7/67 Children Receiving Skysona Gene Therapy Develop Blood Cancer
nejm.org
nejm.org
A single course of treatment of this cure costs $3 million, making this the second most expensive drug in the United States
So gente treatment for very serious and fatal disease causes very serious issues in 1/10 of patients?
As opposed to, you know, the effects of the original disease?
I think I would go with this treatment regardless
Presumably the other children may manifest cancer down the line. Off target insertion was random
Also the blood cancer has already killed one of the patients
There are times where tolerating treatment-related mortality is the best alternative available.
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.
Without the off target gene insertion, it sounds like it'd be a winner.
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.
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.
> > ...if you are informed of those risks...
It's the fact that options laid out on the table with unknowns can be preferable to people than no options, or fewer options.
The same dichotomy exists for crime vs agreements:
- You'll be compensated if a party to an agreement breaks a clause or costs you money (say they fail to provide $30k of goods to your business)
- But if someone steals your car, the government won't put $30k in your account for a new one.
The same loss occurred. The second case was totally unwarranted, yet the individual outcome is worse.
Why? Because doing it any other way would make things worse. People would lie about stolen goods, conduct insurance fraud, etc.
Unfortunately in some situations, things "which are worse" are a different category and don't have an answer with government intervention.
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 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".
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.
When the alternative is degenerative illness like psoriatic arthritis, sometimes you just have to roll the dice.
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.
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.
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).
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.
https://www.pc.gov.au/inquiries/completed/pharmaceutical-pri...
It also pays an additional $5B p.a. in drug subsidies.
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.
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.
Next up: (e^-1)/3*3 children who got the COVID shot...
There's this saying in UX: don't make me think.
When the sample sizes are small, it's better to just give the fraction IMO.