4 ms·

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. A

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.

ceejayoz 18d ago
> 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.
- niklas_anzinger 18d ago
  That is the same with all post-market monitoring for drugs. And maybe we can do better because more nimble/startup approach in a small state
  - ceejayoz 18d ago
    Isn't this pre-market, though?
    - niklas_anzinger 18d ago
      Relative to the federal approval it's pre-market. In Montana, if your treatment is approved by a review board it's in market
  - estearum 17d ago
    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.
estearum 17d ago
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.
- niklas_anzinger 15d ago
  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.
  - estearum 14d ago
    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.

ceejayoz 18d ago

> 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.

niklas_anzinger 18d ago
That is the same with all post-market monitoring for drugs. And maybe we can do better because more nimble/startup approach in a small state
- ceejayoz 18d ago
  Isn't this pre-market, though?
  - niklas_anzinger 18d ago
    Relative to the federal approval it's pre-market. In Montana, if your treatment is approved by a review board it's in market
- estearum 17d ago
  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.

estearum 17d ago

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.

niklas_anzinger 15d ago
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.
- estearum 14d ago
  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.