45 ms·

I am dying of squamous cell carcinoma, and potential treatments are out of reach

• orzig 3y ago
  Read the rest of his blog; it’s wonderful and I’m devastated that someone I followed for years is going to vanish. Thank you for giving us all some of your time on this earth.
• chris_va 3y ago
  Anyone more knowledgeable know why they can't file for a single patient IND, assuming the vaccine is even acquirable?
• tux3 3y ago
  There seems to be a growing sentiment that FDA delenda est, but if we don't fix the underlying problem that led to this situation, it will just come back. 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.
  • riffraff 3y ago
    I remember at least three instances in my country where the authorities were forced to allow people to use experimental treatments due to media clamor. 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.
    • nextos 3y ago
      Immunotherapies are still immature but far from snake oil at this stage. They routinely achieve miraculous remissions but also lead to many adverse events. For most late stage tumors, chemotherapy will only, at best, increase life expectancy a bit. In my opinion, the choice is obvious.
      • deleted 3y ago
        [deleted]
      • Retric 3y ago
        Imminotherapies very quickly kill a significant percentage of the people who try them. So it’s a complicated balancing act when someone is likely to die in say 1-2 years but also likely to survive another 6 months. For people who are likely to die very soon the case is more clear cut, but the people developing these treatments don’t have the resources or infrastructure to try and treat more than a tiny handful of people. Allowing people to pay for such treatments also opens the door for a huge range of bad actors which also costs lives. I am not saying the current system is great, just that there’s a lot more complexity than is obvious on the surface.
        • eilertokyo 3y ago
          Your comment makes something that happens quite rarely (1-5% based on agent, and not quickly) seem like it happens quite frequently.
          • Retric 3y ago
            Some of these trials had double digit percentage deaths within 30 days.
            • adamredwoods 3y ago
              Because the trials sometimes take on people with very little time left.
  • melling 3y ago
    This has been a common complaint for decades. We always want to try rush experimental drugs/procedures that “show promise”. 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/ 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.
    • EA-3167 3y ago
      People are, perhaps understandably, very eager to advance at a faster pace, always a faster pace. Nowhere is that more evident than in the case of people facing death regardless, but in addition to the issues you raised (early failures can lead to even worse delays) there are other ethical concerns. Terminally ill people are often understandably desperate, and I would be too in their position, no doubt about it. Their families are desperate, often their doctors are desperate. That doesn't mean that their remaining life doesn't have value though, or that cutting it weeks, months or years short isn't a problem. 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.
      • causality0 3y ago
        We let people have assisted-suicide for reasons from homelessness to hearing loss, twenty thousand people a year. Why do we respect their self-ownership when they're trying to die but not when they're trying not to die? Why do we get to decide what is and isn't worth the risk?
  • lr4444lr 3y ago
    The FDA could create a standard consent form for nonapproved treatment that patients and doctors would sign, which would indemnify the physician from legal repercussions, but they don't, and they bear full blame for that. The AMA would still have its own regulations about the limits of ethical experimentation, with the revocation of a medical license as punishment to serious offenders, and malpractice safeguards could still be there. State laws about gross medical negligence are also an option.
    • adrr 3y ago
      You can get get experimental drugs/treatments that aren’t approved by getting them under the clinical trial for that drug. The question is who pays for it? Cancer treatments are millions of dollars.
      • zaroth 3y ago
        Clinical trials are, I think always, free for the patient. The company developing the drug foots the bill.
        • junofan 3y ago
          That’s just not true.
          • DennisP 3y ago
            Not always, but it's usually true. > Patients generally do not have to pay extra out-of-pocket costs for treatments studied as part of a trial. Every trial is different, but the clinical trial’s sponsor usually pays for all research-related costs and any special testing. > Typically, the patient or his or her insurance company is asked to pay for any routine tests, treatments, or procedures that would be required as part of standard cancer treatment. Before you join a clinical trial, you will receive an informed consent document that spells out exactly what you’ll have to pay for and what you won’t. https://www.mskcc.org/cancer-care/clinical-trials/frequently-asked-questions https://www.mskcc.org/cancer-care/clinical-trials/frequently...
  • chroma 3y ago
    For those who are curious, the term was coined on this blog post[1]: > 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.jaibot.com/the-copenhagen-interpretation-of-ethics/ https://web.archive.org/web/20210125231725/https://blog.jaib...
    • unglaublich 3y ago
      So, you would flip the railroad switch to kill only 1 instead of 5 helpless people?
      • roflc0ptic 3y ago
        the math checks out
      • pjot 3y ago
        MIT recreated this thought experiment to research the “morality” in autonomous vehicles. https://www.moralmachine.net/ https://www.moralmachine.net/
        • bandyaboot 3y ago
          It interprets telling the vehicle to crash into a barrier rather than hit pedestrians as not placing as much value on protecting passengers. But really, this is just a recognition that the passengers are far more protected in a crash than the pedestrian are when getting hit by the car. Instead of a barrier, it should be a large cliff or something.
  • yieldcrv 3y ago
    > Even if you want the experimental treatment, you cannot get it. 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.
    • stbenjam 3y ago
      Off label prescriptions are legal and very common.
      • yieldcrv 3y ago
        huh thats exactly what google says. 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
  • batch12 3y ago
    > Killing someone in an attempt to help is deeply unethical, while merely letting them die is not your fault. 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?
    • y-curious 3y ago
      Moreover, if a terminal cancer patient wants to commit suicide, they can legally do so on several jurisdictions. Why then, can they not instead opt for this treatment as an alternative to suicide?
  • aantix 3y ago
    There's this overabundance of the need for safety that's overcome society. 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.
    • vore 3y ago
      Uhh, given recalling defective toys or car seats is a pretty straightforward way to prevent further injury, isn’t that a pretty good trade off over knowingly potentially causing more injury just to save money? 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?
    • sleight42 3y ago
      You had me to a point. And then you began the usual conservative dog whistles. "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.
  • duxup 3y ago
    One issue is, who pays for it? 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.
  • lettergram 3y ago
    I think what really drives me crazy is that the FDA approval can limit me taking something. 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.
  • agumonkey 3y ago
    It's a high worth debate, maybe there's a ethics curve.. for most cases you have the normal "don't do harm" but for life threatening you need another board to remove some stops without letting the scams proliferate.
    • theptip 3y ago
      "First do no harm" sounds good on paper, but it fails the trolley problem. It leads you to harmful beliefs like "we should not use the AZ Covid vaccine to save thousands of lives because it might cause one person to die of blood clots". 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.
      • agumonkey 3y ago
        Thanks, very interesting.. i dont know if it's worth setting up a group to raise statistical understanding or just wait for society to suffer enough to finally learn.
  • stared 3y ago
    I call it Pontius Pilate's Ethics. 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.
  • Kamq 3y ago
    I still think they need another category than what they have now. 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.
    • abecedarius 3y ago
      The requirement to test for efficacy and not just safety was only added in response to Thalidomide (which disaster had nothing to do with testing for efficacy). So what you're asking for is very not radical.
      • Kamq 3y ago
        > So what you're asking for is very not radical. Shame. Radical seems to be all the rage these days. Maybe I can brand it as radically conventional.
  • haldujai 3y ago
    > Even if you want the experimental treatment, you cannot get it. 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.
    • tux3 3y ago
      >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. 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.
      • haldujai 3y ago
        > 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. 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.
  • grammers 3y ago
    > 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. 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.
  • thrashh 3y ago
    Is there really a growing sentiment? 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.
  • WalterBright 3y ago
    Sam Peltzman goes into this and the statistics in his book Regulation of Pharmaceutical Innovation: https://www.amazon.com/Regulation-Pharmaceutical-Innovation-Amendments-Evaluative/dp/0844731285/ https://www.amazon.com/Regulation-Pharmaceutical-Innovation-...
  • npsomaratna 3y ago
    Going on a tangent here: there's a reason medicine is heavily regulated—because of the lessons we've learned the hard way. The same reason law is also heavily regulated. Some of these lessons have become obscured by time, unfortunately. Other lessons may not be fully applicable to the modern state of the field. (I say this as a qualified doctor and lawyer with some appreciation of the history of both fields.) 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.
  • deleted 3y ago
    [deleted]
  • atmosx 3y ago
    > 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. 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."
  • alpineidyll3 3y ago
    Imo the whole US medical complex needs competition from a small but sophisticated libertarian state without any of the regulations that formed the present system, and I think it will happen. The market demands it, and the world would be better off with some competition. Most likely it will begin with some small nation that sells anti-aging therapies. 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.
  • msteffen 3y ago
    I think it could be ethical to let people try experimental treatments in the context of a phase 1 clinical trial, ensuring that something is learned from the attempt. However, I have also heard that an issue with that constraint is that connecting patients to clinical trials is a huge logistical problem that has not been well-solved. 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.
• changoplatanero 3y ago
  I get that the FDA is bad but how come there is no country anywhere on earth where these treatments are legal and available?
  • etothepii 3y ago
    This is a very interesting question. Why are medicines regulated federally? As I understand it (and IANAL) doctors, pharmacists and nurses are regulated on a state by state basis.
    • adventured 3y ago
      It would be an extraordinary disaster to kick that regulation back to the states. 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.
    • WarOnPrivacy 3y ago
      > Why are medicines regulated federally? As I understand it (and IANAL) doctors, pharmacists and nurses are regulated on a state by state basis. 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.
  • adventured 3y ago
    Because the mRNA vaccine approach is just about as cutting edge as it gets right now and only a few countries on the planet are any good at it. The same is true for a lot of cutting edge treatments. It quite strictly limits where such treatments are likely to be available.
  • Arainach 3y ago
• Sinidir 3y ago
  Must feel especially shitty to have your freedom curtailed like this, looking at the probable end of your life.
• tb_technical 3y ago
  What I don't understand is why the FDA doesn't automatically rubber stamp approvals on medicine approved in Canada and the European Union. We all know their medicine is good - so why can't I get it?!
  • m3kw9 3y ago
    So all drugs will always be trialed at the most lax country and get approved at a stricter one? Then why make rules because the laxest country will be the one used and in control of approvals
  • deleted 3y ago
    [deleted]
  • LordShredda 3y ago
    Because they might make mistakes? See: thalidomide
  • tekla 3y ago
    > USA rubber stamps probably safe food items. Europe goes WTF how dare they think its safe. > USA refuses to accept rubber stamp European experimental medication. Europe pikachu face
  • Latty 3y ago
    Somewhat famously the US not doing this stopped Thalidomide being such a large issue in the US, but of course one example of it failing doesn't mean it is inherently bad policy.
    • casefields 3y ago
      It’s strange how people trot that out and never discuss the inverse.
  • maxbond 3y ago
    Thalidomide was widely approved, and I'm glad the FDA didn't rubber stamp it.
• banana_feather 3y ago
  There are "right to try" laws both Federally and in 40+ states. It's unfortunate the author doesn't address those, I'm curious how they interact with his case.
  • amanaplanacanal 3y ago
    It appears that he is in New York, which doesn’t have a right to try law on the books.
    • arbuge 3y ago
      If that is the reason, perhaps temporarily moving to another state might help? The author appears to still be mobile.
      • codexjourneys 3y ago
        Absolutely, the closest state to NY with a "right to try" law is Connecticut. Pennsylvania also has one. https://righttotry.org/in-your-state/ https://righttotry.org/in-your-state/ Edit: Also, the federal law applies regardless of location: https://righttotry.org/rtt-faq/ 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."
• deleted 3y ago
  [deleted]
• dr_dshiv 3y ago
  I really sympathize with the author and feel his position has great moral clarity. Wish there was some country or state where you could try more freely.
  • deleted 3y ago
    [deleted]
• pstuart 3y ago
  This could change if there was enough political pressure. Easier said then done, but it should be done.
• user_7832 3y ago
  I wonder - both in this specific case, and in general - if anyone here on HN has enough media/political reach to spread this message. 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-Claussen.pdf https://www.ntsb.gov/news/events/Documents/child_safety-Clau..., https://www.ucsf.edu/news/2003/10/97119/airline-infant-safety-seat-rule-could-cause-more-deaths-it-prevents https://www.ucsf.edu/news/2003/10/97119/airline-infant-safet...)
• latchkey 3y ago
  If we block someones ability to try novel procedures, how is that any different from blocking their right to assisted suicide?
  • kif 3y ago
    I think guaranteed death is much more different than potential death and potential cure. It's a tough line to walk on, still.
    • latchkey 3y ago
      The only guarantee in life, is death.
      • _Algernon_ 3y ago
        ...and taxes.
      • tekla 3y ago
        The difference is in how painful and how long that death will be
  • amelius 3y ago
    Because access to novel procedures gives us valuable data.
  • lr4444lr 3y ago
    A steel-man response would be that assisted suicide is more about pain reduction, whereas experimental drugs could have terrible side effects. 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.
  • zer8k 3y ago
    But we allow physician assisted suicide. Strange isn't it? Almost sounds like soylent green. Maybe I'll skip McDonald's this week.
• kepler1 3y ago
  I think the consideration that an article like this misses is the unintended consequences of speeding a process up / reducing the criteria for review, approval, etc. 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.
  • jojobaskins 3y ago
    If he's going to die why not give him the option to try medicine that is developed by large players with a good reputation? Weighing historical success into the process seems to make sense to me. Beta programs from Google are usually very functional...
  • tpmoney 3y ago
    >I think the consideration that an article like this misses is the unintended consequences of speeding a process up / reducing the criteria for review, approval, etc. 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.
• deleted 3y ago
  [deleted]
• prmoustache 3y ago
  The author seems to forget that entering a trial doesn't necessarily give you access to the treatment. You have as many chances to be in the control group receiving a placebo. 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.
  • janeway 3y ago
    To note, the placebo group is typically getting the best current existing treatment. Rather than nothing. Although in some cases the best treatment is still nothing, which sucks.
    • peter422 3y ago
      It is against medical ethics for the placebo not to be the current standard of care, and experimentally it must be… If you can’t beat the current standard of care then your drug isn’t effective.
  • timmytokyo 3y ago
    Right now he has a 0% chance of survival. If the experimental treatment is at all effective, the probability of not being in the control group multiplied by the probability of effective treatment is greater than 0%.
    • Arainach 3y ago
      The probability of a more painful death is also greater than 0%. So it the probability that something could go wrong and cause publicity which would interfere with the testing or approval of the drug and affect other people's access in the future*. There are always tradeoffs. *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.
• sam2426679 3y ago
  I'm sorry this is happening to you. Thank you for fighting to improve SCC outcomes for yourself and others. Please keep us updated with your progress.
• gburt 3y ago
  Regulators are, in general, too sheltered and disconnected from the impact of their actions. 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.
  • janeway 3y ago
    I would disagree with this. Regulators that I have worked with in US and EU knew what it meant. Maybe not the most amazing technical people I’ve ever worked with, but they were as competent as any random sample of pharma/tech types. Their jobs is to verify that you have completed all the valid documentation to demonstrate that your drug does what you claim it does. Their opinion on personal emotions are irrelevant. They are supposed to be guideline checkers.
    • gburt 3y ago
      I understand some people are checking boxes, with no space to consider their impact. Please generously reinterpret my point to extend to their management stack and the political establishment that is responsible for the system. 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.
      • Arainach 3y ago
        The consequences to their actions are that when a doctor prescribes me a medication or when I purchase one off the shelf, I have incredibly high confidence that if I follow the dosage instructions it is safe - safe now, safe in 15 years, not worth further thought on my part. That hasn't been true in most places for most of human history. 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 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
• strangescript 3y ago
  There is no 100% correct answer here. As soon as you speed up the process, there are still going to be people that just miss out, and then we will have people saying 4 months is too long, it should take 2 months, or let AI approve it, etc. Desperation skews perspective. 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".
  • baggy_trough 3y ago
    [flagged]
    • bruce343434 3y ago
      ...why?
      • baggy_trough 3y ago
        [flagged]
        • bruce343434 3y ago
          You are enabling quack doctors
          • baggy_trough 3y ago
            You are killing people under the guise of helping them.
            • kortex 3y ago
              Giving someone Treatment X likely means they won't be pursuing Y, whether it be to avoid polypharmacy drug interactions, or simply because the patient prefers X's side effect profile to Y. Y may be the proven treatment, with worse side effects but better efficacy. Even if the doctors inform the patient "X may not work at all and may make it worse", the patient might still choose it. "What's the harm?" you say. The problem is, without any threshold, there are countless Treatment X. Every single snake oil comes out of the woodwork.
• jbullock35 3y ago
  Highly relevant: research on ways to accelerate the medical-trial process. Here is one example from JASA: https://doi.org/10.1198/016214504000001790 https://doi.org/10.1198/016214504000001790.
• kepler1 3y ago
  Another topic for discussion related to this (and I hope we can be detached enough to discuss the intellectual side of this impersonally): 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.
• deleted 3y ago
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• light_hue_1 3y ago
  Blaming the FDA is popular but the problem is not the FDA itself. Drug manufacturers are part of the problem. 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/2022/07/22/1110830985/drugmakers-are-slow-to-prove-medicines-that-got-a-fast-track-to-market-really-wo 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-business-premature-births-a6032ee4b00ee507335892e9f3ce466b 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.
  • zer8k 3y ago
    You're missing the part where the FDA has a revolving door with the biggest pharma companies. To think the FDA is some flawed, but impartial, judge of drugs is the key problem. The FDA is controlled by big pharma. No more, no less.
    • 3y ago
• tqi 3y ago
  > think how bad it will look that we approved the drug so quickly 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.
• devwastaken 3y ago
  When a gov org cares about how it will "look" over wether it kills you - you live under tyranny. 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.
• A_D_E_P_T 3y ago
  There's a very easy regulatory solution: Roll the drug approval process back to the way things were done prior to 1962. Back then, safety testing was all that was required. Efficacy testing -- which is difficult, expensive, arguably unethical in itself, and in some cases effectively impossible -- was not required. Drugs cost ~20-50x less to bring to market, and were brought to market faster. Indeed the 40s and 50s are still known as the pharmaceutical industry's "Golden Age." And it wasn't only because of low-hanging fruit. 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.
  • TheRealPomax 3y ago
    The world "simple" is doing so much heavy lifting here, it might as well be written as ______ instead.
  • throw9away6 3y ago
    I’m fine with that as long as insurance does not pay for any drugs without efficacy testing. Also no advertising of any sort.
    • fnord77 3y ago
      insurance already gets away with not paying for some highly efficacious drugs, so I don't think this will be a problem
    • sterlind 3y ago
      generally, insurance companies do require efficacy. many of them have decided not to cover aducanumab for that very reason. though this also gives insurance an additional excuse to deny stratospherically-expensive gene therapies and other life-saving new treatments, since even the most effective new drugs won't have e.g. 10 year survival rates until they're a decade old. agreed on advertising though.
• gabea 3y ago
  There is a clear problem, and a good idea here. (i.e. Right to Try) What needs to happen to make this move forward? 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?
  • Google234 3y ago
    What about the right to refuse? I don’t think there actually is a problem here, there’s no reason for this Pharma company to provide an expensive untested drug- only potential negatives
• gumby 3y ago
  This quote sounds like self interest in the article, but there is more to it: > 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.
• abhaynayar 3y ago
  I read this, and while I felt immense sadness, I will obviously move on with my life soon enough and forget about it. I wondered, if I were ever in the same situation, and someone read what I wrote before dying, they might empathize for a bit, but then they'd move on as well. 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_Byte 3y ago
    Let us say that there is nothing beyond death, does that really mean that life is ultimately pointless? I'd argue that the idea that there is nothing beyond death is what makes life all the more meaningful. We are only going to get one shot at all of this, we better make it count. 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.
    • kortex 3y ago
      You can philosophise yourself into just about any position: death is real & life is meaningless, death is real and life is meaningful, death is but a transition & this life is meaningless, death is merely an illusion and life is meaningful,
  • grrdotcloud 3y ago
    Wishing is one thing. Hope is the better thing. There is something beyond death. I don't know how anyone can think otherwise. Who else has the words of life?
    • deleted 3y ago
• woodpanel 3y ago
  Things don't happen in a vacuum. It’s understandable that people want access to the experimental medicine for themselves or their loved ones when the fear of god has been put into them. When one has nothing to loose, what’s there to loose? 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.
• dghughes 3y ago
  My Dad was dying of IPF and I was always on the hunt for anything that may help. Of course there are countless quack "cures" but I came across a study that said metformin may help. Metformin is what people with type 2 diabetes use to help to control blood lipids among other things. My Mom actually takes metformin since she has type 2 diabetes. My Dad's doctor wouldn't even discuss it and dismissed it outright. 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.
  • serial_dev 3y ago
    Anecdotal. When "quack" * science helped. 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
    • datori 3y ago
      I had a similar presentation: sudden 1-3% PAC load at rest following an asymptomatic COVID infection. Cardiac MRI showed minor residual scar tissue. I noticed my diet was pretty seriously deficient in magnesium, and after starting a supplement the abnormal beats have nearly vanished.
• deleted 3y ago
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• youssefabdelm 3y ago
  There was a drug called Rapamycin discussed in this Radiolab episode https://radiolab.org/podcast/dirty-drug-and-ice-cream-tub https://radiolab.org/podcast/dirty-drug-and-ice-cream-tub 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.
  • adamredwoods 3y ago
    This is an mTOR inhibitor, I believe there are others already in clinical trials. I know PIK3 inhibitors are used to block some cancers. Most inhibitors stop cancer for "a while" but cancer cells have the ability to circumvent pathway disruptions. https://en.wikipedia.org/wiki/Sirolimus https://en.wikipedia.org/wiki/Sirolimus https://en.wikipedia.org/wiki/MTOR_inhibitors https://en.wikipedia.org/wiki/MTOR_inhibitors
    • xk_id 3y ago
      PI3K inhibitors only work if the tumour tests positive for that special mutation.
• snitty 3y ago
  There's a wild book from 2006 called Overdose (by Richard Epstein), that makes a more or less libertarian argument for deregulation of the drug industry. What the book misses is that before the FDA and the Pure Food and Drug Act is that anyone who thought they could make a buck by poisoning as many people as possible did exactly that. And, especially given the "tort reform" prevalent in red states, there is fairly minimal disincentive for corporations to kill people in the US.
• xk_id 3y ago
  The reality of cancer is a bit paralysing to anyone who has had direct exposure to it and there is nothing really overstated about it in the common sense of culture. Everyone’s fight is different, because there are so many paths to take and they keep changing as research advances, but the time available is limited. Besides being restricted, cancer drugs are also notoriously expensive, but for the more self-determined individual there is the option of off-shore pharmacies (many based out of India) and perhaps even Chinese chemical labs (anything on AliBaba must be assumed to be completely unreliable; however, some of the labs which are referenced by scientific papers as suppliers are actually happy to do business after only a little bit of social engineering). Unfortunately this approach is limited to relatively simple pharmacological interventions; anything that requires complex medical procedures (including palliative care) and has to happen in a hospital will require normal legal compliance and the cooperation of a doctor. There are stories of people who have gone far and beyond to enrol into cutting edge clinical trials [1] – usually with the energetic support of a loved one. In those cases, even with the consent of a doctor, the logistics and expenses aren’t trivial. There is much to say in general about the questionable role of institutions who gate-keep access to medical procedures under the pretext of protecting reckless consumers – but it wouldn’t feel right to steer the conversation too much away from cancer. > 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-trials-cancer/ https://time.com/4270345/immunotherapy-pembro-clinical-trial...
• rediguanayum 3y ago
  (not an MD) The FDA does allow for compassionate use of experimental drugs. My understanding the many early stage drugs are tested this way, but access is limited to trial centers. The author should try search drug trials database to see there's a group that is recruiting: https://classic.clinicaltrials.gov/ct2/results?cond=Head+Neck+Neoplasms&term=vaccine&cntry=&state=&city=&dist= https://classic.clinicaltrials.gov/ct2/results?cond=Head+Nec... My brother is a physician who is working with patients in drug trials. He had the very good fortune to see an experimental drug successfully treat a very sick, critical patient who was declared a candidate for "compassionate use".
• balderdash 3y ago
  Not having some level of “right to try” is abhorrent. 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?
• formulathree 3y ago
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• gilbertmpanga12 3y ago
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• eilertokyo 3y ago
  Unclear to me why clinical trials seem unavailable to him, which is how he would gain access to these treatments. The major cancer centers generally will have options for his exact situation, though sometimes they require he finish conventional and salvage treatments first.
  • balls187 3y ago
    I understood your question to mean “why can’t he have access to clinical trials?” 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.
  • abecedarius 3y ago
    He wrote "The FDA was loathe to approve initial mRNA human trials". The implication seems to be that if not for this added delay, this potential treatment would be further along.
    • eilertokyo 3y ago
      That doesn’t make a ton of sense, because there are plenty of phase 1 early stage trials at the largest medical centers in the country that use pre-“FDA approved drugs“. The whole purpose of the early trials is for the companies to go get FDA approval. 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.
• ilamont 3y ago
  How horrible and frustrating. There is a fast-track approval procedure described here, I wonder why it doesn't apply to these treatments? https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track 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-documents.html https://www.nytimes.com/2020/03/23/health/thalidomide-fda-do... 2. https://thalidomide.ca/en/the-canadian-tragedy/ https://thalidomide.ca/en/the-canadian-tragedy/
  • deleted 3y ago
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• rdl 3y ago
  I hope more and more medicine/science moves outside the reach of FDA. If someone is willing to pay for a treatment (or recreational product), and is reasonably informed/competent to decide the risks, and it's not a risk to anyone else, go for it. If someone tries to prevent consensual commerce (possibly while wearing a silly uniform or carrying a printed ID badge from a state), violate the law if on balance it's worth it to do so, and if it were literally my life at risk, I'd use whatever force required to accomplish the transaction. Hopefully treatment would be on the table before I lost the ability to run a carbine properly if needed. 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.
  • denhaus 3y ago
    two questions: - 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…?
    • rdl 3y ago
      I mostly go to expat/tourist-focused high-end clinics in Asia (there are a bunch of options; mostly depends on which country you're going to be in -- Thailand, Singapore, Japan are particularly strong). You can get a really comprehensive physical (cardiac calcium, exercise stress test, imaging, comprehensive blood panels, multiple doctors/specialists, etc. for <$1k -- something comparable in the US would be $5-10k (e.g. https://my.clevelandclinic.org/florida/departments/executive-health https://my.clevelandclinic.org/florida/departments/executive...). I travel enough for work that I can just go a few days before/after a meeting or conference, so no incremental cost (maybe an extra day or two of hotel). 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.)
• balls187 3y ago
  I don’t know why, but this was heart breaking to read. I don’t think it was meant to be.
• can16358p 3y ago
  There should be a universal law in human rights stating anyone can take anything into their own bodies and governments can never, ever prevent them. 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.
  • hotpotamus 3y ago
    I mean, fentanyl? Obviously it's a whole subject that could be unrolled/debated, but I don't have any interest in doing that today. Really, I thought I'd just point out one interesting and often unknown aspects of US/State law (assuming this is mostly a US-based forum; I know you said universal human right) - taking drugs isn't a crime anywhere that I'm aware of. Possession of them certainly is, but just having taken them is not.
    • can16358p 3y ago
      Yeah. 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.
      • SanderNL 3y ago
        It’s not about any one person in particular. It’s about whole communities descending into hell for what could be multiple generations. I know that in theory we all should be free and take whatever we want, but I know that in practice this doesn’t end well. There are good reasons to control consumption of some classes of drugs that don’t have anything to do with religion and/or fake sense of morality. Some things just mess us up too hard and especially some people who are susceptible to it for various reasons.
• fnord77 3y ago
  separate efficacy testing from safety testing
• swayvil 3y ago
  There is a religion. In it, good people are wealthy. Bad people, less so. It's a popular religion among the well-to-do. As you might imagine.
• jstummbillig 3y ago
  This seems bad. Help me understand. 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?
  • HWR_14 3y ago
    If it were for science alone, it might be fine. The problem is when Steve Jobs is sick and I walk up to him and sell him an experimental cure for $2,000,000,000. He can trivially pay it, but I have a huge incentive to lie. At a smaller scale, a more efficient conman can take all of someone's assets. And not just the dying person's. How many people wouldn't sacrifice everything to save a spouse's life or their child's? The other concern is when you turn down medicine that might work for snakeoil that doesn't.
    • tlb 3y ago
      The lying seems like the problem there. Present the patient with accurate records of previous trials and outcomes, let them make an informed decision (with help from their doctor, if they're not scientifically literate) and I don't see a problem.
    • jstummbillig 3y ago
      Could the former be fixed by requiring experimental drugs to be free of charge, or would that break how drug roll-out works? Somebody suggested an escrow account in this thread, which also seemed interesting.
      • HWR_14 3y ago
        I mean, the experimental drugs are already provided free of charge to the group participating in the study. I have no idea how it would change the economics if the ones given under any kind of "right to try" were also required to be free. As of now, "right to try" drugs are supplied by the manufacturer under whatever terms they want at their discretion.
• deleted 3y ago
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• sam537 3y ago
  You should also try immunotherapy (pembrolizumab) with chemo if your CPS is >1% before experimental early phase trials.
• sgseliger23 3y ago
  This is my brother. I love you. 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-with-jake-s 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.
  • ayewo 3y ago
    The surname is very familiar. I checked and Jake is in fact a long-standing member of this community as jseliger [1]. I’m sorry for the very difficult times your brother is going through. I hope and pray that his situation improves. 1: https://news.ycombinator.com/user?id=jseliger https://news.ycombinator.com/user?id=jseliger
    • croisillon 3y ago
      more specifically, this topic has been brought here https://news.ycombinator.com/item?id=36393383 https://news.ycombinator.com/item?id=36393383
  • Mizoguchi 3y ago
    Lost my brother to a carcinoma, a disease that most likely will be successfully treated, cured and even prevented in my lifetime. I feel for your brother but also for you.
    • sgseliger23 3y ago
      Sorry for your loss. I hope you’ve found some peace.
  • thumbuddy 3y ago
    This is the real reminder for young people. Every single one of us is one tiny cellular mishap away from an untreatable, agonizing demise. Be KIND to one another, because when it is your turn to die, you will have wanted to have had a positive impact on the world and those around 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.
• robomartin 3y ago
  Trump got a federal Right to Try experimental drugs act passed in 2018: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7309195/ 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.
• foobarbecue 3y ago
  I thought the recent New Yorker article on FDA's drug approval policies, "When Dying Patients Want Unproven Drugs", was balanced, insightful, and well-written. It covers Thalidomide, and focuses on ALS and HIV. https://archive.is/u6vmI https://archive.is/u6vmI
• HWR_14 3y ago
  I would hate to ask the author this, as it seems cruel, but is the FDA denying him access? As he points out, Moderna has two drug trials going. The FDA "right to try" page[0] clearly spells out that Moderna could give him unproven medicine currently in a clinical trial under the right to try laws. So Moderna could let him into one of the two studies, or give him the medicine under the right to try laws if it rejects him. 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-other-treatment-options/right-try https://www.fda.gov/patients/learn-about-expanded-access-and...
  • CogitoCogito 3y ago
    I think the issue is that Moderna just doesn’t have much incentive to do it and it’s more a distraction than anything. But yeah it could also just be too late. My dad was recently in a similar position, but by the time he theoretically could get his hands on the drug, he was too weak too handle them.
  • adamredwoods 3y ago
    I believe the oncologist has to present the case to Moderna for use. If insurance is involved, insurance will deny it.
    • hgsgm 3y ago
      Fairly certain that someone who has exhausted all supported treatment options doesn't care about insurance anymore.
  • haldujai 3y ago
    Actually the Moderna trials are recruiting metastatic and treatment resistant disease. From his description he has not met inclusion/exclusion criteria for the studies due to treatments received and not received rather than being too advanced in stage. 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.
• technick 3y ago
  I feel somewhat closer to this person because I was once in his position as a child fighting a neuroblastoma in 1981 and some how my mother found a way to get me on an experimental list which saved my life. Half of the kids that took the drug died by the time they were teens due to heart failure, obviously I wasn't one of them, but am thankful for the chance to live a full life. 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.
  • A_D_E_P_T 3y ago
    > When the odds are against survival, there shouldn't be any rules limiting treatment, if it means even a slightly higher chance of winning. 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.)
    • happysadpanda2 3y ago
      I am not yet done thinking on this area, so my thoughts here are incomplete, but thay said: 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.
• olliej 3y ago
  This is blaming the FDA for lack of treatment, not the recentness of the treatment. 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.
• ayewo 3y ago
  Jake, even in the face of declining health, don’t despair. I know it’s difficult but please, don’t lose hope.
• donall 3y ago
  One aspect of the problem here is the difficulty in running a clinical trial, particularly at the recruitment stage. The covid-19 trials all had a surfeit of participants because of a pandemic, but with modern cancer treatment trials the qualification requirements significantly cut down on the eligible population. 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!]
  • nradov 3y ago
    Yes, very true. Beyond just access to clinical data there are often major differences between how the same conditions are recorded between different provider organizations based on EHR data models and local practices. Researchers who want to use data from multiple organizations typically have to put a huge amount of work into their data pipelines for cleansing and normalization. Some standards development organizations such as HL7 (including their various FHIR accelerators) are now writing more detailed and specific implementation guides to improve data quality and consistency so I would encourage technologists to contribute to those projects.
• kortex 3y ago
  Why don't we just have a schedule/tier system? 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.
  • haldujai 3y ago
    > 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 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.
    • 3y ago
• jddj 3y ago
  I was an early investor about 20 years ago (nothing to write home about) for a company in this space. Small molecule therapy though, not MRNA. 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.
• pengaru 3y ago
  According to wikipedia, many types of squamous cell carcinomas are associated with HPV infection. [0] 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_body_location https://en.wikipedia.org/wiki/Squamous_cell_carcinoma#By_bod...
• adamredwoods 3y ago
  For the record, FDA has fast track / breakthrough therapy options: https://www.fda.gov/patients/learn-about-drug-and-device-approvals/fast-track-breakthrough-therapy-accelerated-approval-priority-review https://www.fda.gov/patients/learn-about-drug-and-device-app... Biden's Moonshot program is trying to drive money to support new innovations: https://www.whitehouse.gov/cancermoonshot/ https://www.whitehouse.gov/cancermoonshot/
• notme1234 3y ago
  Some FDA APPROVED cancer medication cause many severe side effects, for example, Opdivo/Nivolumab[1], my father used it and it seems to help, but caused other long term side effects, which seems will never pass. 1: https://www.opdivo.com/potential-side-effects https://www.opdivo.com/potential-side-effects
• oaktrout 3y ago
  Hopefully he makes it into a clinical trial. In case anyone is interested in reading what trial options are available for this type of cancer you can find the trials here: https://www.clinicaltrials.gov/search?cond=Squamous%20Cell%20Carcinoma%20of%20the%20Head%20and%20Neck https://www.clinicaltrials.gov/search?cond=Squamous%20Cell%2...
  • HWR_14 3y ago
    There are 469 studies for that type of cancer that are accepting males of his age, of which 260 are in the US, on that site. Obviously, many are going to be inappropriate, but hopefully with something is available to him.
• raygelogic 3y ago
  I am so sorry for what's happening to this man, and I hope he finds peace in his last days. 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.
• sbuttgereit 3y ago
  Parts of this problem are part of what a company a friend of mine works for is trying to solve: https://xcures.com/ https://xcures.com/ 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.
• happytiger 3y ago
  Ok, so how do we fix this? Can we put this community into action to fix this problem? 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?
• FreshStart 3y ago
  Spare the condokescenes, spoilt he action. Less wasteful comments, more letters to representatives.
• jbritton 3y ago
  I’m not a Dr., I’m not recommending anything, but you might want to look into the following: I see the FDA approved drug cemiplimab-rwlc for squamous cell. https://www.skincancer.org/blog/new-treatment-for-advanced-cutaneous-squamous-cell-carcinoma/ 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.
• refurb 3y ago
  As other comments have pointed out, I'm not sure why the author can't access experimental therapies. Clinical trials or even compassionate use program (prior to FDA approval) are very common.
• BrandoElFollito 3y ago
  I saw both of my parents go through cancer (they both, so far, survived), my wife has MS - so I was quite interested in the "perspective" treatments. 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
• agumonkey 3y ago
  Courage and best of luck for jake, his closed ones and anybody hit by hard to cure diseases.