Are you sure about that? I've not run a phase-1 but I'm often told the CMC is the most gnarly and expensive part of it & its due to requirements, not sponsor choices.
If you don’t need to make a drug that actually works, you can choose to make drugs that are very easy to produce instead, therefore CMC becomes way way way simpler.
Maybe, but if it's GRAS and supplements/non-drug interventions, can't you do it anyway? This law is addressed at products that are regulated as drugs, biologics and medical devices
- Strong demand from biotechs (first approval decision made and public, Parley Neurotech), we're talking several dozens with $5-300m in funding each
- Clinics a bit more lagging, because licensing takes up to 90 days; in a few weeks we hopefully see the first clinic
- Business models: I'd personally be very excited about open-sourcing phase-1 assets to encourage further development to better drugs by more people
- Yes, this was an explicit hope from the geroscience community to address gaps e.g. in frailty, age-induces diseases with multiple possible endpoints
- No explicit rules on telehealth, but administration needs to be in Montana under a brick-and-mortar clinic; telehealth probably possible within those limits
I think what you're describing is a risk to the sponsor/biotech, not the physician. It is not a clear-cut question though for the sponsor, because US states regulate medical practice. Federal jurisdiction applies when there is interstate commerce, and that risk exposure depends largely e.g. on how you do manufacturing and past FDA enforcement history. Also there are options where some of the activities you need to do you can do through federal right-to-try. So it depends on what you're trying to do but is not "you can't use it" by default.
Physicians get a pretty good liability shield in the law; is that strong enough against malpractice suits? I don't know, but it hasn't been raised much as a problem relative to the sponsor risk
Well that'll be a pretty sophisticated scheme, requiring $5-10m funding per phase-1 trial. I suppose that's possible but it would be a highly legible scheme.
Also what would be the bad outcomes here? If those drugs end up not being safe (that is prevented by the requirement to not withhold safety data), or being effective?
If effectiveness is what you're thinking of, keep in mind things like off-label are already allowed, so even in the current system you have drugs that aren't proven effective for what they're used and we don't call the physicians that prescribe off-label bad actors necessarily.
In what way would it be legible? It would look identical to exactly what the Montana law is designed to produce: more drugs that aren't known to work being released to patients. Obviously a large number of those will end up actually not working (which is why trial failure rates are so high to begin with).
Producing $5-10MM in revenue with a snake oil drug strikes me as trivial.
And keep in mind a substantial portion of the Phase 1 + IND costs are incurred in preparation of actually trying to bring a drug fully to market. If your approach is just to Phase 1, milk consumers, then bail, I'm quite confident you can do this much much much more cheaply.
> Also what would be the bad outcomes here? If those drugs end up not being safe (that is prevented by the requirement to not withhold safety data), or being effective?
A requirement not to withhold safety data is different (and easier to enforce at Phase 1) than a requirement to produce the relevant safety data in the first place. Again, designing a Phase 1 study with no ambition to succeed in Phase 2 and Phase 3 will both enable and incentivize a lot more "creativity" in study design to not detect a problem early on. Whereas today, all incentives are aligned toward detecting problems as early as possible, because finding out your drug is unsafe at Phase 3 is financially very bad.
Is the scheme you describe a risk? Sure. Any new mechanism is, until it has a track record.
What I meant by legible: to run it, you'd have to pass FDA's IND review and reporting requirements, IRB review of the Phase 1, and then Montana ETRB review, with public annual outcome reporting and no withholding of safety data from patients. That's a high bar.
If your view is that nothing short of full approval is enough of a bar and no alternative should be tried even at small scale, fine. We should agree to disagree here then.
No, that’s not my belief and it frankly reads as a little petulant.
The risk I’m pointing out has nothing to do with a track record. It’s a design problem. Implementing solutions that are knowably bad is actually really stupid and we shouldn’t do it.
> Also what would be the bad outcomes here? If those drugs end up not being safe (that is prevented by the requirement to not withhold safety data), or being effective?
Barring a time machine, safety data reporting is gonna lag at least some of the bad outcomes.
But there are, obviously, proportionally way fewer bad outcomes created by drugs that pass Phases 2 and 3 than those that only pass Phase 1. The steepness of that attrition is literally the entire value prop of the law to begin with.
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