The one silver lining of having such a terrible diagnosis should be that you are immediately unbound from normal FDA requirements and are able and try anything in the pipeline that might work.
Jake Seliger (RIP) wrote about his frustrations with it also:
https://jakeseliger.com/2023/07/22/i-am-dying-of-squamous-ce...
https://jakeseliger.com/2023/08/02/if-youre-involved-in-drug...
The first problem was finding the damn trial in the first place since nobody wants to share information as it might tell their competitors something.
And then he had to pass the prerequisites for the trial. The problem there was navigating the US health system that moves like a glacier and either couldn't get a test run fast enough or wouldn't take a test from somewhere else.
Finally, after all that, then you reached the "Right-to-try" level where there were different questions of whether to subject him to the treatment based upon whether the doctor thought it might or might not be effective.
And through it all, he and his wife understood that the odds were very, very bad.
To me, the counterargument to "Right-to-try" are the Alzheimer's drugs. The underlying Amyloid hypothesis appears to be broken and fraudulent and all the drugs based on it have failed miserably. And, yet, one got through FDA approval because the consumer push is so huge and everybody wants something--even if it doesn't work.
There is big money behind pushing the boundaries--be very careful about eroding them.
If the drug is available to the desperate, then the push by the public to shortcut the process goes away and the process can be done correctly. Those who are desperate can try, with the understanding that it might not work. The process can continue unmodified because there isn’t a pressure to get the drug out to the public because it is available.
If you allow that, you're back to HCQ for Covid territory. Or all the autism "treatments".
The problem is that most people think of medical outcomes as having 2 results when there are 4.
The 2 everybody thinks about are "drug given->patient gets better" and "drug not given->patient gets worse". But there are two more cases.
"drug not given->patient gets better" is wonderful in the super-rare cases when it happens and is totally unproblematic.
The problem is that the case "drug given->patient gets worse" is common--doubly so for experimental treatments--exponentially so for experimental cancer treatments. And the problem is that it can be due to either the disease or the drug or both. This case is the one that you can't explain in a soundbite. And it's one of the big hurdles in drug discovery.
The vast majority of people simply will never get this. We know this. We watched it play out in real time--multiple times.
Sure, if you made everybody pass a Bayesian statistics exam before they could get their treatment, that would work. But then you'd be "gatekeeping a cure".
Stupidity is the norm, not the exception, and greedy, evil bastards are not unrare. Your rules have to, sadly, take that into account.
It is not actually super rare. One might even say this is the most common case. I guess what you can say is that spontaneous remission is super rare to happen for “serious” diseases. But that is circular reasoning because we kinda define serious diseases by the fact that it is rare to just get better without treatment from them.
Why is this important? Because this is one of the main wrinkles with drug testing. If some people just get better on their own then you have to be very carefull with statistics and double blind protocols. Because if not you might confirm your belief in an innefective, or maybe even harmful treatment by random happenstance. Therefore i would say this case is also one of the big hurdles in drug discovery.
Otherwise i agree with the general gist of your comment.
A child gets a run of the mill cold, parents give them all the OTC things, kid gets better and assumes it must have been because of the drugs. In reality, they didn't need any of it.
One of the difficult things about ethics (including medical ethics) is that everyone has slightly different opinions.
But in MY opinion, if someone's medical situation has an extremely high likelihood of extremely poor outcomes (terminal cancer and severe Alzheimer's both qualify), then I think it is perfectly acceptable to allow them to use a treatment which is well understood to have no useful effect and many harmful side effects.
I have an ethical problem with encouraging them to do this; I have an ethical problem with profiting from them doing this; but I think that prohibiting them from doing it (as we do today, mostly) is also wrong. EVEN IF the patient is "too stupid to understand it correctly".
There's a lot that can be said on this, but it's harder to defend that position if the decision has an effect on others. The obvious situation that arose during the pandemic is someone experimenting and then creating more work for an already overwhelmed hospital system. Then you have to ask about paying to clean up the mess - should it be health insurance, that is paid by everyone else? If you die, what are the effects on your family? How do you draw the line where "well they don't have much time left anyway" vs "they killed themselves experimenting with flu treatment"?
> Clinical use of this treatment was driven by women with breast cancer and advocacy groups, such as ACT UP; they believed that the FDA treatment approval process was too slow.
The problem with a nominal fee is fraudsters can be really clever at finding ways to bump up production costs.
You might give the company doing the research relaxed liability, but even then you'd want to be careful. After all, you wouldn't want a coal mining operation conducting "research on lung cancer treatment" by sending in miners without PPE.
If this is a legitimate research into curing cancer then eating the cost to treat test subjects is the least a company could do. The results are what's can be worth a lot of money.
Fraudsters simply love preying on the desprate.
My concern with lowering the bar for advertising such services is it makes it easier for someone to jump in with "Our clinic is actively researching cancer treatments and offers free experimental drugs".
I know that a drug company can't skirt regulations around clinical trials simply by making participation free (in fact, clinical trials usually pay the subjects.)
Here's the scenario I have in mind.
John has an uncommon cancer X with no cure. John contacts Cancer R&D Corp. John pays 80% of his life savings to R&D corp for research into his specific cancer X and provides himself as a test subject in the meantime. R&D corp attempts to find a cure for the disease, if the mission fails and John dies, in the future if other patients with cancer X appear, they can take over from where they left. Payment can be split into an upfront half for research and bonus paid upon a successful cure (or year of quality life milestones) to give incentives for actual success. So as to avoid companies from benefitting from never finding a cure.
That already happens very frequently. For example, bleach enemas to cure autism [1]. Colloidal metals to cure cancer [2]. And a whole host of other cure-alls.
Now imagine you have a mechanism where they can claim to be legitimate research AND there is an incentive to bilk people out of 80% of their life savings.
Heck, even imagine what happens if legitimate for profit companies can use this route for revenue generation. If they have a route to sell drugs which has less regulation and liability, why would they ever release a drug through the full FDA process? And why, you might ask, is regulation needed for this sort of stuff? Well, think Vioxx.[3] Sure, maybe this cancer drug works but it also might give you a heart attack. Without getting it fully approved or researched drug manufacturers have no reason to actually look into downstream effects. They have every incentive to just keep it on the market (like they did with Vioxx) and ignore evidence of adverse results.
Taking profit out of the equation makes it so that the research isn't and can't be endless. It makes sure there isn't a perverse incentive to make lethal drugs that work good enough for some diseases. I'd love it if we could have a win win, but the free market loves to reward bad actors.
[1] https://www.nbcnews.com/tech/internet/moms-go-undercover-fig...
[2] https://www.healthline.com/health/colloidal-silver-cancer
So?
Because there's ilegitimate companies you should outlaw legitimate companies?
Should we ban banks and stock brokers because ponzi schemes exist? What makes you think there would be less ilegitimate companies, it's possible even that there would be more ilegitimate companies.
"Heck, even imagine what happens if legitimate for profit companies can use this route for revenue generation. If they have a route to sell drugs which has less regulation and liability, why would they ever release a drug through the full FDA process? And why, you might ask, is regulation needed for this sort of stuff?Without getting it fully approved or researched drug manufacturers have no reason to actually look into downstream effects. They have every incentive to just keep it on the market (like they did with Vioxx) and ignore evidence of adverse results."
I'm not an expert on the subject, but I do know that the subject of the article and what you are talking about are 2 distinct domains. One thing is developing a drug or treatment for a common disease. And another is treating a specific patient. Right? Two very distinct services/products with very different regulations. I do know at least from the import regulations my country has laxer requirements for infrequent diseases. Of course a treatment for diabetes can support stricter regulations than for Cancer of the Biliary Duct.
Additionally what I suggested wasn't even treatment, I proposed just Research & Development, this is already possible through donations.
No, you should have regulations and enforcement to ensure that stop illegitimate businesses from operating. Those regulations might make it harder for legitimate businesses to operate but that's to ensure public health isn't sabotaged by bad actors.
> Should we ban banks and stock brokers because ponzi schemes exist?
No, we banned ponzi schemes through regulations. That's what I'm proposing.
> What makes you think there would be less ilegitimate companies, it's possible even that there would be more ilegitimate companies.
There's always going to be people looking for loopholes in the law to make a buck. That's why the law needs to be constantly updated and these loopholes closed. You'll never 100% close everything, but that doesn't mean you shouldn't be constantly looking for improvements.
> I'm not an expert on the subject
You should read into it. The fact is that, particularly with medicine, we have to be careful about the leeway we give to companies. You simply have to assume that anyone selling something is amoral. Without regulation, companies can and do run the calculus of "How much will these deaths cost us vs the money gained from selling the product".
Vioxx isn't an isolated example either. Look up "Dalkon Shield", a product that had about a 1% chance of causing sepsis which killed and crippled literally hunderds of thousands of women. A product that, after this was found out, after the lawsuits flew and it was removed from the market, was shipped to and sold in Africa for several years.
My point is, that whenever anyone starts talking about relaxing regulations and making it easier for a drug company to profit, we should be thinking about the above abuses. Because companies can and will abuse the system.
You may still say "so what" but you should be thinking about "So what if the treatment cures cancer, if it causes a stroke, heart attack, or premature death when other maybe less effective treatments may have extended life". Cancer isn't the only thing that kills people and there are fates worse than death that drugs can cause.
> Two very distinct services/products with very different regulations.
The discussion here is how do we make individual treatments like the one in the article more available. The points I'm raising is that any increase in availability needs regulations to ensure it's not abused. I've given specific examples where regulation/enforcement has been lax which has allowed grifters and drug companies to directly and knowingly harm the public.
We just have a very different view. I think regulations HELP businesses.
For example, a well meaning company wants to make a cure for AIDS, a good regulation would require a study of length and size proportional to the volume and lethality of the disease. If the study is small, the regulation may allow small trials, and its first batch of patients be itself a trial for bigger batches.
If there were no such regulations, the well-meaning company may have had a lot of success, but maybe they would have gone to market too fast and detected a side effect in a big trial instead of a small trial.
Conversely, a terrible company might either choose to comply with the trials, and genuinely pivot to legitimately seeking a cure. Or avoid regulation altogether.
Good regulation is not an equal cost imposed to both parties, and never the cost is higher to the good parties. Regulation is an incentive that when followed leads inevitably to the results desired.
"That's why the law needs to be constantly updated and these loopholes closed. You'll never 100% close everything, but that doesn't mean you shouldn't be constantly looking for improvements."
Also not at all how I view the law. The law is written once, what is updated is the case law, the court rulings. Only when there is a change in technology other foundational changes, or very biig learning cycles (50 years), do we make another attempt at the same problem. You don't update a law every 3 years patching for loopholes playing a game of catch me if you can.
Fraudsters and scam artists are happy to rip off people trying to avoid death.
1- Honest researchers who can't charge too much. 2- Unscrupulous quacks who will take all your money regardless of law.
With the second option being much more common due to incentives. If however you allow research facilities to take donations/payments to fund research related to a specific disease, then you will increase the incentive for legitimate companies, even if the odds are still low or close to 0.
Jessica Tanenbaum, "Delayed Gratification: Why it Took Everybody So Long to Acknowledge that Bacteria Cause Ulcers" (2005)
<https://www.jyi.org/2005-february/2005/2/9/delayed-gratifica...>
Do you doubt there are people who refuse to accept the consequences of their own actions and seek to place blame anywhere else they can?
Or indeed some third party might do the claim with hindsight. Prosecutors or whatever. People can be irrational, in general.
It would require legislative bodies to pass laws against the wishes of the people. Maybe in China :)
Now what? The irrational FAR outnumber the irrational.