Who would sign up for your study? The diligent person already on truvada - would they risk getting placebo? No. Or the person too careless to take truvada? Would they go to the trouble of participating in a study for a 50% chance of protection? No.
> That would really suck if a loved one was in the program and died because they happened to get the placebo.
Trials like this are performed in desperate situations, and you only ever hear about the success stories. Most such trials probably result in no measurable benefit, and run the risk of an even worse outcome than the placebo.
I think there would be difficulty just to get enough willing participants out of the 5%. I suspect that group at this point has to be the most extreme skeptical holdouts or religious objectors and would likely be adverse to any clinical trials.
The same kind of people who already enroll in any other kind of drug trial. "Who would volunteer for that study" doesn't make much sense of a question considering people are willing to do phase 0 studies of new drugs which are theoretically far more dangerous.
to be clear, I think I'd be even more comfortable with some sort of socially accepted procedure to sign up for really experimental trials done by some non-profit seeing entity. Some sort of procedure where you get several conventional doctors to sign off on, you know, that conventional medicine isn't going to cure you, then sign up for something that looks good, but hasn't been fully tested yet.
I'm just saying, going to a for-profit entity and saying "give me something experimental" and then paying the same on success or failure seems like a pretty bad idea to me.
I wonder if you could run a paid study with a proper control group. Perhaps have everyone pay to participate, administer the treatment to half of them, and at the end of the study refund some amount of money (all? all + interest?) to the ones who received a placebo. That way they've only donated their time.
That it's unethical to continue giving half the participants a placebo makes sense.
I am curious though: do they ever continue with the trial, with the patients on the actual drug? Might that be useful for monitoring rare-ish side effects, even if the drug is highly effective?
> Yes, but patients always consent to be randomized to the possibility of being treated with placebo.
This is false as a blanket statement. For specific categories like Cancer treatments, nobody is given placebo in Phase III for example, because their life/survival is at stake. For such clinical trials you prove the efficacy by comparing with the typical survival in other studies/other treatments (overall survival chart) like this: https://media.revlimid.com/wp-content/uploads/mm-hcp-overall...
IIRC There's already a special case where you can volunteer to try things if you're dying. If you have a rare cancer and doctors are like "Six months maybe less" and there's some crazy Hail Mary drug, which could work but isn't tested yet, you can volunteer to try it and see what happens. You can't pay (so it's for science, not a chance to get rich selling false hope), and you must be advised by doctors who have no financial interest, something like that. Judges were like, eh, you're dead anyway, what's the worst that could happen?
This trial was phase I/IIa which I believe means it's combining safety in humans with initial efficacy data. The thinking is, the real treatment is a single dose. If we give participants less than a full size dose we don't learn much from that. If we give them a real dose, we might as well see if it works.
So their endpoints were firstly how is the treatment tolerated, then secondly does it reduce the need to take clotting factors? Hence this good news story.
I don’t think this should be allowed as a treatment, if it’s efficacy hasn’t been established. But I wish there were a trial. I want to be able to live healthily to 100.
It would be damn near impossible to conclude anything from such research without a control. “Randomized double-blind, placebo-controlled trial” is the critical sentence - at best you’d get a correlation which probably has been influenced by the availability of research. Finding truth is freaking hard.
how do they find volunteers for such a trial? If I get it right, the risks are high (including death?) so seems like a big risk, and I suppose they need to start with someone that's healthy to avoid confounding factors?
Right. Most clinical trials don’t work out. Even the ones that do often involve a placebo group. Participating in a trial is probably most suitable for someone interested in altruistically helping to advance medicine. Expecting to get a personal benefit from participating is probably misguided thinking in most situations, unfortunately.
[Edited]: I should clarify that I mean phase 1/2 trials where there’s no efficacy data yet in humans. Phase 3 is a different matter.
How would you suggest a placebo-controlled study (we can easily make it double blind once placebo controlled) for any disease be structured? Please tell me your study design, because an ethical study design that doesn't put lives at risk is the issue.
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