[xr4ti]

The FDA specifically (and famously) does not regulate the practice of medicine. They regulate the marketing of drugs and medical devices - mainly that you cannot market something as an safe and effective treatment for a disease/condition without proof of (a) safety and (b) efficacy (or effectiveness), which is obtained through clinical trials.

Adherence to "evidence based" therapy guidelines is typically enforced via malpractice claims. Obamacare tried to introduce incentives for outcome measurement/tracking and evidence based care by either cutting or supplementing medicare reimbursements, but even that has been difficult to implement nationally across the 5-7 most frequent and costly conditions and procedures (e.g. acute myocardial infarction, COPD, knee replacement, etc).

While the FDA's regulatory approach does slow the pace of innovation, they exist for a reason -- I acknowledge that GP is not calling for any policy change. Snake oil salesmen were a real thing, and still abound in the valley albeit in different form.

[maxander]

That the FDA does not regulate the practice of medicine is technically true- doctors can and do use (approved) medicines for non-approved uses. But as you also mention, this informal, "non-evidence based" approach has downsides such as the risk of malpractice suits. If a doctor tries treating a sepsis patient with corticosteroids and vitamin D, instead of with the current "evidence based" standard of care (massive doses of antibiotics,) and then the patient dies, the patient's next of kin can sue for massive damages, potentially ending the physician's career- despite it being the medically more effective thing to do. As far as the courts would be concerned, this could be equivalent to the doctor opting for homeopathic medicine in place of antibiotics; one treatment not supported by the evidential standards of medical care is about as good as another, to a lawyer. Since doctors can't risk their medical licenses on a daily basis and stay doctors, they treat patients with suboptimal methods, and people die because of it.

So to say that the issue is funding for FDA approval is probably an oversimplification- equally powerful studies without FDA involvement may or may not suffice, that's a question having to do with law and other non-science factors. But the FDA has established what is the broadly recognized "gold standard" of establishing the medical effectiveness of an intervention.

[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.