[geofft]

I don't quite understand this - what is the purpose of the FDA process for approval for "compassionate use" drugs? That is, what does completing this process let a doctor do that couldn't otherwise be done?

https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAcc...

(My understanding was something along the lines of, the FDA regulates which drugs can be used/sold but not how, so if a drug was approved for any purpose a doctor can use it "off-label," but a new drug still requires FDA approval. Is that accurate?)

[xr4ti]

I tend think of compassionate use more in the context of medical devices and biologics (e.g. synthetic antibodies) than small molecule drugs. And to reiterate FDA regulation has more to do with marketing (and prescription/reimbursement) than use.

For example, nutritional supplements are often recommended by doctors and incorporated in their practices, but are marketed as "promoting general health and wellness" and not treating a particular condition. Here you don't need any evidence of efficacy (or even safety), you only need to be able to show a "history of use". Many "structure-function" claims push the envelope (ex promotes liver health), but are still legal and FDA has been pretty consistent and clear about where they draw line.

[sjg007]

Compassionate use concerns drugs not yet FDA approved. The doctor needs permission to treat a patient with an investigational drug outside of a clinical trial.

Doctors can use anything already FDA approved off label but they don’t... unless there’s compelling evidence.