to pay this much for the drug, but the inexplicably decided no revenue was better than lowering the price.

Re: A million-dollar drug

#147

Post by ineedasername »

tptacek wrote: post #40
WhompingWindows wrote: post #36

Earlier quoted context omitted.

Interesting post. Hopefully some innovators can come through and figure out a way to make it more cheaply. Even if it's expensive now, if humans exists for 100's of years, then eventually the price will come down when this inventor is toast, right? Either that or people will figure out cheaper, DIY ways to do some of this biological manipulation.

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.

Re: A million-dollar drug

#148

Post by gus_massa »

ekianjo wrote: post #99
gus_massa wrote: post #75

Earlier quoted context omitted.

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.

The point is to discover real medicine that really cures the people.

If there is no clinical trial, how are you so sure that this treatment is better than the previous one?

You can trust the gut feeling of the experts, but that is not enough. The rules for a clinical trial are complex, but they are created to avoid some mistakes (or frauds) of the past.

In particular of the recent changes is traying to force preregistration. https://en.wikipedia.org/wiki/Clinical_trials_registry Because it's too easy to massage the results after the trial to get a lot of results and then report only the "good" result and hide the others. The "good" result is an statistical fluke, so any attempt to reproduce the trial will fail.

Another older requisite is having a control group with a double blind setup if possible. For example, they tried this drug in 31 people. Did this 31 people get better average results than the other patients with the old treatment? They are probably applying the experimental treatment only to people that is not near death. If someone is going to die in a month and is very weak, you don't want to kill him because the new treatment has an unexpected side effect, or get blamed that the treatment killed him instead of the previous condition, or that the family later says that he was too weak to give an informed consent, or get random problems because the organs are too broken, or ... So there are some explicit and implicit filtration rules. Does the patient has any additional illness? Is s/he to young or too old? When you take the average result of the patients in the study and the patients with the normal treatment, are you using the same explicit and implicit filtration rules. What about long term effects? What about the periodic medical checks during the trial that detect early signs of other problems? Does they eat more healthy food during the trial?

So you need a control group chosen at random after you decide to include or exclude the patient in the study, so you try ensure that you have the same population. Preferably a double blind experiment because the patients may give inaccurate reports if they thing that the treatment is good, and the medics and nurses also are influenced by their expectations.

And in these trials, the control don't get a placebo, but the usual treatment, because using a placebo when there is some good treatment is unethical. This is a rule, because some people try to do this anyway, because it's easier to show that the new treatment is better than the placebo than to show that it's better than the current treatment.

Also, they have only 31 patients, that is barely usable to make statistics. The error bars for 31 subjects are huge (something like 5%). The difference must be huge to get a statistical significant result.

You can trust the gut feeling of the experts to think new treatments, but they must prove that the treatment is effective. Otherwise, you can't distinguish between a expert with good ideas, a moron and someone who wants to sell you snake oil.

Re: A million-dollar drug

#149

Post by ineedasername »

mirimir wrote: post #142

> But it's the only way scientific discoveries ever get to patients, because universities don't make drugs. Why not? If there are so very few patients, a lab could easily produce enough for them.

Yeah, they had to make some quantity to run their trials. presumably it cost much less than $1mil/dose or they'd not have research funding at their level

Re: A million-dollar drug

#150

Post by akvadrako »

refurb wrote: post #128
apo wrote: post #106

Glybera was never sold in North America and was available in Europe for just two years, beginning in 2015 The patent will expire within a few years, setting the stage for generic manufacturers to step in, but the article doesn't mention it. 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…

As you can see from the lack of biosimilars in the US, manufacturing a biological is a hell of a lot more complex and expensive than doing it for a small molecule. And in fact, since this is version 1.0, I’ll bet a better version comes out before any generic company launches.

If they come out with a new version, they must be expecting it to sell in a quantity that makes the investment worth it. So maybe the pricing worry is just a temporary concern.
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