Okay, now consider the scenario where the patient lives. Are either of the drugs safe individually? Can't tell. Again, maybe the two interacted in the human body in such a way that a potentially lethal treatment was rendered ineffective at curing or killing.
Now, do I expect regulatory recognition of that fact in proportion to what you would get from a proper phase 0/1 trial? Probably not, and I'm OK with that because of the moral hazard involved, but it strikes me as more than a little ungrateful to pretend that the experiment was scientifically worthless. Someone put their life on the line for all of us, we should thank them and move on.
And btw: applying it to herself is a PR stunt. Just like politicians drinking water from Flint or Fukushima. Though iIt may even be that she had to do it because she would not get FDA permission. If not, still I would not thank her. She put a life (or health) unneccessary at risk, if it is hers or someoneelses does barely matter at that point.
An enormous amount of science has been built on scientists testing things on themselves. Here's another excellent example:
http://discovermagazine.com/2010/mar/07-dr-drank-broth-gave-...
Medicine has a long history of self-experimentation:
https://en.wikipedia.org/wiki/Self-experimentation_in_medici...
In my view, every person has the right to do whatever they want with their own life as long as they are in full use of their mental faculties, act wilfully and don't harm others. And risking life for a good cause (advancing human knowledge) is admirable. And of course, very different from risking other people's lives...
For better or worse, the amount of information you could extract from an experiment through Bayesian analysis is much larger than what would be allowed as "kosher" science. Regular scientific process is safer because it plays safe - but that's also the reason why you aren't organizing double-blind studies when your application crashes. You can move faster by careful inference and in case you get stuck or it leads you wrong, you can always revert to a "by the books" scientific method.
Anyway, it reminds me that someone (that I have forgot) said that the first human that will never die is already born.
As for Aubrey de Grey, he can state whatever he wants to, but that doesn't make it true.
It is, however, noteworthy that a scientific article published in the fairly prestigious EMBO Reports [1] concluded that "In his writings, de Grey fails to mention that none of these approaches has ever been shown to extend the lifespan of any organism, let alone humans."
[1] https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1371037/#__ffn_...
For clinical trials, this leads to very strict guidelines. One of my university professors once told us about a drug they had to test in dogs for some side effects. Nothing happend to the dogs but afterwards they would have been eutanized because it was regulated that these exact dogs cannot enter another study. They finally adopted them, but still. Similar, people entering clinical trials are usually not allowed to take certain drugs, may need to abstinent from alcohol etc.
Keep in mind. This is not an experiment, this is a clinical trial in a human.
(Note: I have one drug in clinical trials, for which I designed the phase I and phase II protocols, and have appeared before the FDA in support of clinical trials of new medications.)
Actually this is not a trial, it is considered an experiment. Trials are controlled (not always blinded or tasted against placebo, as many people think). Strictly speaking you cannot give an unlicensed medication without getting in trouble -- in fact permission to do a trial is actually agreement by the FDA that they won't prosecute you for breaking this law, basically because you followed one of the procedures spelled out in the law and the FDA agreed with your proposal.
You are completely right on interactions. And yes, the animals are pretty much always sac'ed at the end of the experiment, even those in placebo arms :-(
There are a few quasi-informal exceptions to the no unapproved administration. One is if you develop the drug yourself -- hard to prosecute. In a modern corporation however you can't really do that -- if you're a research scientist developing a drug, lawyers could claim that you were forced into it by your boss (another example is that the FDA requires that QC be an independent function). So in practice the only person who can try such a drug is the CEO.
In my case I was both the CEO as well as one of the developers so was confident that the in vivo data we had showed that I would be fine. As a result I was a pincushion (these were true experiments in PK, diffusion etc -- I didn't have the indication we were treating for). The drug hasn't yet gone for approval, but if it does, somewhere in the approval application there will be a description under "previous human experience".
You can do other experiments and trials someplace not subject to the FDA (i.e. other countries). Unless they are also formally part of your FDA process you can't use them in support of approval -- but if you paid for them you have to include them as previous human experience as well.
And there are also various "compassionate use" cases though they are also somewhat-formalized now because some people try to stretch them into trial territory. By definition they are not adequately powered.
There are amusing examples of previous human experience. My favorite is someone who shall go nameless who argued in support of an erectile disfunction treatment in front of his board by dropping his trousers and injecting his penis in front of them. I have only heard of this second hand but I bet there was quite a reaction!
NB for anyone else in pharma: yes I glossed a few terms like calling the NDA an "approval application" in the interest of clarity for those not in the industry.
That was Brindley's (in)famous lecture on papaverine: "How (not) to communicate new scientific information: a memoir of the famous Brindley lecture" http://onlinelibrary.wiley.com/doi/10.1111/j.1464-410X.2005.... The reaction was just as you suppose and everyone here might enjoy reading Klotz's recollection of witnessing it.
If I tell you I invented a cure for cancer, you should be highly skeptical of me. But if I tell you I have a cure for cancer, prove to you that I have cancer, then cure my own cancer - you shouldn't give that treatment FDA approval, but you should sure as hell start the clinical trials process. Whereas if I do not treat myself or anyone else, you should just write me off as a crank.
That is the difference between the world before and after this self-experiment. It upgrades the probability in everyone's mind that this is a real thing and it can really work. It doesn't mean we should all go blindly get the treatment done, but it does mean we should take this research much more seriously. That is its value.