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Comment by yellow_lead | Hacker News Reader

> One of these groups (Group 1) received iloprost by vein (intravenously) for 6 hours daily for up to 8 days. The two other groups received other medications that are unapproved for frostbite, given with iloprost (Group 2) or without iloprost (Group 3).

> On day 7, the bone scan finding predictive of needing amputation was observed in 0% (0 of 16) patients receiving iloprost alone (Group 1) compared to 19% (3 of 16) patients in Group 2 and 60% (9 of 15) patients in Group 3. The presence of the bone scan abnormality was significantly lower in the two groups receiving iloprost. Most patients had follow-up information on whether they subsequently underwent at least one finger or toe amputation. The need for amputation was consistent with the bone scan findings.

It seems like an effective drug based on this result.

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Ah ! It would have sucked to have Frostbite not be in Group 1 and then need to undergo amputation. Talk about bad luck ! However, I totally understand that this is the main way for the science to progress and treatments to get validated.

The flip side is that it could have made things worse. Epistemology and ethics get complicated fast in this kind of thing:\

this is why PrEP has no double blind study. it was considered unethical

In the future this sort of thing will inevitably be viewed the way we view bloodletting with razors and leeches -- if not more harshly. They didn't know any better, way back when. We ought to know better.

It's as simple as this: Double-blind, placebo-controlled studies are absolutely not required in drugs that are have extremely large effect sizes. In these cases, open-label trials, compassionate use programs, and post-marketing surveillance can be employed to continue gathering data on safety and efficacy without denying any patients access to critical treatments. And, in the end, the data you gather is just as useful, if not more useful.

3/32 vs 9/15 is -probably- a pretty big effect size, but I am not sure how you know that a priori without trying it on a couple dozen people first and comparing. They didn’t require huge n for approval, but it took a long time to just try it on a minimal number of patients.

Does anyone on here know if this is due to the fact that it's a general vasodilator, or if there are specific pathways (eg: from activating prostacyclin and prostaglandin receptors) which have the effect of reducing tissue/bone damage?

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