• dhtseany@lemmy.ml
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    1 year ago

    I know modern scientific processes require blind studies to prove effectiveness of a new drug but imagine being in a grave spot and you’re given access to a potential life saving treatment, you get some hope back that you might pull through but you end up being one of the placebo recipients. Damn.

    • Chetzemoka@startrek.website
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      1 year ago

      I think it’s important to remember that you’re also talking about maybe or maybe not receiving an experimental, unproven treatment that has the potential to make things worse rather than better. If that’s the case, you’d be happy to be the one receiving placebo.

      • ColeSloth@discuss.tchncs.de
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        1 year ago

        It’s just a concentrated high dose of vitamin c, though. It’s already used for other things and deemed safe. You can just buy it off Amazon.

        So in the case of most trials, I’d agree. Less so with this trial, though.

        Also, the article is pretty vague on the details of how much help it really wound up being.

        • Chetzemoka@startrek.website
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          1 year ago

          Oh, that in no way means it’s not going to be harmful in the context of life-threatening sepsis. One of the things the study authors checked for is evidence of oxalate crystals forming in the kidneys because high dose vitamin C can cause kidney stones.

          In healthy kidneys, that’s a survivable inconvenience. In a severely septic patient? Their kidneys are already not working. That could shut them down completely.

          (I’m a critical care nurse. First thing I wondered about was kidney stones.)

    • Pirky@lemmy.world
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      1 year ago

      This was actually a big topic during the AIDS epidemic back in the '80s. However, I don’t remember how they resolved that.

      • n00b001@lemmy.world
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        1 year ago

        I think I’ve heard one semi solution is:

        You end the study before people die/get permanent damage. You should have enough data at that point to show efficacy.

        Then you offer All placebo people the treatment if they want it (after the study)

        • Chetzemoka@startrek.website
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          1 year ago

          You are correct that this is still done in studies where efficacy is very clear.

          In fact, this just happened a few days ago in a new trial of semaglutide to treat kidney disease in diabetics. With diabetes being one of the two major causes of the need for dialysis (the other being hypertension), the drug was so successful at improving kidney function that it would have been unethical to withhold it from placebo recipients.

          https://www.fiercepharma.com/pharma/novo-nordisks-unstoppable-semaglutide-ends-kidney-disease-trial-early-positive-efficacy

          In studies where efficacy is more marginal or harder to demonstrate, then drugs are sometimes given for “compassionate use” to people who are almost guaranteed to die otherwise, so providing an unproven treatment that may work or may harm them is less ethically fraught. That was the basis for a lot of AIDS treatments being distributed back in the day, and in fact AIDS activists were crucial to getting compassionate use rules liberalized.

    • Ranvier@sopuli.xyz
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      1 year ago

      I wouldn’t lose to much sleep in this one though. There’s been a string of many failed vitamin c trials that were much more rigorous than this.

      To more seriously answer your question, there are platform trials, like in the ALS Healey trial, where a single control group can be used as a comparison for many different treatments, reducing the odds of being in a placebo group.

      Ultimately we don’t know if thess experimental treatments work, otherwise they’d already be prescribed. Most drugs fail. And being in the placebo group would shield you from risks that end up being found in the treatment group. The placebo group may in many cases end up better off in the end. So even being in a treatment group shouldn’t really be approached imo like you’re getting “early access” to a treatment, because we don’t know if it’s a treatment yet. I don’t mean to discourage patients from helping in research, but it’s better to look at it mainly as a service that patient is doing for the for the community affected by that disease as a whole and for the researchers.

      If these drugs aren’t rigorously studied we can sometimes end up with even bigger problems for patients down the road. A drug that gets approved on the basis of flimsy data is going to be much more difficult to study further after it gets approved. And now patients and doctors don’t have much information to help them tell if the drug is worth it or not, potentially exposing tons of people to unnecessary side effects or financial costs for something of questionable effectiveness.