I am dying of squamous cell carcinoma, and potential treatments are out of reach
jakeseliger.com
jakeseliger.com
Anyone who has suffered through this disease or has a loved one that experienced it knows the feeling of helplessness.
More on Jake’s story below, and a link to the fundraiser to support him and his wife: https://www.gofundme.com/f/help-the-fight-against-cancer-wit...
Be kind and good to your loved ones. Life is short and no one knows how much time we have left. While the FDA’s antiquated bureaucracy does no good, the true enemy is the disease itself. I believe in science and I hope that someday no one else will needlessly suffer as my brother has.
You be good. I love you.
Stabbing backs to maybe get a pay raise will mean nothing to you or your family. It's just one more person who won't honor your temporary existence.
Be KIND.
I’m sorry for the very difficult times your brother is going through. I hope and pray that his situation improves.
This is such terrible news and I wish Jake and yourself the best.
>We get wise too late
>and old too soon.
>—Danish proverb
Quite.
I'm now four years older than my mother was when she passed away after two really exciting years. I can't remember the exact description of the killer but "squamous" was involved. Radio, chemo, surgery bordering on butchery etc. Bout one seemed to be in remission and all clear sounded only to be rescinded a few months later, second run was a negative outcome. Faith healer for a laugh "but you never know" - mum wasn't daft but when you are out of reasonable options, you might as well invent a few more!
However, there is still hope whilst breathe still flows and there is absolutely nothing wrong with hoping for a miracle. They do sometimes happen. Keep fighting and kicking and being a pain and only give up when it is obviously hopeless.
Love you too. Take care.
(Not sure about Cuba's current healthcare infrastructure).
Likely, Moderna doesn't want him to take the drugs because they will probably be ineffective this late in the cancer stage. It doesn't want its treatment to be associated with his death.
[0]https://www.fda.gov/patients/learn-about-expanded-access-and...
It's highly highly highly unusual to be offering an experimental therapy on compassionate grounds when conventional options that have potential to actually work well haven't been tried yet. In this post he only describes locoregional therapy and it appears he has not tried anything systemic yet.
The closest I've seen is we at least try a very short attempt of something in clinical use and then say patient refused/intolerable side-effects/treatment failure and move on. I can't imagine a circumstance where medical ethics would allow to skip a trial of validated therapy for something without even phase I data. It's not like this mRNA by Moderna is known to actually work.
Really the best and only chance for curative intent with most cancers is radical resection with negative margins which unfortunately did not work here (most likely due to micrometastatic disease and perineurial invasion). All of these new and fancy systemic options are not believed or intended to be curative-intent.
If we're talking about meaningful prolongation of life the best treatment would be a PD-L1 inhibitor like pembrolizumab if he has expression. He doesn't mention systemic/immunotherapy in his post but on the donation page his brother does so I'm unsure if he's in the early resistance/treatment failure arm for pembro, if that's the case it's very devastating.
He's correct that gem/plat chemo isn't great but it's something. Patients in his position are best served in clinical trials (also in the treatment guidelines).
With that said it also seems he had an aggressive bone cancer in his childhood, this suggests something unique to this person (e.g. some genetic driver mutation or hereditary cancer syndrome) that may complicate any of this prognostication. Unfortunately something very atypical and aggressive is happening in this person.
H&N SCC in younger patients or HPV+ usually has good outcomes.
Given his history it's probably not relevant to this case but best advice for any HN reader is to go get your HPV vaccine and reduce the risk of SCC.
As an aside Keytruda is closer to 150k/year.
When the odds are against survival, there shouldn't be any rules limiting treatment, if it means even a slightly higher chance of winning. That's something our government has forgotten about or doesn't care about. If I was in this persons shoes, I would make it as personal as possible against those in control of the FDA. Show up at their houses, work, kids soccer practice, get in their face and let them see what their inaction is doing in person.
I agree with you. But the FDA would ask you to rigorously define "slightly higher chance of winning" -- and prove it. In the event you cannot do this, they believe that letting you try an experimental drug would be more unethical than letting you die of neglect.
So I'd rephrase that statement of yours: "When the odds are against survival, there shouldn't be any rules limiting treatment, regardless of type of treatment or odds of efficacy." ("When the odds are against survival" can be quantified, e.g. a 90% chance of dying within a year.)
Would this not also open the door for any snakeoil salesman to prey on what might be medically "hopeless" cases?
If I am ever diagnosed with something currently uncurable, but there are experimental stuff in the works, of course I'd like a shot at those experimental drugs, but I hope that me/my family would also not squander whatever assets we have on snakeoil, in which case I'd rather my surviving family was not impoverished in the fight.
I assume that, at least partially, this is why regulations in this area were put in place to begin with.
If you're diagnosed with something uncurable, then by definition you have a doctor or a medical team that has performed the diagnosis and is overseeing your treatment. There is, invariably, some form of treatment; in extremis, even "here, I've booked you into a hospice where they're going to load you up on opiates" is a form of treatment.
Any experimental drugs you're administered would go through your doctor or medical team. If those drugs are transparently snake oil, they should usually be very strongly advised against. If they have a halfway plausible mechanism, doc will probably say, "go ahead, roll the dice."
In any case, the snake oil salesmen aren't preying on sick people alone -- it's the sick person, plus the professionals who are in his corner, plus family and friends, etc. I don't think it would necessarily be trivial to make lots of money peddling something known to have no efficacy.
On a much more general philosophical note, there was much debate in Ancient China between Legalists, who viewed humanity as inherently evil, and Confucianists, who viewed humanity as inherently good. Where you come down in this debate seems to depends on that view. If you believe that men are inherently evil wreckers, you need stringent and indeed draconian regulations to keep them in place. If you believe that men are good, and that lives saved are on balance the greater aim, you should argue against draconian regulations and limitations on treatments.
Category A - approved by the FDA, basically what we have right now
Category B - approved by some other established regulatory body (EU). Comes with all the warnings, doctors can write it off-label, insurance companies may not cover it but doctors can ask for a variance
Category C - approved by some non-OECD body. Insurance companies under no obligation to cover
Category D - experimental, this is stuff maybe still only in animal models, but pharmacies can still order and dispense it
Category E - experimental and basically limited run from pharmaceutical companies. These essentially need to be tailor-made or produced by a GMP kilo lab. There are plenty of drugs in this category - I worked on them - and the intended recipients are entirely animals, QA, and regulatory agencies. But maybe if some crazy S.R. Hadden type (billionaire in Contact) wants to guinea pig themselves, let em.
The latter category also opens the door for custom therapies (gene/mRNA) that you basically can't test the active pharmaceutical ingredient for efficacy on.
We can already prescribe off-label if the FDA has approved it for at least 1 indication, and it mostly gets reimbursed.
> Category C - approved by some non-OECD body. Insurance companies under no obligation to cover
There's very little that's approved by a non-US body and approved by the EU or non-OECD body that warrants clinical use, and if it is it gets reviewed quickly. The US is the largest market for manufacturers so they almost always start here.
> Category D - experimental, this is stuff maybe still only in animal models, but pharmacies can still order and dispense it
Pharmacists and physicians are ethically bound to prescribe to the best of their ability and avoid harm, by prescribing something only validated in animal models it means we are not prescribing/dispensing something validated in humans and therefore not meeting or exceeding the standard of care.
This sounds like a recipe for killing people.
> The latter category also opens the door for custom therapies (gene/mRNA) that you basically can't test the active pharmaceutical ingredient for efficacy on.
Huh? There are many ongoing gene-directed and mRNA studies being tested.
Do you know what else sounds like a recipe for killing people? Not allowing people to access therapeutics that might save their life because it hasn't yet gone through regulatory approval yet for whatever reason (delays, too expensive to submit), etc.
> There's very little that's approved by a non-US body and approved by the EU or non-OECD body that warrants clinical use, and if it is it gets reviewed quickly
LOL. What are you talking about. There are so many examples.
One of the most tragic is amisulpride. Amisulpride is an antipsychotic medication used to treat schizophrenia and other psychiatric conditions. Some key notes about its regulatory status:
- Amisulpride was first approved in France in the 1980s and is widely used in Europe.
- It was never approved by the FDA for use in the United States, and at this point there's not organization that can afford to go through the approval process because there's no patent.
- The reason often cited is that the manufacturer did not apply for approval with the FDA. It was likely not considered commercially viable for the US market at the time.
- Amisulpride is believed to have comparable efficacy to other second-generation antipsychotics like olanzapine and risperidone, but with a lower side effect burden according to some studies.
- In Europe, amisulpride is considered a first-line treatment option for schizophrenia, but American psychiatrists do not have access to it. According to some sources, it is literally recommended as the best antipsychotic in other countries.
Should we at least demand more specific criteria than "X _might_ save their life", like threshold of suggestive evidence? There will always be lots of stuff that hasn't been closely studied, the effects of which we can only partially describe. You could isolate any new molecule from some previously unknown bacterium and say it "might" be a treatment for any disease, but that's just a statement of our own ignorance right?
If we say, "so long as it hasn't been conclusively shown to _not_ beneficial for the patient's disease, then it _might_ help them, so it should be fair game", then that seems to open the door to quacks selling snake oil to desperate dying people and their families. And of the unenumerable list of potential "it might work because we haven't yet shown that it doesn't" chemicals, why shouldn't unethical practices pick the most expensive options available?
"Of course you must understand there can be no guarantees with any treatment, and this may be a long shot, and precisely because of the lack of prior studies we cannot even give you any efficacy numbers. But we're at the cutting edge of medical science! Please make out a check for $500k and sign this waver and we can begin treatment as soon as possible."
You're assuming a therapeutic not tested in humans has a better chance of saving someone's life than something tested and available. Do you have any evidence to support this?
> One of the most tragic is amisulpride. Amisulpride is an antipsychotic medication used to treat schizophrenia and other psychiatric conditions. Some key notes about its regulatory status:
There is little randomised evidence comparing amisulpride with other second generation antipsychotic drugs. We could only find trials comparing amisulpride with olanzapine, risperidone and ziprasidone. We found amisulpride may be somewhat more effective than ziprasidone, and more tolerable in terms of weight gain and other associated problems than olanzapine and risperidone. These data, however, are based on only ten short to medium term studies and therefore too limited to allow for firm conclusions.
This review compared the effects of amisulpride with those of other so called second generation (atypical) antipsychotic drugs. For half of the possible comparisons not a single relevant study could be identified. Based on very limited data there was no difference in efficacy comparing amisulpride with olanzapine and risperidone, but a certain advantage compared with ziprasidone. Amisulpride was associated with less weight gain than risperidone and olanzapine.
What's so tragic about this? Equally efficacious antipsychotics are available. Once again the alternative to non-approved drug isn't nothing or inferior substance.
I will concede that legacy off-patent drugs are part of the "very limited" gap with the FDA, this doesn't hold for new drug discoveries as discussed in the article.
Even then, there is an ongoing US trial for amisulpride and the substance is approved for nausea/vomiting (granted not in oral form).
[0] https://www.cochranelibrary.com/cdsr/doi/10.1002/14651858.CD...
Edit: Also, the federal law applies regardless of location: https://righttotry.org/rtt-faq/
"I do not live in a state with a Right to Try law. Can I still use Right to Try?
"Yes. S.204 makes Right to Try the law of the land. So long as a patient and treatment meet the qualifications of the federal law, Right to Try applies, regardless of whether the patient’s state adopted Right to Try."
Very slow going, as is all speculative biotech stuff but it made it to approvals on the vetinary side (dogs and horses) and is sold in the US for those purposes. However, I believe the human trials are somewhere around P2 currently for H&N and other indications are even further back.
Only mentioning it because I still follow the annual reports, and I know that despite having no approvals they've treated a few patients via the various avenues that are available in Australia to people who have exhausted all other options.
The drug is tigilanol tiglate. It's had some success to date, with some immune responses in distal tumors after the initial application.
There is plenty of published research available, so please don't anyone take this comment as any kind of recommendation or advice.
This, in itself, isn't a huge obstacle. The problem is the state of healthcare data systems. It's next to impossible to perform high-quality search (even by individuals approved to do so by the IRB). The state of the art in most places is regex searching in SQL.
This is something we have the power to contribute to. Bringing modern search capabilities to important datasets like health (while maintaining HIPAA-conpliance) is a much better use of engineering time than mining spyware data for creepy insights...
[Disclosure: I contributed heavily to one of the major medical search products on the market. We dealt with organisations that expended tens of thousands of dollars and many months per candidate for recruitment. Using some very straightforward IR tech we literally found all their candidates in a few minutes, plus many more. But there is so much more to do!]
Thank you for giving us all some of your time on this earth.
This case reminds me a lot of the FAA realizing that safety restrictions for toddlers would increase deaths as more people would drive. (https://www.ntsb.gov/news/events/Documents/child_safety-Clau..., https://www.ucsf.edu/news/2003/10/97119/airline-infant-safet...)
As the author says, no one ever blames the FDA for the people it failed to save. But approve something without the certainty that it isn't potentially going to kill someone, and there will be hell to your doorstep.
Even if you want the experimental treatment, you cannot get it. Killing someone in an attempt to help is deeply unethical, while merely letting them die is not your fault. This is sometimes also known as the Copenhagen interpretation of Ethics.
In all three instances, the result was that the treatments were snake oil and people died that might have lived otherwise.
It's easy to say "people who have no other options should get access to experimental treatments" but the problem is that people who have other options want those too, because nobody wants to go through chemotherapy or whatever.
Medical authorities have a shitty job.
And these companies get very good at gaming the system, because that is their product. They are mechanisms to commit fraud, stealing all their patients' money and all their families' hope. This is a very bad thing. And, no, I am not making any of this up, but I dare not name names here. These companies also tend to be litigious!
The impact of this is extremely severe. First, people die because they choose a truly worthless treatment that can never work, over bad ones that at least had a snowball's chance. Second, people lose faith in The System, because they think that the fraudulent treatments should have worked, so they lose faith in the good ones too. And third, R&D efforts can get redirected away: rare condition X already has a treatment, so let's put our efforts elsewhere (oh wait, no it doesn't, that was fraud, now there will never be a treatment).
We will not untangle the experimental treatments issue until we confront this problem. Just do not think that it is all in "good faith" here, or even "best effort": there is real fraud going on, committed by monsters that know exactly what they are doing to take their profit.
Then the snake oil manufacturer can peddle their unapproved treatment to as many people as they like, but they are losing money on every dose unless the treatment turns out to be safe and effective.
Obviously you need a big team of scientists on the approval panel to make sure no snake oil salesmen are faking trial data to get their payday, but that seems solvable.
Like Steve Jobs.
Fruit juice kills people.
Then how do you know it's prevalent?
I reserve for myself the right to try to save my own life, even if that means some people who can't handle that responsibility make things worse for themselves. I can't get from, "but other people might hurt themselves" to "therefore you simply have to die, sorry."
No, I think we just have to be straightforward about this; I don't want anybody to purchase snake oil, but that possibility does not move me to eliminate options for myself or my family.
Well, I of course don't know what you personally would do. But some people would do this, and it turns out, actually a lot of the people in that situation would do that. And so something gets done.
This is the same thing as crypto, people "reserving the right to take risks with their own money", and after the money is lost asking why the government didn't protect them. And the same as many other things. Humans suck at accepting the consequences of their own choices.
Nobody has a fetish of taking your choice away from you. People ask for things to be this way, because people are just human.
I think the ideal of regulation is to try to remove the guesswork & expertise required for the end user to do due diligence. It's not perfect, but at least some minimum level of regulation, such as mandatory efficacy disclosures, would be needed.
One of the reasons my father died of cancer quickly is that he was convinced by a chiropractor/osteopath that he had trouble with his back which could be cured with manipulation.
He was not an idiot, but sick people will make bad choices.
The abstract world of "either I die or I try this experimental treatment" is far more blurry, and there lies the problem, in my very humble opinion.
Also we should stop patronizing everyone. If someone understands the risk and their doctor agrees, just treat them.
It's easy to understand the logic of desperate patients who will try anything but it doesn't seem right to allow manufacturers to profit hand over fist selling treatments that may not even do anything, or worse, outright cause harm.
It seems like you're claiming that people who were given medical treatment after media attention weren't down to their last option(s)?
If they were down to their last options, then they made their choice and it didn't work out.
But if the docs say "With our current medical knowledge, this person is destined to die within $TIMEFRAME.", then, if the patient consents to try anything, even if it kills them earlier, why not?
In this person's case, he will not live otherwise, so he has nothing to lose by trying an experimental therapy, although it appears to be too late anyway. Very sad.
So, why can't one go through its own choice of treatment? Disregarding if its a good choice or a bad one.
In my opinion, the FDA ought to be more open to allowing experiments in cases where it is clear the patient is aware of the untested nature of the treatment.
It would result in tragic cases like the three you describe. During COVID-19 it would have resulted in people taking Ivermectin which was ineffective and even harmful.
But during COVID-19 it would also have resulted in determining 6 or more months earlier that the vaccines we made were safe and effective and the number of lives saved in that single instance would have greatly outnumbered the losses from 100 years of experiments.
> The Copenhagen Interpretation of quantum mechanics says that you can have a particle spinning clockwise and counterclockwise at the same time – until you look at it, at which point it definitely becomes one or the other. The theory claims that observing reality fundamentally changes it.
> The Copenhagen Interpretation of Ethics says that when you observe or interact with a problem in any way, you can be blamed for it. At the very least, you are to blame for not doing more. Even if you don’t make the problem worse, even if you make it slightly better, the ethical burden of the problem falls on you as soon as you observe it. In particular, if you interact with a problem and benefit from it, you are a complete monster. I don’t subscribe to this school of thought, but it seems pretty popular.
The post provides real life examples of government programs, nonprofits, and individuals being criticized for helping in the “wrong” way.
1. https://web.archive.org/web/20210125231725/https://blog.jaib...
How often do these drugs, etc turn out to effective? 25 years ago we wanted to rush gene therapy and that didn’t turn out well. Someone died. It probably set back gene therapy:
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC81135/
I think the solution is to invest more into medical research now, then when we, or someone we know, eventually ends up with some horrible disease, (cancer(s), Alzheimer’s, Parkinson’s, etc) we’ll know that we at least we made significant effort, and gave everyone a better chance, rather than settle for a “Hail Mary” drug near the end.
The author is arguing that terminally ill people (with a few months left to live) -- like him -- would be willing participants in trials to help medical research. They already know that mRNA drug or whatever likely won't cure them. It's possible some new scientific knowledge of the drugs' effects can still be gained even when it doesn't cure them.
Given the realistic odds, the primary purpose (from society's perspective) of letting of terminal patients participating in unproven drug trials would be medical research rather than "Hail Mary cures". From the patient's perspective, of course they hope it improves their health.
I think this is a sensible take for someone who is not facing their own imminent mortality. But no matter what we do, the game theory of terminal diagnoses means there will always be incentive to go for a Hail Mary in the end.
This is a big point a lot of people ignore. I feel terrible for the author, but if there hasn't been a single drug invented so far that has any real chance of curing him, it's extremely unlikely that this one out of reach drug is going to be the drug that can save his life. I don't really know much about medicine, but I'm pretty sure that progress is usually incremental as well, so you start out with drugs that cure some patients and work from there instead of having some massive breakthrough.
Who does the investing? Not "The Market". The incentive isn't to cure but to treat. The Market wants (repeat) customers.
The government? At least, in the US, we've been dismantling the government's ability to do this since at least Reagan. Even our so-called Liberals aren't pushing hard here. You'd have to look to the "fringe" Progressives within the Democratic Party to even come close.
And why? Because "fuck you, I've got mine" may as well be the national motto of the USA at this point.
People will bristle at this, certainly. Look at Biden: he's done so much!
Biden is no FDR. We need a real social safety net in this country. And, yes, we need a "right to try", even if it means waiving malpractice rights in those cases.
I think that's often lost on people, and in a more frightening way, people who are all-to-eager to use the terminally ill as ultimately disposable test subjects for unproven treatments. If it was JUST down to safety then I could see the point to an extent, but as you said efficacy is a big open question too. Of course if you're facing death then you may well want to roll the dice, but the question there too is how well you really understand the odds. The understandable implication in a lot of the discussions around this issue is that if only regulators were a bit faster, people like the author wouldn't die.
The reality is grim though, most of these experimental drugs and therapies are not effective, regardless of whether or not they're safe.
I don't understand why we can't let people make risk reward judgments. Yes probably more people will die, but they were not forced.
Says who? In terminal truly no-option diseases (e.g. glioblastoma multiforme) it’s pretty easy to get access to experimental therapies on compassionate grounds.
The challenge in this particular case brings us to:
> Killing someone in an attempt to help is deeply unethical, while merely letting them die is not your fault.
This is a false dichotomy. What’s unethical is offering experimental therapy with ?? effect when there are evidence based palliative treatments (e.g. conventional chemotherapy) with proven overall survival benefit.
In cases where an experimental therapy has at least some limited evidence (i.e. suggestion it might work) it’s quickly approved and pushed into clinical pipelines, osimertinib/Tagrisso for lung cancer is a recent example of such a treatment.
No one is advocating or suggesting we just let people die.
I think that's the main point of disagreement. There are some encouraging cases where the process works well, and it's fair to point to osimertinib as an example.
But there is a good case to be made (although various blog posts likely state it better than I could in a comment) that the current process introduces enough delay that the opportunity cost is higher than the risk.
Most drugs fail. But getting the drugs that do work to patients even a year earlier may be a much larger benefit than the cost of giving people with serious diseases broader choices, in addition to the current best known standard.
No one is advocating we just let people die, but there's an argument about the cost of delaying therapies and the cost of giving people choices that warrants careful consideration. While proven therapies are good and snake-oil is bad, improving standard of care for everyone historically saves many more lives than sticking with what we know for a longer period of time.
This is contradictory. Most drugs fail, how do we identify the efficacious ones other than through trials?
Depending on the anticipated efficacy from early trials as compared to existing ones it will get fast-tracked before the phase II is completed.
Where is the evidence that we’re delaying efficacious therapies where there is no good alternative?
I sound like a broken record right now but this blog post is suggesting we jump to experimental therapies (which would be off label for the author as he does not meet inclusion criteria) before we even have phase I data let alone efficacy, when good validated treatment options exists.
It's as if we can't assess tradeoffs anymore.
You see it with the lack of children playing outside - even though random kidnappings are almost a nil risk and that they're more likely to get by a car.
Defective toys. Defective carseats. Millions get recalled for a failed few.
You saw it with masks. You see it with speech, "words are violence".
Everyone wanting the illusion of feeling completely safe.
How would you feel if your child was injured by a car seat that had a defect the manufacturer completely knew about but refused to recall for the sake of saving money?
"Words are violence"? If you were dox'd, would that be violence? Or your life threatened? Or those you love? Or just people who look or love like you? Where is the line? Wherever you personally believe it should be? No.
Experimental treatments often are very expensive, I believe require a lot of study of the patient and so on ...
It's not like a magical pill(s) you take an go home and live or don't and everyone says "well he consented so he took his chances". The entire process of experimental treatments is hugely expensive.
If a train is coming with someone pinned to the tracks, and they are begging you to hand them an axe so they can remove their own leg-- knowing they may die from blood loss, do you just do nothing?
The problem is that in the real world, it's extremely rarely the case that you can make an intervention that only has benefits, and doesn't have costs, so honestly applying "do no harm" is paralyzing. The medical ethics field understands this, but often the administrators/regulators are way too conservative.
A better model that generalizes without special-casing is "weigh costs and benefits, and be conservative but bounded about unknown risks before making an intervention". In this case the potential downsides are 1) the patient dies which is irrelevant because they are almost certain to die anyway (and they made the informed decision to take this risk), and 2) some reputational damage to the FDA and medical practice if it's seen to be "preying on patients with no other options".
I think 2) is being weighted way higher than it should.
The problem with this model is it requires an understanding of statistics to determine what is ethical and just, and many doctors don't even know how to properly reason about false positives/negatives, let alone the lay public. Yet members of the public aren't willing to forgo their outrage on a subject that they cannot actually conceptualize.
In some sense we are getting the regulators we deserve, not the ones we need; the FDA is probably rationally and correctly evaluating the political risks, media attention, and public backlash around allowing this sort of case.
fortunately, Baja California seems to have everything. Or specifically, a different drug approval strategy that is right in the middle of this.
The FDA's main limitation is that it gives a one to one relationship in approvals. A substance is seemingly evaluated for a single narrow ailment. While evaluation process is a threshold of side effects as well as efficacy in treating that single narrow ailment. This process could be expanded to recognize that fulfilling the side effect issue could expand its use in other ways, more easily than whatever semantical distinction of easier the FDA uses now. There could be more possibilities for one to many approvals.
you’re right though, I think this is still limiting as it gets to currently obscure things, liability concerns for the physician if something goes wrong, or what insurance will cover. so its still hit or miss for the patient
Are the current regulation regimes perfect? No. Would we be better off without it? I doubt it. Should we try to strike a balance between regulation and innovation? Yes—but doing so is devilishly tricky. No matter what you do, someone somewhere will be unhappy.
Most people (myself included) would not agree to that. Experimental treatment should be made possible - at least if there have been promising results with previous studies (animals, small trials etc.). There must be a safeguard, however, to make it impossible to sell false hope to dying people - because they will pay for everything, even if it does more harm than good.
It actually seems like the kind of problem that could be a good fit for tech: have a registry of clinical trials, make sure it’s widely known and consistently used. I don’t know much about the problem, though, so I don’t know if or whether that’s unrealistic.
Interesting. Thanks for sharing the "Copenhagen interpretation of Ethics". I've thought many times about this problem, didn't know it had a name.
Comes to mind Dante Alighieri: "The hottest places in Hell are reserved for those who, in a period of moral crisis, maintain their neutrality."
My understanding of the current categories of drugs are you have "banned" and "has this specific therapeutic effect that costs billions of dollars to prove".
A category of "this doesn't kill you" in the middle would probably alleviate most of this. Insurance only has to pay for things with proven therapeutic effects, but you can shield people from poison and get a lot more data way cheaper by having his middle category.
Put simply, If you believe “my body, my choice” then the current regulatory system is pretty messed up.
To be clear, I’m fine with the FDA existing as a labeling body. Sure label something FDA approved. But don’t limit what we can take because it’s not approved.
Otherwise, you end up with cases like this and many others, where an industry can easily be regulatory captured and ensure competitors are slow or unable to enter the market.
It is (a very rational) approach for administrative workers, who get little credit for getting things better, but are at risk when they can be taken accountable for something terrible.
That way, for example, in Germany, there was a halt on the Astra-Zeneca vaccine after a few cases of thrombosis per million. For comparison, it is one in six for COVID. The halt, most likely, caused many deaths. Yet, these deaths "have happened". If there were a death because an administrative organ didn't take action - the organ would be considered responsible.
For the matter, Pontius Pilate was an administrative worker. And his incentives were aligned accordingly.
In the language of the Trolley Problem, it is doing nothing (no matter how many are there on the tracks) or (even better) finding a way to pass the lever to someone else.
https://www.amazon.com/Regulation-Pharmaceutical-Innovation-...
If you have a system without problems, I’d be more worried.
Edge cases like OP’s can’t be solved by any system. All systems have problems with things falling through the cracks.
That’s why lobbying exists. So people like OP can bring attention to a specific problem and lobby for it.
Taking about changing the system is missing the point.
There are companies from which you can order bespoke mRNAs. Lead times vary but are in the weeks. Many are abroad. The other components of mRNA vaccines can be assembled by someone with 2 years of biochemical Ph.D. experience. There are no laws against a person assembling and injecting themselves with anything.
I have extra sympathy for the writer of this article, since mRNA vaccines (even if they were a random sequence) are relatively lower risk than many other sorts cancer interventions which are FDA approved. I happen to be an ex-professor, who is a director at a drug company. I know that if I were the unfortunate author of this article, whose heart is in the right place. I would probably be injecting myself with my own janky version of whatever offered hope.
Whether that would actually change my prognosis? Probably not. I can say though that I _would_ and I believe others are entitled to positive freedoms I'd afford myself.
And as bad, sad and frustrating as it sounds to someone in a desperate situation, it is for the greater good of many more human beings, protecting us from snake oil.
*this person takes the drug. Two days later they die of a heart attack. Was it caused by the drug? How many resources should be spent to determine that? Should that death be listed in the potential side effects? What if the news story publishes an article and scares people off from the trial? What if it's later proven that the drug had no effect on the heart attack but the distraction from it delayed 10,000 people's access to the drug?
The world is complicated.
This is untrue, in this case.
Typically, only some phase 2 and phase 3 trials are randomised; the treatments that the author refers to are at an earlier stage of development and so not in either of these phases. All patients will receive the active therapy.
It's also worth noting that for cancer trials, while new treatments in these later phases may be tested against a placebo, it's never just a placebo - that would be unethical. If the new treatment is an add-on to an existing standard-of-care treatment, then they'll receive the standard-of-care treatment in the placebo group. If it's not an add-on, then it will be tested directly against the standard-of-care. If there isn't an established SoC, it can sometimes be approved without a randomised trial, if the results are good enough.
And in this case he would be actively helping humanity as well by being a test case. We would all gain knowledges and information from his treatment, if he could get it.
This policy hurts all of us.
Pair this paradigm with extensive postmarketing surveillance and periodic reviews for efficacy in a patient population. These could be done at two years, five years, and eight years -- and approval automatically rescinded if safety issues arise or efficacy is close to null.
Give people with fatal diseases a "right to try" drugs that haven't passed safety testing -- and use that data.
Ban drug advertising in public-facing media.
Simple as.
> it wasn't only because of low-hanging fruit.
Yeah, right. They had such advanced cancer medication back then.
> Give people with fatal diseases a "right to try" drugs that haven't passed safety testing -- and use that data.
That data is practically worthless.
I'm in favor of having people try out medication, but it will need to be heavily regulated. And we know what happens to regulation: a party comes along that doesn't like regulation, or gets bribed, and the regulation goes out the window. It's a good way to start another opium crisis.
This is the reflexive response to every common-sense proposal to roll back the regulatory burden.
I'd raise a couple of points in response:
First, Thalidomide was never approved in the USA -- for safety reasons. The mechanisms that were in place at the time did their job.
Second, the response to Thalidomide was overblown and indeed downright hysterical. "Better to let 100,000 people die of neglect than allow one person to suffer an awful drug reaction" is not rational policy.
> That data is practically worthless.
Enough "practically" worthless data, and you get somewhere. It's a matter of quantity. Obtaining "hiqh quality" data is often unethical in medical practice.
A large data set of highly biased, uncontrolled data is still useless. You can't model yourself out of the factors that have not been recorded. And believe me, the data you'll get on patients that try experimental medication will be very, very incomplete.
It also won't be a large dataset. How many people have squamous cell carcinoma between now and the moment of approval and are willing and wealthy enough to buy this particular medicine? 100 seems too much already.
> The mechanisms that were in place at the time did their job.
Barely, according to wikipedia.
> Second, the response to Thalidomide was overblown and indeed downright hysterical. "Better to let 100,000 people die of neglect than allow one person to suffer an awful drug reaction" is not rational policy.
Thalidomide was never going to save 100,000 people. It did cause 2500 birth defects in West Germany alone, though, plus an unknown number of abortions. Given that it was legal in 46 countries, the number of people with birth defects must be well over 10,000. This drug isn't going to save 100,000 people either, certainly not in the period until admission.
> Say you're a runner. In the beginning of your competitive career, you get the most gains by becoming a better runner. And then at some point, you get more gains by shooting (or bribing) the ref.
agreed on advertising though.
https://www.fda.gov/patients/learn-about-expanded-access-and...
But other than that you can get almost anything even buying it online and get it at home in the next 3 hours.
That's how I buy duloxetine every month.
For those that don’t know, the 1962 change is the Kefauver–Harris Amendment, which was a response to one of, if not the, largest overall global crisis of conscience in the medical field. From Wikipedia: “When first released, thalidomide was promoted for anxiety, trouble sleeping, "tension", and morning sickness.” After some time it became increasing obvious that while it worked, it caused birth defects in pregnant or soon to be pregnant women, with its manufacturers actively trying to quash the information about such cases; again, from Wikipedia: “Use of thalidomide in pregnancy can cause fetal abnormalities such as phocomelia (malformation of the limbs). In males who are taking the medication, contraception is essential if a partner could become pregnant.”
Thalidomide disfigurements were disturbing. Children born with these disfigurements were not linked to Thalidomide until much later. Thalidomide was in fact not allowed a safety regulation by the FDA initially. It wasn’t until the company producing it, Grünenthal, pressured the FDA. You see, the FDA reviewer for the medication was leery about it after some reports had floated over her desk about a possible remote risk that this could potentially cause birth defects. Grünenthal’s international licensee to the US refused to explain multiple papers that linked Thalidomide usage in pregnant women to birth defects.
As it turns out, not only had Grünenthal known about the issue they had actively worked to silence the information, a ploy that worked until a major change in leadership in Germany.
It was denied usage in East Germany.
For a longer documentary on the subject and how it changed medical safety testing, Plainly Difficult has a very good video on it: https://youtu.be/Vi03zz6eCik
(Note: I am explicitly ignoring the context that the Thalidomide development program was run by an actual Nazi who was responsible for unethical and frankly awful experience on unwilling human subjects in concentration camps. If anything, it only amplifies his concern to hide adverse effects, which he did quite successfully until the company was given what can only be considered “A slap in the face”.)
I've had this misdiagnosed medical condition for 25 years by multiple doctors. Nothing serious but extremely annoying. There are approved drugs in Europe for decades which are much better than the ones approved in North America. One day the company making the drug that's approved in North America decided to stop making it! You just could not get it. This was the event that finally led to the correct diagnosis for my condition because I was in so much pain I got to see the expert. Turns out my condition has a very simple non-prescription solution and for 25 years I've been taking the wrong meds that happen to alleviate this different condition as well!
So I am good but this condition is not uncommon. What are all the other people that really need this drug in North America doing?
I'm not sure what's the takeaway here. Maybe that efficient/smart organizations generally don't exist. This is just a reflection of human nature.
A government issued id, like a driver's license, is often required for various services like opening bank account, a telephone connection, avail some government service etc. Thus a local driver's license can be used for deception by a foreigner who wants to pretend to be a local citizen.
> USA refuses to accept rubber stamp European experimental medication. Europe pikachu face
> If anything goes wrong,” he argued, “think how bad it will look that we approved the drug so quickly“
People who take an approved drug are relying on the result and authority of a scientific process — across all drugs. Some scary results and people may start to fear the process, which may end up killing more people.
That said I’m sympathetic to his plight and think that he sounds like a candidate for the compassionate use program. Any result would not help approval as who in such a program would volunteer to be randomized into a placebo arm? They’d just plead in a prior human administration disclosure section of any NDA.
(Also, pragmatically, immunotherapy grate nets are customized and extremely expensive. Who will underwrite it? Not the drug company who would have nothing to gain, not the insurance company, and I doubt the patient could afford it).
It’s a sad story all around.
Part of what they do is they match patients with clinical trials for whom they would be candidates and then facilitate the process on an on-going basis. Apparently this bypasses a number of bureaucratic hurdles which can be faced by patients; for example a patient receiving care from a particular research institution might not be informed about trials being conducted by other, non-affiliated institutions.
Naturally, there's rather more than this to them, but this part sticks out in my mind.
And finally, take what I'm saying with a bit of a grain of salt because I am remembering from casual dinner-time discussions that I had some time ago with my friend. My memory might be bad, they may have changed what they do since I last talked with my friend about it, etc.
Dad died in 2021 and I find it difficult to think metformin may have helped him live a more comfortable life. I know it's not a cure but the research seems to indicate a significant improvement for anyone with IPF taking metformin along with anti-fibrotic medications which Dad did take. We should have just used Mom's metformin really Dad had nothing to lose but it's hard to see that at the time.
I can buy 60 slow release 500mg tablets online for about $10 usd and they'll send them to my home for about 1 extra dollar.
Fuck the government, fuck their controls. If I or someone I love is dying, I'll make sure to try ANY and all options, even if it has a slight chance of working. Everyone has a right to fight for their live.
[1]https://www.fahorro.com/metformina-500-mg-liberacion-prolong...
Given metformins safety and cost profile, your dads doctor sucked. They should put it in the water of the 60+ crowd.
After COVID (both the disease and the vaxx), I got heart arrhythmia, I had around 5000 irregular heart beats a day (PVCs and PACs mainly), and those all happen in a matter of hours, so when they happen, it's pretty debilitating.
After a while, I invested in a Bluetooth stethoscope and ECG watches, the doctors finally believed I have something. However, the cardiologist's advice was to learn to live with it, because it's completely harmless and in young people, ablation, beta blockers are not worth the risk.
Having these irregular heart beats is a very uncomfortable feeling, so I was looking for articles about the root causes and potential treatments.
After trying a couple of things (both pills and lifestyle changes), I found an article that says arginine and taurine reduced the number of irregular heart beats in their experiment. I tried it out and I have basically 20-50 irregular heart beats a day. When I stop taking them, the irregular heart beats come back.
Now, I know I'm doing something that is potentially risky, but to me it is worth it (and I reduced my dosage, and I try to quit using them every couple of months).
* it's not really quack science as the science is real, just that me applying it in my situation was not recommended by a doctor
In moments like this, I think it is appropriate to find a second opinion, sometimes just to attempt something new.
I just really wish that there's something beyond death, that there's something out there, and that all the suffering and unfairness and randomness in life is not for nothing. I hope we someday truly figure out what the fuck we are doing here in this universe. And if not that, at least we reach a point in biology where we have the ability to solve a lot of problems of the mind and body and make life better for everyone.
I’m surprisingly okay with my lack of experience of the billions of years before the earth existed, and I won’t experience the billions after.
That said, just because we can’t stay at Disneyland forever, we might as well go on the rides while we are here.
We are all dying. Some sooner than others. As a student of philosophy, this perspective has driven me to avoid the common status concerns that many people chase.
I DO NOT want the cessation of experience.
Ideally, I'd like to continue in perpetuity, to see the sun rise from Venus, or visit my tenth generation offspring on Saturn.
All of those missed experiences would be deeply saddening. To see our species conquer to universe, to see intelligent life bloom in the darkness of space, that is the goal.
Actual immortality achieved would just push concerns to the heat death of the universe, and folks acting as Cyber-Punk Holden Karnofsky would be spilling ink about the need to invest in star-moving technology to create a big crunch. I don't mean to be trite here. The point is 10 years, 100, years, 1000 years, or 100 billion years, the dilemma is the same, because the linearity of time means any non-infinite amount of life is ultimately minuscule.
The psychological dilemma of existentialism is a challenging one, I'll fully admit that. It's one I've accepted. The benefits also exist though. It forces you to live in the moment to some extent, when others are solely focused on the future.
What you want isn't always what you get. There are a lot of people that have all kinds of urges that they know aren't going to happen. That's perfectly normal, but for so called 'rational' people that fraction that has these irrational desires is a bit strange to me. Rationally: everything dies, sooner or later, and so will you. So the way to get the most out of it is to make sure that you make every minute count.
And even to a lesser extent -- why do we suffer -- is it a law of physics that can't be bent? I don't agree with people who believe more pleasure equals more pain. There will be people who live amazing lives, and those who don't. And so I feel that we as intelligent beings can reduce randomness, and take control of our environment and make sure we can all go on Disneyland rides at least to some extent, and hopefully eventually figure out what's outside of Disneyland.
We suffer almost certainly because it's an evolutionary adaptive feature for us we picked up somewhere along the way. It is no surprise that the general suffering we find in developed countries, namely loneliness, is directly related to human reproduction.
Is it a generally happy or sad state of affairs? Who's to say. It's our state of affairs, and we best do what we can to make the most of it.
From this I derive the motivation to work hard at what I do and ultimately try to contribute to the problems we face as a species before I pass. It also makes me appreciate the raw human connection that we can all experience: love, passion, friendship.
I may not be the one to light the altar of discovery that allows us to say, cure cancer or become a spacefaring species, but I will proudly carry the torch and pass it on.
Matthew 7:17 - "Ask and it will be given to you; seek and you will find; knock and the door will be opened to you."
I was given a very clear sign 17 years ago, not when I asked God to prove himself, but only incidentally when I was seeking his guidance on something; it's nevertheless lasted as an undeniable proof for me. Though, it's better to believe without seeing.
John 20:29 - "...blessed are those who have not seen and yet have believed."
Acts 2:38 - "Repent and be baptized, every one of you, in the name of Jesus Christ for the forgiveness of your sins."
Disclaimer: I do not identify with American/political 'Christianity'.
Or are you saying that billions of Buddhists and Hinduists are delusional?
There is no cheaper way to turn swine into gentlemen.
Now we just need to trick them into eating the stuff.
They are a doorway to new perspectives. You still gotta internalize the message.
The drug supposedly helped delay the doctor's cancer and extend his lifespan until he stopped taking it, then it came back "with a vengeance" and he passed away. He wanted to stop taking it to verify whether it was actually working or not.
I wonder if it might help this person? He might have to be on it for life though.
Don't take my word for it though, this is FAR from my field of expertise.
It isn't a convincing argument to me, because possible death avoidance is a higher level of morality to me, but harm reduction is extremely prevalent in medicine as a driving factor.
Strange isn't it? Almost sounds like soylent green. Maybe I'll skip McDonald's this week.
It's a tough line to walk on, still.
I am getting so mentally exhausted learning about a specific problem, and then experiencing the helplessness of not knowing how to push on driving towards some mutually agreeable resolution.
Add in the fact that there is no way to seemingly coordinate the push towards a resolution. Individuals taking action without coordination feels just the same as taking no action at all. Are there any tools to coordinate the push and keep track of progress?
You have the whole usual bunch of idiots that will advertise magnetism and cosmic rays to cure you, then new treatments that may or may not be available in your country (but available in others) and finally the trial ones.
I discussed at length with an acquittance of mine who works in the clinic trial branch of a pharma and she first gave me the usual double blind trials explanation (which I am aware of), but then could not really answer why the pharma companies insist in not having people, for free, that would test if the treatment is not obviously lethal (or heavily impacting).
If I was faced with the perspective of dying soon (or going through something like Alzheimer) I could not care less if the treatment is fatal to me. Again: not that is not working (this is something one can assess statistically through normal trials) but that it is clearly harmful.
This would at least be a clear indication of "don't try it" (or "be very careful monitoring for this and that")) if there are enough patients who react badly
Incentives matter. When you get to make decisions that impact others, but not feel any of the costs associated with that, you do not have the correct incentives. I hope the staff of FDA read this and can’t sleep tonight. We can hope they feel some emotional pain, even if it is only some small subset of the pain they have and continue to cause to others.
FDA delenda est.
Those who establish, support and tolerate that system should be as directly exposed to its consequences as practicable. It is good for them to see these stories and feel the consequences of their decisions.
The consequences to their actions are that millions of people are saved from the consequences of consuming seemingly promising drugs such as Thalidomide: https://en.wikipedia.org/wiki/Thalidomide
I could sleep just fine knowing that some people looking only at a tiny local example while ignoring the big picture considered me the villain
I think we can all see incidents of where some oversight process is loosened because of some legitimate desire to help some, but then you learn that the bad / incompetent / exploitative actors come out of the woodwork to test and take advantage of the changing of rules. Not even saying intentionally, but just by sheer numbers, the change you made allowed things that were being held back by (hopefully proper) regulation to now happen.
Anyway, not saying that the FDA and other bodies couldn't move faster -- that is always the case. And there will always be examples of people/cases stuck in the cracks with legitimately sad situations.
But I would hope that people reading such stories think about why there is a process. It's not like the FDA's purpose in life is to stop people from benefitting from new treatments. It's to prevent the flood of bad effects of companies/individuals from being able to say they offer some drug that doesn't work in the way it says it does.
In general I feel like the sick should not be held responsible for the unintended consequences of things. The principle that it is better for 10 guilty men to go free than 1 innocent man to be punished applies here as well. How many suicides have happened because it took this long for the use of psychedelics and THC for the treatment of depression and PTSD to be approved (partially, in some places). How many people are suffering today, right now in pain or under the care of doctors they would otherwise leave for poor treatment but the "opioid crisis" has limited their access to the treatments they need? I've faced this problem personally more than once, and while I am sympathetic to the law of unintended consequences, I just can't be convinced that it is the duty of myself or my loved ones to suffer because someone else might abuse something. There is a balancing act to be had for sure, but any borderline case should ALWAYS err on the side of reducing the current actual harm in favor of preventing a nebulous potential future harm.
Even if the drug/treatment method were approved, how much would this cost, would the author even be able to pay for / have the insurance company pay for the treatment?
And by the way, what limits are there on the price of a drug that an insurance company or the government will cough up the money to pay, for one person's extension to life for a couple years?
If someone has a very rare or just very advanced cancer, how much should the rest of us (as individuals paying taxes/premiums/etc) be on the hook for paying for last ditch efforts to prolong that person's life?
These are genuine questions I think are legitimate to ask, if not in polite conversation, then at least at the level of policymaking bodies in govt or insurance companies. I'm sure that people in the UK are quite familiar with this concept or debate.
You cannot just say you'll pay whatever it takes to save someone's life no matter what the circumstances.
Wish there was some country or state where you could try more freely.
Allowing trials to be bypassed and allowing people to pay for early access to unproven drugs would bring a flood of such be vendors and be bad for society, bad for medicine, and bad for ethics.
The article even cites a case where the bureaucrats are confident in the safety of a trail drug but unable to approve for political reasons.
Not to mention the FDA allows plenty of snake oil supplements, etc. to be sold, so it's not particularly good at the goal you claim it's optimizing for.
The same is true for a lot of cutting edge treatments.
It quite strictly limits where such treatments are likely to be available.
Dr's typically practice in ~1 state while drugs are available in all states simultaneously.
Fifty states - of varying ability - trying to independently qualify all available drugs would be a nightmare of redundancy.
Florida would probably outlaw all mRNA technology. Their FDA would refuse to approve any mRNA-based therapies, and so on.
We have seen the disaster that approach can cause in healthcare, insurance, abortion (reproductive health broadly), gay rights, etc. It's insane that we have state by state drivers licensing as an example that is merely highly inconvenient.
It's not a good thing that doctors and nurses are regulated at the state level. It's a horrible thing that contributes to the US healthcare system's vast dysfunction.
Medical tourism is an excellent opportunity for "network state" and state alternatives. I already get virtually all elective medical care outside the US for commercial and service quality reasons, despite having US insurance.
- where do you go, and why?
- how do you get your US insurance to cover/work with providers outside the US? does it just automatically work or…?
I do carry Blue Cross/Blue Shield PR coverage ("PPO Gold", $230/mo), although it doesn't cover anything outside of PR except for emergency care. I should probably get a secondary insurance plan (which might include full coverage outside the US, or might even include shorter visits to the US as well), but for now I'm comfortable self-insuring medical costs, particularly since I think I could get insurance negotiated rates in the US even if they're paid out of pocket.
Better quality, lower cost, than anything I've found in the US. I live in Puerto Rico, which has particularly bad medical care; if I lived in Boston or SFBA I'd possibly have a local doctor, but I haven't found anyone in PR, except for expat friends who are neurorads/etc., who is a competent doctor. "Have a pain? Get on a plane" is the plan, and I have medical evacuation insurance, an ALS bag in my house, etc. for that.
The other upside is my records remain under my control; they don't get put into some weird insurer/employer accessible system protected only by laws. I can request/receive raw files and keep them myself.
(So far, I don't really have any serious or chronic conditions besides being overweight and slightly high blood pressure, but if I had a screening discover cancer or something, I'd want to have full flexibility on how to proceed with that.)
(Relatedly, I've deferred getting a dental implant for a failed root canal since right before Covid, so currently looking for the best dental implant medical tourism option -- Mexico, Colombia, and Asia are all pretty solid. It's 3 visits (plus possibly orthodontics since it's been so long with a missing molar), but internationally is maybe $2-3k vs $5-15k.)
https://www.fda.gov/patients/fast-track-breakthrough-therapy...
There is some important context for the following comment:
“If anything goes wrong,” he argued, “think how bad it will look that we approved the drug so quickly.”
In the 1980s, when the comment was made, there were still people at the FDA who remembered the Thalidomide disaster, which would have been a lot worse had the FDA approved the drug. In the U.S. several thousand women took thalidomide during the clinical trials, and some doctors took it, too:
In one case, a doctor had been using thalidomide himself and prescribing it to his wife. In addition to the wife’s loss of vision, the doctor mentioned peripheral neuritis, nerve pain that is a side effect of thalidomide.
The other report is even more alarming — a nurse had given birth to a baby without arms or legs and, as a registered nurse, “she may have had access to the item.” (1)
Other countries including Canada, Taiwan, Japan, and West Germany did approve the drug or allowed it to be sold.
On December 2 1961, the drug was taken of the German and British markets, after several doctors brought up concerns as it appeared more and more plausible that thalidomide, when taken by pregnant women, was responsible for severe birth defects. Thought the Government of Canada was informed of these suspicions about the possible teratogenic effects of thalidomide, we had to wait until March 2 1962 for the Canadian authorities to react and, in their turn, withdraw thalidomide from the market. As unbelievable as it can appear, thalidomide was legally available in Canada for three full months after being withdrawn from its origin country. (2)
1. https://www.nytimes.com/2020/03/23/health/thalidomide-fda-do...
Governments/regulators deciding what you can put into your body is beyond ridiculous: applies to all substances and drugs.
Inform about the potential risks and effects: sure.
Prevent: never.
"Regulation" vs "Information" also gets complicated, fast, when you consider global supply chains - for recentish news, checkout the stuff on heavy metals in Trader Joe's chocolate - how does all that information propagate to the final consumer? How does that consumer have the knowledge, skills, and time to process it?
(As with all things, IMHO, it's about balance, and, IMHO, balance comes from forces in opposition. "You are the ultimate authority on your body" is one force.)
And if you protect the drug companies and doctors against those lawsuits, then you open the door to them exploiting desperate people.
"IL-6 Activities in the Tumour Microenvironment. Part 1"
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6765074/#:~:tex....
"Serum Level of Interleukin-6 in Patients with Oral Tongue Squamous cell Carcinoma"
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4461844/
"Interleukin-6 role in head and neck squamous cell carcinoma progression"
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5698512/
I also know from Google Scholar, that copper increases IL-6, penicillin breaks down into penicillamine, used to treat Wilson Disease, a copper metabolism difference, and I also know that Omega 3's look promising for reducing IL6.
https://pubmed.ncbi.nlm.nih.gov/29803716/
And thats just an example of a cursory look into SCC.
Socialised medical costs, isnt always a panacea, knowledge and honesty is the closest you will get to a panacea for medicine.
People who aren't in medicine really have a hard time understanding just how grisly the things the human body can go through might be.
Medical staff saw during COVID the mismatch between the degree of fear the average Joe had about the consequence of intubation and the degree of fear and panic of the average Joe when actually getting a tube down their throats.
A drug that goes wrong in a bad way can go REALLY wrong in a bad way. Dying from cancer sucks, but it's still several degrees less horrible than dying from your CNS being eaten away from an autoimmune response to an experimental drug which somehow leads to opiate-resistant neuropathic pain.
But if we simply caution "we don't know if this drug will cause horrible side effects" versus "this drug may likely cause unthinkable pain that we can't control in your last conscious moments" that's a very different level of informed consent.
You might be surprised by the number of doctors who privately lament how patients and patient families in general will so often choose to shoot for low odds outcomes that mean terrible conditions for the patient in their last months of life. Even when risks are known, people tend to be bad at actual risk assessment, and will downplay risks and focus on potential rewards, and this extends into medical care. And I'm skeptical that descriptions of what might go wrong versus actually seeing firsthand what it looks like when things go wrong still represents adequately informed.
So while in spirit I agree that completely criminalizing personal choice around consumption is not ideal, when there's insufficient data for truly informed consent I'm not sure I still see eye to eye on the topic.
It should be legal too. I'm not defending the use of it in any way, this is not about drugs but a more fundamental problem: governments deciding what you can and cannot put into your own body.
They should inform people about potential dangers and effects of substances, and try to prevent the underlying reasons for people to, say, do heroin or fentanyl.
But if someone wants to do it anyway, making it illegal just makes things worse as people will be stigmatized socially where they should be welcome/accepted the most, and they'll obtain the substance illegally and dangerously (who-knows-what-it's-laced-with and exact actual dosage) anyway.
So just inform and support the people but make it legal and relatively safer to consume for harm reduction.
But if someone wants to OD and kill themselves, I mean, it's sad but it's their own body and their own life.
Thought experiment: There is a magic drug or parasite where any dosage (whether deliberate, accidental, or fraudulent) will force the person into a single-minded violent quest to get another dose.
For the next month, another hit is the most important thing in their world, even if that means selling everything they own, cutting off their own leg, or murdering their children. Repeated doses cause mental confusion and are eventually fatal.
Are you still comfortable saying that nobody can make any law against the distribution or consumption of THAT substance?
Laws against consumption? A terrible idea.
What we should want to do as a society is funnel people interested in trying things we think they really shouldn't do towards legal chokeponts that are less onerous than DIY access under controlled distribution but still allow for attempts at prevention of the end outcome.
The clearest example of this even more than your drug scenario would be a drug that just immediately kills the user.
There's a lot of people every year that seek out that end result. While some can fall under a narrow scope of legal options under dignity laws when faced with terminal situations, there's many who seek out that outcome without physical ailments.
If there were a legal way to seek it which was overall less traumatic of a route, but which was also only on the other end of intervention measures like counseling, how many lives might be saved as compared to the rather ineffective prohibition that we see today which largely fails to prevent access and use, but whose illegality does prevent aspects of both research and prevention that might otherwise occur if distribution was the only thing targeted in laws and not attempted consumption?
If people are aware of the life ruining consequences of a drug but value their own lives so poorly that it doesn't deter them from throwing them away to seek out a drug, then society doesn't have a drug problem as much as it has a human experience problem.
There's few things more cruel in concept than ensuring people keep living under conditions where they'd rather not live at all. Whether they are seeking that result all at once or gradually throwing their life away, criminalizing their seeking rather than the conditions that motivate their seeking is wildly messed up.
It doesn't matter if insurance companies aren't required to pay for it. People will max out credit cards, borrow from friends, and mortgage their house. All for something that could sit anywhere on the continuum of: does nothing to kills you immediately. Probably with a side of "did this rather than something that would have actually worked".
the economics, ethics, and effectiveness around early approvals are really difficult to manage. companies have a strong incentive to get earlier approvals. but once the cat is out of the bag, it's hard to get it back in. if a sub-standard drug makes it to market and gains wide adoption, it makes later drug trials of more effective drugs really hard. where's the outrage for potential beneficiaries of better therapeutics?
I don't mean to say this man and others shouldn't get a chance at a hail mary when they're staring down death. just, it's risky business for others in the future. I hope the best for him and others.
I see the FDA approved drug cemiplimab-rwlc for squamous cell. https://www.skincancer.org/blog/new-treatment-for-advanced-c...
And if your willing to consider alternative therapies there is fenbendazole(see joe tippens protocol). It’s a dog dewormer and is available at pet stores as panacur-c.
Also see Albendazole, Flubendazole.
Unfortunately it’s our dignity (or at least others diagnosed with „x remaining months“). As soon as this becomes normalized, the moral impetus will change that those wo didn’t make it, probably weren’t brave enough to try the new stuff.
In what ways does always letting individuals choose to take an experimental drug not work, if we make sure they know what is known and what is not? They are desperate, and that's a horrible place from where to make a decision, but dying is a really bad alternative.
What are the pitfalls that would allow for this to not always be the option? How would this go more horribly wrong than just letting these people die?
The other concern is when you turn down medicine that might work for snakeoil that doesn't.
Having said that there are two elements that I’m not quite sure how to adjudicate 1) where does one draw the line with regards to how sick someone needs to be to qualify for right to try, and 2) how do ensure that patients are actually getting a treatment with some resonance likelihood of success as opposed to some snake oil?
The media (both traditional ans social) owns a large chunk of the underlying blame, due to their rush to create simplistic narratives in the wake of anything that "goes wrong" without nuance or examination of tradeoffs.
A person has to qualify for the clinical trial by meeting a set of specific set criteria to ensure the results are interpreted with the least amount of error.
If he said he went to Mayo, MD Anderson, Harvard, Sloan Kettering, and none of the trials were available to him that would make a little more sense. But a great number of the trials in his disease site are for treatment refractory patients, so it’s pretty surprising there’s no apparent trials for him.
That all the MRNA treatments they refer to are in trials tells you everything you need to know: we don't know which work, we don't know how effective they are, and we don't know what they work on.
It is possible to enroll in trials for these treatments, but as they say they "may" be denied as they're so far along. But that's what you want: if a trial takes someone on who would be highly likely to die even if the treatment works then depending on the trial size it's possible that that one patient might skew the results such that the treatment is denied, or alternatively, another person who applied for the trial gets denied even though they had a higher likelihood of survival.
I can understand it being incredibly hard for this guy, but we have been through the alternative:
* Snake oil treatments: drugs that do nothing but bankrupt people
* Actively harmful treatments: drugs that literally make things worse, while bankrupting people
* Paid trial scams: you can pay to be part of a trial, which immediately allows for the above two despite a regulatory environment that ostensibly requires trials.
etc
Things like the FDA exist in response to prior actions, and once they've existed for a while, people forget that the only reason that they don't seem necessary, is because they are there. Much like the "unnecessary" financial regulations that were removed, and immediately resulted in banks creating the Great Recession.
Hence, it is not possible for the FDA to create a "patients can be 'treated' with untested treatments" loophole that is not trivially exploitable by the kinds of people that resulted in the FDA existing in the first place.
This is after all a group full of hackers. I don’t know this domain area, but isn’t there anything we can start or do to change this obviously stupid and immoral system?
1. When the FDA has fast-tracked drugs, drug makers have taken advantage of this to hide data, lie, and refuse to comply with timelines for proving those drugs work. https://www.npr.org/sections/health-shots/2022/07/22/1110830...
2. When these accelerated drugs are found to not work or cause harm, the manufacturers continue to push for them and the FDA has tremendous trouble trying to their approval revoked. https://apnews.com/article/science-health-medication-busines...
3. Accelerated drug approval gets mired in advertising. The general public has no idea how to judge if a drug works or not. Plenty of people believe that homeopathy works. The FDA gets massive pushback when trying to take a drug that is worthless away, because people think it works. This hurts us all and creates an incentive for companies to hide data and deceive customers.
4. The FDA is as fast or faster than the Canadian or EU equivalents. The FDA is not specifically slow, bureaucracy everywhere has become much slower because there are no incentives to be fast but countless incentives to be slow.
5. Lawsuits. It's easy to say "I'm hurting, I'll take anything". But, then, there are rights you cannot give away. If a drug harms you, you will sue. If it kills you, your family will sue. The whole system is bogged down by lawsuits with massive disproportionate payouts. The early biotech companies and scientists involved often cannot deal with even a mistaken lawsuit.
We need a much more comprehensive overhaul of the system, starting with open access laws to all data pertaining to any drugs that are on the market (no manufacturer can keep anything secret for a drug that people take), an FDA that has much more authority and much less industry capture, medical tort reform, to create a system where in exchange it actually makes sense to provide fast tracked approval.
But that's not the main problem here. You would expect the FDA to approve everything by that theory.
A common example why government doesn't solve problems well. Because it's an absolute governing law, there's nowhere else to turn to. This creates an industry of snake oil that preys upon people both as regulated medicine and homeopathic remedies. In practice vertical integration is a requirement for economies of scale. This means all medicines must be created by singular centralized corporations. These corps buy out the regulatory and now we have an incredibly inefficient and locked out market that prevents innovation.
All orgs should be sunset after 20 years by law. Repeal the FDA, replace it.
> This makes it nearly impossible for patients to find an appropriate clinical trial, discouraging all but the most stubborn–people like Mike Hindt, and people like Stephanie Florence. By her own admission, Florence, 44, a photographer living in Lewiston, Idaho, had to “bulldoze” her way into a trial by being persistent to the point of obnoxiousness.
> When she found out she was a candidate for a new trial, she also learned another hard truth: trials don’t come to patients. Patients have to go to the trials.
> In August, she and her husband drove from Raleigh to Boston, despite having no place to stay. After a week in a hotel room paid for by their daughter, they wrote about their circumstances on a community blog. Several people in the area offered the couple rent-free housing during Price’s treatment. In order to pay for food and other expenses, the couple are trying to sell their home, and Price’s husband, laid off from his job at IBM, took a couple of part-time shifts a week at the outdoor-apparel store REI.
[1] https://time.com/4270345/immunotherapy-pembro-clinical-trial...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7309195/
Not sure how this intersects with the author's reality. It is sad to see article after article spewing nothing but hatred towards Trump --almost purely from an ideological perspective-- when something like this could help so many.
This, in more ways than one, demonstrates how difficult of a problem this is. When people's lives become game pieces in political battles, the people lose and politicians, well, use them to score points towards their own career objectives.
I read in a comment that the author lives in NY, where, apparently, Right to Try isn't available. If find this interesting when a federal law has been in place since 2018.
This is one of the things that can be perplexing about the US system of government. Here we have a state preventing people from having access to treatments that could materially affect their illness when federal laws allow it.
While I do understand the many advantages of the independence granted to states by the US constitution, sometimes it feels like the US has devolved into a fifty regions pretending to be united as a nation when they are actually not and, as a result, end-up conspiring to damage the very societies they claim to protect.
Education is another example of this. All nations with excellent systems of education (and the results to prove it) have national-level planning, management and standards. In the US, not only is our system of education fragmented at the State level. Our schools are run by fucking unions organized as districts, each with their own axe to grind. It is no surprise the results are what they are.
We somehow manage to extend some of this "excellence" (sarcasm) into every level of healthcare.
Another area where the US has lagged which would prevent a lot of these cancers from ever developing in the first place is HPV vaccination. Last I heard it was only females who were being routinely vaccinated, and relatively recently at that. We should really be prioritizing eradication of HPV.
[0] https://en.wikipedia.org/wiki/Squamous_cell_carcinoma#By_bod...
https://www.fda.gov/patients/learn-about-drug-and-device-app...
Biden's Moonshot program is trying to drive money to support new innovations:
It's a popular religion among the well-to-do. As you might imagine.
The issue with having a standardized approach to people getting experimental drugs is it opens up its own kind of mini-market unless the implication is they would be required to be free by law. And desperation is not the right mindset to make law or rational decisions.
Not trying to sound heartless, its just not as easy as saying "go faster".