A Company called BlueBirdBio was developing a gene therapy treatment for children with a rare neurological disease - Cerebral Adrenoleukodystrophy. It looks like the gene insertion went off target in many cells resulting in blood cancer, with one of the children dying as a result.
A single course of treatment of this cure costs $3 million, making this the second most expensive drug in the United States
There's all kinds of approved treatments that have a high risk of a bad outcome; if you are informed of those risks, you're not going to prevail in such a lawsuit.
Indeed, hoping to be compensated for an ordinary bad outcome through a malpractice lawsuit is one small reason why the US medical system is so screwed up: bad things can happen without it being anyone's fault, but we require to lay blame to get any recompense. Ideally, we'd insure patients against those bad outcomes.
You can if all the risks were not disclosed, especially if others come forward and it becomes a class action lawsuit. If a waiver is all it takes to shield oneself from malpractice lawsuit, then it would not be such a big problem.
Yeah, I didn't mean that the patients or their families should or would win the lawsuits, but the lawsuit potential still seems like a potential cost driver.
Nah, you usually have to sign all kinds of waivers to get access to this sort of thing.
It's customized, and very few treatments are sold for the amount of research and effort put into it, compared to some chemical formula that they can hand off to a factory to mix and stamp into millions of pills.
How do you compensate for death? How do you insure patients against death? Do you realize how cynic your comment is? Even though it is full of facts, these facts are pretty much beside the point. Ruining someones body isn't the same as a casual parking damage. I, for instance, have been a victim of deliberate malpractice as a child, which resulted in 100% blindness. NO money on this planet could actually compensate for what I have to cope with on a daily basis.
> But if someone steals your car, the government won't put $30k in your account for a new one.
I understand your point and get that you didn't say this to argue with what I've said. I just want to emphasize that the situation here is different.
Here, though, we have a situation where people who are significantly harmed medically try and find malpractice even if none occurred, and juries are usually sympathetic. The consequence is medicine has become a much more defensive and adversarial system.
I'm sure that an effort to insure for morbidity from bad outcomes will cause fraud, but it might also save a lot of malpractice litigation costs and reduce the amount of resources spent on defensive medicine, too.
> It's the fact that options laid out on the table with unknowns can be preferable to people than no options, or fewer options.
Assuming you're talking about me, that's not exactly my point. I didn't intend to make any assertions about what patients or their families should or should not do, or in general any normative points (though I did use the word "justification", which I shouldn't have).
Rather, what I was saying is that businesses that aren't profitable cease to exist, and if the business's product is an experimental medical therapy that could end up killing a substantial fraction of its patients, the business might lose money when those patients' families sue the business. It's fairly predictable that a good fraction of experimental therapies will have this outcome, even if you don't know which ones in advance. Consequently, businesses that don't price it into their business model will, in most cases, go bankrupt after their first few successful therapies, making them unattractive for venture capital.
So the high price tag is somewhat predictable, whatever you may think of its moral status.
Separately, your comment seems to imply that damages on breach-of-contract and similar civil tort cases are paid to the plaintiff by the state rather than the defendant. That is not correct. They are paid by the defendant if they are paid at all. In the US, you can also file a civil tort case for conversion against someone who stole your car and get awarded damages in the same way.
I understand what you mean and we seem to agree which is great.
I also find economic arguments like you make here generally convincing, arguing against this (that risk increases costs which must be priced in) would be similar to arguing against the Laffer curve. This is essentially close to my position, but different to the person I originally replied to.
I do have an optimistic bent that, through either ignorance, arrogance or risk-seeking, people actually over-risk on experimental therapies, for example, launching Biomed spin offs. Because the personal gain (curing a disease, helping people, or becoming popular) can be so high, it can motivate risk beyond other busiensses. This is probably where I most differ from you: I know many businesses which should not exist, have never turned over profit, and maybe never will. But a rich family, or the savings from a lifetime of earnings sometimes get put into these shots at success. It's not like they get cut once the net profit reaches 0. Same with local shops: A number of the local shops you can't understand running profitably survive because they have paid-off mortgages and only have stocks/staff/licensing to deal with. It makes little financial sense to run them over renting the shop to someone else for more profit, yet people do.
Perhaps this extra reason to stay invested/risk on Biomed is matched by the fraud in that industry, though (Hello Theranos).
Regards the later point, I understood that though my comment could have been phrased better. The meaning was "the government will force [as necessary by asset seizure or bankruptcy] the breach-of-contract party to pay you".
People can be harmed by medical procedures when someone does something wrong, but people can also be harmed during medical procedures without anyone doing anything wrong.
Ideally patients would be protected not only if someone made a mistake, but also if they were one of the unlucky people who got a bad outcome from a procedure that is normally beneficial.
Is a 10% risk of potential death a good justification to cure the disease?
Actually - more realistic would be, What if you had the disease in question and knew the cure might (then again might not) have some unknown side effect, up to or even exceeding a 10% chance of death. Would you take it?
Obviously the answer is hidden somewhere in the details but most of those details are unknown or unknowable.
Which is to say I bet any legal action's success will depend upon just who knew what and who they told.
>Actually - more realistic would be, What if you had the disease in question and knew the cure might (then again might not) have some unknown side effect, up to or even exceeding a 10% chance of death. Would you take it?
Yea.
That exists commonly. It's called high risk surgery.
This genetic disease is more like you die in ~5 years and also all the nerves start to fail including ones involving thinking, memory, personality (cerebral)
Same with cancer truly. Many patients treated for childhood solid tumors go onto get blood cancers due to the treatment/chemo being genotoxic. Oh, and infertile also.
So I think gene therapy should be considered totally valid when these other potentially deadly treatments (surgery, chemo) are used - all of it in deadly situations. Gene therapy also has ways to go, lentiviruses are well known to randomly integrate sometimes in the wrong spot (a tumor suppressor gene) whereas CRISPR can be more targeted.
On the other hand, cerebral adrenoleukodystrophy would be expected to kill a significant fraction of those 67 and rob the rest of their cognitive capacity within a few years.
There are times where tolerating treatment-related mortality is the best alternative available.
Which is why these dangerous experimental treatments are used on patients with these classes of terrible diseases first. If it doesn't work or fails horribly it's not much different than what would happen naturally.
> Also the blood cancer has already killed one of the patients
This isn’t a fair interpretation of the death - the treatment of the drug-linked cancer bears a risk of multiple complications. One of these complications led to a patient’s death.
I must admit I'm not a very medically literate person - someone please break this down - is this good news or bad news? Were we expecting more people or lesser people to develop blood cancer?
We didn't expect the rate of oncogenesis from off-target gene insertion. It's bad news.
At the same time, this is a treatment for a terrible, terrible disease; so far the consequences seem smaller than what cerebral adrenoleukodystrophy would deal out on its own.
So it's bad news about something which is probably still a good therapy (but we sure would like a better one).
It's a death sentence, in the sense that you either die from it or you become a vegetable. If I'm not mistaken, the treatment cures the disease in 90% of the cases, so it's definitely worth rolling the dice. You literally have nothing to lose, and the payoff can be infinite. The Kelly criterion on doing or not doing the treatment is all in, all the time.
Pharma love when governments are on the hook for their outlandish prices. It's why there's a revolving door between public and private...to smooth deals like these.
If the government wasn’t allowed to hire from private, it would be starved of talent. That would be a great scheme to hobble any agency you don’t like.
You can cut it more finely but I think you still run into the same issue. The best candidates to run an agency that regulates a particular industry are going to highly overlap with the best candidates to be leaders in that industry, because they have knowledge and experience over how that industry works.
Sort of like how many top law students choose between clerking at federal court and joining big law, or do one after the other. And people who become judges often did both. If you ban people who worked in private practice from being a clerk or judge, you would have a lower quality judiciary.
Ultimately, I think you end up with the B team trying to regulate the A team.
Can you describe the incentive structure that makes sure Australians require less drugs in general? That's the bigger picture. Getting a discount on something you shouldn't need in the first place is not a win.
Honestly, $3 million for a tailor made (needs to be customized for every patient individually) single shot cure that saves a person's life is pretty reasonable.
The median lifetime earnings for an American is about $1.7 million though. For a mother, a father, and the patient, it might be worth it.
Lot of negativity in the comments here. The disease is likely to kill you without treatment and the "cancer" is not solid organ cancer like most people are used to thinking of but mostly (5 out of 7 cases) myelodysplastic syndrome (MDS) which is different and curable in some cases, as this paper demonstrates. If it were me, I'd take my chances.
What's worse Cerebral Adrenoleukodystrophy or the hematologic cancer? I mean if you just get cancer on top of the original disease that's obviously bad but if you're trading one problem for another that could potentially be a step in the right direction. I'm not an oncologist but I read The Emperor of All Maladies so I know a lot of blood cancers have high survival rates.
In spite of this horrible outcome, can this tell researchers anything about what causes the cancer? As in, if they know what happened (the problem with the insertion of the gene) then does that tell us anything about what might cause a cancer, and with this knowledge help prevent it or treat it in others?
To insert the gene, you use lentiviral transfection.
The gene can wind up inserted in the wrong location of the genome, causing a mutation.
Worst of all, natural selection can happen where landing in a region of the genome that promotes growth (i.e. cancer) will give those off target cells a selective advantage.
Inside the petri dish, Darwinian natural selection means the most cancery cells are the fittest.
There are ways of mitigating this. Like through selecting and screening monoclonal populations, but right now that process is painstaking and expensive.
Alternatively (and concurrently), we could invest into gene insertion technologies with lower probability of off-target effects.
Just one of the many reasons a treatment like this is expensive. You need to screen clones from every single patient because the treatment is unique to every single patient.
It's probably very similar to virus causing cancer that is well known https://en.wikipedia.org/wiki/Infectious_causes_of_cancer It may be helpful to understand why exactly this genetic treatment cused this cancer and fix it for future similar treatments, but not as generic improvement for all cancers.
A single course of treatment of this cure costs $3 million, making this the second most expensive drug in the United States