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Comment by gumby | Hacker News Reader

It's an orphan indication which has special rules to promote development. Otherwise, the thinkingngoes, nobody would develop treatments for uncommon diseases.

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The downside of this is a treatment that you can get approved for something rare, but now it is on the market and they can tell doctors it has promise for some common X. Getting approval for treating X would be more expensive because you have to do a larger trial. (you should do a large trial for the rare thing, but since there are not enough people with the rare thing you cannot)

That's almost true, and is a well known part of pharma strategy. For example Botox was initially approved for hyperhydrosis (excessively sweaty palms and armpits) and was used off label for its most well-known application, wrinkles. It was finally approved for wrinkles only recently having been approved for other things along the way.

Any MD can prescribe any (non-opiate) drug for any indication. But the drug companies are prohibited from doing what you say: telling docs about usages they haven't proven in trials. Sometimes the sales rep will drop off a scientific paper (often funded by the drug company) about some other use of the drug -- enforcement in this area is uneven.

If they do want to advertise new uses they need to do new trials. And though existing trial data can also be used, if the dosage regime is different or, say the patient pool tox risks are different (perhaps the new indication is less serious than the orphan one) whole new tox studies may be needed. Which seems fair.

Since the US v. Caronia decision, off-label promotion is more complicated. The judge said restricting off-label promotion violates the 1st amendment, as long as the speech is "truthful"

I'm pretty sure Botox was originally used for Strabismus.

> they can tell doctors it has promise for some common X

That's not authorized, that's called "off label promotion" and it is severly punished when discovered. Also, doctors who use a non-approved drug for another indication can be tracked and investigated as well if needed (conflict of interests). Reimbursment also works only in certain conditions and may not apply if you use the drug outside of its allowed/approved indications.

Slight correction: as noted by others above, off label use while extremely illegal enforcement has been eroded and is patchy. And US law allows any MD (but not other prescribers such as podiatrists or NPs) to prescribe any approved drug for any medical purpose (opioids have recently been somewhat of an exception here).

However, as you point out, insurance companies in practice have a say. Also if the usage is implausible and there's an adverse event I think there could be a malpractice issues so those insurance companies also have an impact.

This is US law I'm talking about BTw.

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