Earlier quoted context omitted.
>In cases where an experimental therapy has at least some limited evidence (i.e. suggestion it might work) it’s quickly approved and pushed into clinical pipelines, osimertinib/Tagrisso for lung cancer is a recent example of such a treatment. I think that's the main point of disagreement. There are some encouraging cases where the process works well, and it's fair to point to osimertinib as an example. But there is a…
> Most drugs fail. But getting the drugs that do work to patients even a year earlier may be a much larger benefit than the cost of giving people with serious diseases broader choices, in addition to the current best known standard. This is contradictory. Most drugs fail, how do we identify the efficacious ones other than through trials? Depending on the anticipated efficacy from early trials as compared to existing…
One idea is to make trials somewhat less costly by relying more on post-approval monitoring, but there are more subtle critiques of the processes that try to address perceived inefficiencies at the organisation/bureaucratic level rather than just adjusting a single big strict/lenient dial.
Some drugs do not make it to trial at all due to the overwhelming cost of the process. We would identify a larger number of efficacious ones if there were less costly, wider trials. A very expensive approval process results in delays for drugs that do make it to trials, but also causes many candidates to never reach trials.
>Depending on the anticipated efficacy from early trials as compared to existing ones it will get fast-tracked before the phase II is completed.
Part of the argument is that this is good, and we should consider going further in that direction
>Where is the evidence that we’re delaying efficacious therapies where there is no good alternative?
Unfortunately, the best I can point to is a series of blog posts and informal discussions, I'm not aware of any formal published evidence. I remember a blog post on ACX post aducanumab (https://astralcodexten.substack.com/p/adumbrations-of-aducan...). The "FDA delenda est" catchprase leads to several more posts.
I'll admit this is not very compelling. I believe it's worth having that argument, and I'd be happy to be wrong on that one, but all of this is hard to quantify.
(I also want to reaffirm that there are good alternatives, it's a question of whether we could be losing less people than we are losing under the current process, not that there are no current alternatives)
>I sound like a broken record right now but this blog post is suggesting we jump to experimental therapies (which would be off label for the author as he does not meet inclusion criteria) before we even have phase I data let alone efficacy, when good validated treatment options exists.
I'm sorry if I'm repeating myself as well or failing to make a good argument. I think your position is very reasonable. It's a complicated topic and I might not be doing it justice.