I read an article about a new drug which may stop neurodegenerative activity in the brain and this triggered a question I had for some time already.

Why such novel drugs are not tested on seriously/terminally ill patients, to check whether they are not dangerous, and maybe useful?

I completely understand the need for double - blind trials which can assess that the drug works or not. This requires a lot of tests beforehand to make sure that the drug is reasonably safe for humans.

On the other hand I am sure that seriously or terminally ill patients would take the risk to, maybe, have a chance with the new drug, with the risk of complications (all in reasonable boundaries). I know I would.

Beside some indication that the drug works or not (again, I understand the implications of a lack of double-blind trial but if the result is 100% success or failure, it is at least one data point) it could be an indication of its lack of safety. If all patients develop an unrelated organ failure (as it was the case in the article I linked), it would help research which otherwise relies on similarities between animals and humans.

Is it just an ethical concern or are there scientific reasons not to use these opportunities?

  • 1
    $\begingroup$ For one reason: It is extremely unethical. Additionally, such patients are not really the optimal candidates, as they have underlying diseases and treatments, which might affect the outcome of the trial without anybody noticing. Then you want to have a homogenous treatment group to detect negative effects of your treatment. There are some examples of new drugs being tested in patients, but these are then the last hope to get some treatment. $\endgroup$
    – Chris
    Commented Apr 24, 2017 at 8:13
  • 3
    $\begingroup$ Sometimes they are, as mentioned by Chris. But keep in mind, please, that without proper testing (and even sometimes with), drugs may shorten a lifespan. There is an ethical and scientific responsibility to avoid administering drugs willy-nilly, just because someone is close to death. With trazadone, there should be ample evidence out there already, visible through the retrospectoscope. Many drugs have found a new use through this method, including minoxidil and amantadine. $\endgroup$ Commented Apr 24, 2017 at 14:31


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