Is it needed and / or ethical in this case? Alzheimers patients don't spontaneously recover, seems immoral to deny them treatment via a placebo when there is no possibility of improvement from their current state. I'm not a scientist or anything, just curious what normal procedure is here
Needed, I would say yes, definitely. Having controls is really a core tenet of science.
I wouldn't really think it's any more unethical than any other clinical trial for any other disease. The whole point is you don't know if the drug/treatment works, so I don't think it's unethical to withhold it. All participants are informed that there is a chance they'll receive placebo treatment as well, no one is being tricked. The point of the trial is to test the treatment, not to just fix a small group of people before the big roll-out.
We know what Alzheimer will do to a person. Don't we have enough data to determine if a new treatment works. I lasted 10 months in clinical trials, and still think it is unethical in the extreme to put in control groups for things we know will kill you. Having watched a control group lose 70% of the people while the people getting the real drug survived is just inhuman.
> Having watched a control group lose 70% of the people while the people getting the real drug survived is just inhuman.
Again, at this stage you're just assessing if the treatment even works. The point isn't to fix one group and screw over another, the whole point is that you don't know if the drug is going to do anything. It's much easier to tell if a treatment is worth giving to everyone if you first do the correctly controlled trials.
It seems like you're coming at this from a place of assuming the treatment is going to work from the get go, which is not true for the majority of treatments that undergo this kind of trial. Moreover, many treatments have side effects that, again, are much easier to look at if you can compared them to a group that went through the exact same handling. Even small, seemingly innocuous things like going to a hospital to talk to an interested person about how you're doing can have an effect on health readouts that may be relevant to a study. Or maybe the stress of sitting down having this weird hat put on your head affects something. Or maybe some much less obvious aspect of the study. The point of the control is that you cancel out all of these kinds of effects, obvious or otherwise, so that the ONLY difference is the treatment.
Controls also help with allowing double blinded studies. If the researchers know who is receiving treatment and who is receiving placebo, they can unconsciously influence the results, especially for things like behavioral surveys that aren't as objective as, say, taking blood and seeing if you have more or less cancer cells. Obviously you can't have this kind of blinding if the researchers know every single person is getting the real treatment.
In certain fields, such as oncology, the control group receives the "standard of care," that is, the standard drug of choice. Even for terminal stage cancer, I believe such trials are ethical because there is no guarantee that the new drug is going to be more effective or even not kill you.
The control group doesn't need to be treatment-free.
So, you can't have enough data to know if a new treatment works without doing a study with a proper control. You need to control to know if the new treatment works, generally.
But, you are right that sometimes the 'control' portion of a study is halted and the control people are given the treatment if a study seems to be going especially obviously well, on ethical grounds.
It can be some difficult ethical decisions. But whether you have 40 people in the experimental group and no control group, or 40 people in the experimental group and 40 people in a control group, you still only have 40 people in the experimental group.
Not having a control group doesn't somehow get the treatment to all effected people in the world. And it would be unethical to give the experimental treatment to everyone in the world _before you have evidence of it's efficacy_ anyway. It might be ineffective, it might even make the condition _worse_ or have other dangerous side effects in addition to being ineffective. You don't know this until you study it properly. Studying it properly means using proper valid statistical techniques, and may mean having a control group. I think it is unethical to give a treatment to people widely _without_ having done a study with a control. Being a part of a research study can be _dangerous_.
Anyone that participates in a research study, knowing the risks, is providing a valuable service to everyone else that may later benefit from what is discovered, whether they end up in the experimental group or the control group. They are taking on risks of a still experimental treatment to help get knowledge to help others.
I'm not saying it's simple. I'm just saying that it is definitely not as simple as thinking having no control group is somehow more ethical than having a control group, or that it's good idea to widely distribute experimental treatments which haven't yet been properly studied. You will harm people by doing so.
You need to control to know if the new treatment works, generally.
That's my dispute. We know X will kill you in a certain time frame. We have thousands if not millions of data points. I fail to see how the control group is really telling you anything you do not already know. Ignoring what we already know is unethical.
It is just is insane that we set people up for false hope and watch them die in droves. I guess it looks better on the marketing material to doctors. I wonder how many actual studies are halted because of die off in the control group. It sure seemed like that wasn't going to be an option.
> That's my dispute. We know X will kill you in a certain time frame. We have thousands if not millions of data points. I fail to see how the control group is really telling you anything you do not already know.
I don't think you understand the purpose of the control group.
It is irrelevant how much we already know about what Alzheimer's disease does, because the control group is not intended to tell you anything about that at all. The justification for the control group is not that it will tell us something about what Alzheimer's does.
The purpose of the control group is to determine if the treatment works. That's it.
You seem to be suggesting that when doing a research study of a new treatment, it would be unethical not to give this treatment to everyone on the planet who has the disease. That makes no sense at all. The experimental treatment may do nothing for them, or may actually harm them. Giving an experimental unproven treatment to everyone would be unethical.
If you want to argue that it is unnecessary to have control groups in an experiment, you need to first understand what the purpose they are said to serve by those who believe they are important (which is pretty much any statisticians or methodologists).
One of the reasons for a control group is to provide opportunities for blindness and double-blindness. Placebo effects are (hopefully) very well-known, but that's not the only common cause of systematic error. Allegiance effect also exists--a doctor may score the same patient with the same symptoms differently if there is a reason to favor one interpretation over another, and it need not even be a fully conscious effect.
Obviously you can't give all Alzheimer's patients your experimental drug/treatment that isn't even shown to work yet. That would not make any sense, and would not in fact be ethical.
So, yeah, that is how controls work, generally. If there is some other standardly accepted treatment for Alzheimer's already, it might be unethical to _refrain_ from giving that, perhaps the control would be given that too. But it's not unethical not to give someone the treatment that you don't even know if it works yet.
Usually. As a general principle. These are certainly interesting things to talk about in the field of medical ethics, and you can find lots of discussion of them in article and such, it's not always totally straightforward or universally agreed upon and of course medical researchers do unethical things all the time.
But simply having a control group that does not get the treatment you are researching is not by itself considered unethical. Which seems to be what you are suggesting. Remember that you don't know if the experimental treatment works at all, that's why it's being studied -- it may even make the condition _worse_, or have negative side effects.
And yes, having a control group is normal procedure. And that's what a control group is, starting off in the same place as the experimental group, but not receiving the experimental treatment, with other conditions being normalized the same. If the control group is 'double blinded', then neither the subjects nor the research staff know if a given subject is receiving the experimental treatment or control.
Yes, because there are more factors than the treatment itself, and it is important to be able to ascribe benefit/harm to the treatment vs. the other factors.
As mentioned in another comment, the sad state of Alzheimers patient care today includes infrequent attention. It is plausible that two visits from another human each day would in itself cause the same kind of improvement seen in the study.
Without a control group, it is impossible to ascribe the results to the treatment or the other aspects of the study.
It is definitely needed and ethical. Consider that most there are many many people in the world that don't get to participate in this experiment at all, neither as active nor as control.
Being control just means you are still part of that status quo.
If we had like 99.9% confidence that the procedure would work, sure roll it out to everyone ASAP, but I don't think we are there yet.
It'll be needed eventually, if progress with the treatment continues, to quantify exactly how much of a difference it makes.
But say you've developed a treatment device for heart attacks. Untreated, let's say the death rate is 90%. With your treatment, the death rate is 20%. Even without running a controlled experiment where half the heart attack patients get the deactivated device, you can state pretty strongly that your device has better-than-placebo results.
In before "the placebo effect is real!" Well, sure it is. But it's most effective for subjective complaints, such as for pain relief, where the person's mental state is responsible for a large part of the experience. Placebos aren't so great for objective issues like "cardiac arteries are experiencing insufficient blood flow", "liver has cancer", or "patient has late stage Alzheimers".
The standard is to have a placebo/control group because then you know for sure if the treatment is responsible for any improvement. Most new treatments either fail or provide a small benefit, however if the treatment is found to be extremely effective the trial is stopped and all participants are given access. As you pointed out, it would be unethical to deny access. This happened recently with two Ebola drugs that were being tested during an outbreak.
But what if the procedure somehow makes brain functionality worse? Wouldn’t a control group of Alzheimer’s patients falsely attribute that decline to the disease rather than the treatment?
Though I suppose you run into the same issue the other way, too. If the treatment somehow improves everyone’s cognitive function it’s difficult to measure because your “baseline” has moved.
