You have a sample size of one, and no control group. The patient dies. Are either of the drugs safe individually? Can't tell. Maybe one caused death and the other was fine. Maybe the two interacted in the human body somehow.
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.
Nope, Bayes makes the second situation (and to a lesser extent the first) informative since the probability of a life-saving second-order interaction between otherwise dangerous drugs is small compared to the probability of the toxicity having a more or less cumulative effect.
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.
> but it strikes me as more than a little ungrateful to pretend that the experiment was scientifically worthless.
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.
Again, no. Just because the result turned out okay, the way how it was done does not become good (or scientific). There is no reason why it could not have been done in two persons.
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.
She put a life (or health) unneccessary at risk, if it is hers or someoneelses does barely matter at that point.
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...
No one is claiming the result is scientific or in any way equivalent to a proper study. But that doesn't make it worthless either. It demonstrates that it is at least possible to do this successfully in one human subject, which lends substantial credibility to the pursuit of the endeavor.
An enormous amount of science has been built on scientists testing things on themselves. Here's another excellent example:
If she is successful with all this, she won't be able to sell it in an FDA approved manner, but she will have an extended life/health span which is its own reward. Not everything is about money.
>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.
Medicine has a long history of self-experimentation:
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.