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.
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.
To me, the Alzheimer’s drug story is an argument for right to try laws.
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.
Except that the drug has negative side effects and does not work-not even with the most optimistic interpretations.
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.
> "drug not given->patient gets better" is wonderful in the super-rare cases when it happens and is totally unproblematic.
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.
I think this belief is how you end up with people who think you need some cocktail of OTC drugs every time you get a cold.
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.
That doesn't sound like an argument against the right to try, that sounds like an argument for requiring specific, intentionally frightening language around informed consent. The thing is, probability is a bitch, and emotionally filtered probability is the devil. An experimental treatment for a terminal illness can be 99% likely to result in a quicker or more painful death, but is it really right to deny the 1% chance to someone who understands the statistical choice in front of them?
Another facet of this is: should insurance pay for it? Or should the patient bear the financial burden when they want to try a shake-oil cure? Especially in this case when the drug is now FDA approved?
> Except that the drug has negative side effects and does not work-not even with the most optimistic interpretations.
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".
> 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.
With the caveat that they can only be charged a nominal fee, say the actual production cost of the drug. It would be good to avoid fraudsters stripping desperate people if all their assets
I'd probably go further and say it must be free of charge.
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.
Actually, thinking about this further, even free of charge could be problematic. I can 100% see a "cancer research and spiritual healing" clinic popping up that uses the promise is a cure as a way to get someone in the door so they can later be sold on energy healing to increase effectivity of the sugar pill they give away.
Depends on how it's marketed. As long as they avoid making specific health claims (say "boost immune function" instead of "cures disease X") they can get away with it. If it's "homeopathic" they can even be sold on the same shelves as actual medicine, with similar looking labels.
Ish. You can't say that a pill or treatment cures cancer if there's no evidence to back that up. Of course in private I'm sure that's said all the time.
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 could be wrong, but my understanding is that "free of charge" is already legal, and the FDA only regulates drugs that are sold and not ones that are given freely
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.)
I don't see the issue or point of limiting profitabilit, what am I missing?
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.
The issue is that fraudsters would like nothing more than to call themselves R&D and to sell BS to desperate people looking for 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.
"The issue is that fraudsters would like nothing more than to call themselves R&D and to sell BS to desperate people looking for a cure."
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.
> Because there's ilegitimate companies you should outlaw legitimate companies?
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.
"regulations might make it harder for legitimate businesses to operate"
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.
I responded to this in other posts, but this would be throwing the baby with the bath water. And it's especially bad as the good suffers more. Legitimate companies will follow the law, but the ilegitimate actors will just sell mystical crystal anyways.
Conversely, if you ban actually charging for last-attempt experimental research services, then those desperate people will only be left to seek:
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.
This is an hilariously straightforward and brilliant idea. It's no holds barred, but the goal has got to be a real, approved drug in the future. Not profits right now.
I think you also need a caveat that failures must be recorded, and a specific treatment can only be used so many times without success. That will avoid providrrs who are self-deluded about the efficacy of their favourite drug/therapy.
Patients with incurable or hard to cure diseases are susceptibile to be exploited by companies or doctors that want to try unproven treatments on them. On the other hand, telling someone they are not allowed to do everything they can to save themselves is equally bad.
Centres of disease control (in the US and elsewhere) track morbidity and mortality on a wide range of conditions, which is one standardised answer to your question. There are also (a small number of) independent or unorthodox researchers who pursue specific avenues. You'll of course find the frauds amongst these (e.g., Andrew Wakefield, who promulgated anti-vax theories and falsified research toward same), but there are also notable medical advances which have emerged in such contexts, such as J. Robin Warren and Barry Marshall's identification of Helicobacter pylori as the principle cause of stomach ulcers, rather than stress:
Jessica Tanenbaum, "Delayed Gratification: Why it Took Everybody So Long to Acknowledge that Bacteria Cause Ulcers" (2005)
The “might work” is doing a lot of heavy lifting in this sentence. How strong must the evidence be for the hypothesis of efficacy to be considered reasonable?
The person being treated, or their family, might change their mind after the treatment fails, and cry bloody murder. And get sympathy. So the only defense is to deny treatment. In hindsight, with the bad consequences fixed, they will claim they should have been prevented from getting the treatment and whoever did not prevent them is at fault.
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.
The legislative institutions are not isolated from the people. The opposite in fact, in democracy they must respond to the will of the people. So if enough people are irrational and wish to assign blame somewhere else after they assumed a certain risk (and/or enough people support this even when not involved), there can be no legislative solution.
It would require legislative bodies to pass laws against the wishes of the people. Maybe in China :)
I find this comment somewhat disingenuous. People have been arguing about allowing more experimental end of life treatments for at least 40 years of my life. I grew up during the AIDS crisis and there were all kinds of quacks selling this or that to take easy money from desperate people. If what you say is true, then why hasn't it already happened? After all, isn't 40+ years long enough to argue about it?
Because decades of arguing in no way implies that "the truth" or correct policy will fall out of politics. Abortion and marijuana legalization are two contemporary (and much more prominent) examples.
It sounds like this might be a bit like off-label medicine though. It would need to start with a doctor. Maybe any plan needs an independent sign off from another doctor who is qualified enough.
