Yes, you can. The problem with testing is that a candidate treatment may make things worse for patients, not that it may improve things.

If your treatment works, that’s an improvement of what you had before. Once you know that, you can treat all patients. For some, that will be too late, but without your tests, it would be too late for them, too.

If your treatment doesn’t do anything at all, it keeps things the same, but the patients in the test group likely will have had some inconveniences (having to visit a doctor, getting an injection, etc), so you shouldn’t do the test.

If your treatment makes things worse, you of course shouldn’t do the test.

Problem is that you typically only can only know in hindsight which of these applies.

So, you think carefully on whether a treatment could fall in category 3, and, if so, first do it on a group of patients who consent to be Guinea pigs and, often, are already terminally ill, as any negative outcomes will cause less harm to such patients.

Then, as soon as during the test your stats tell the drug does or doesn’t work, you stop the test and either treat all patients or stop treating the test subjects.

When there is already a known treatment, the control group gets the old known treatment and the other group get the new proposed treatment.

So the comparison is between old and new, to ensure the new treatment is better than the current standard one.

I responded below already but since writing that, I've come up with a few more great bad reasons not to bring a drug to market (sorry for the parseability of that one...)

Best bad reason: it would cannibalize an inferior drug currently in your portfolio that's still under patent protection.

I think this behavior is at the far "evil" end of the spectrum of behaviors that drug developers systematically engage in (which I believe is far more banal + less evil than what they're accused of), but it does happen and it's a really nasty

Where it gets especially nasty is when companies buy drugs in development or pre-development from other companies in order to squash (or at least delay) a potentially competitive asset before it reaches the market.

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Another great bad reason, but mostly applies to devices/procedures: the device/procedure is fantastic but for various structural reasons outside of the control of the device/procedure developer, there is insufficient incentive for healthcare providers to actually deploy said device/procedure.

A trivial example would be a pacemaker that requires fewer leads than the competitors and has fewer complications. Great for patients, but potentially totally uninteresting to the electrophysiologists who install it and would get paid less due to the less complex procedure.

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Best good reasons all fall under: making a therapy is unfathomably difficult and most efforts are destined to fail OR (separately) proving a therapy works is unfathomably expensive and most efforts can't produce a positive ROI after that process.

Ultimately most of these decisions come down to economics and not mustache twirling villains. The banality of evil.

Simplest "bad" reasons are the wealth of the patients. Malaria, river blindness, guinea worm, etc are terrible diseases that mostly impact poor people out of sight from Western eyes. Spending $X billion developing a drug for a population that can barely afford to feed themselves is not going to make a financial return on investment.

Agreed it isn't necessarily a bad reason. In some cases it's a good reason for failure (like the one you describe).

In other cases it's a bad reason for failure: it's also incredibly expensive to prove your drug works even if it does work for a lot of people.

That's bad! It'd be better if it were cheaper.

Actually counterintuitively, due to a weird drug approval and payor reimbursement policy arbitrage, pharma companies are highly incentivized to produce drugs for tiny populations.

One of my hobby horses is railing against this specific dynamic.

About Doxepin. As many seniors do, I also suffer from extreme inability to stay asleep at night. I have trialed through all the known prescription and non prescription possibilities, only eszopiclone and baclofen seem to show some promise, however, eszopiclone is DEA listed, requires higher and higher doses, and if I take it more than say 2 weeks, it has rather serious side effects attempting to withdraw, addictive, serious anxiety, trying to wean oneself off it. Doxepin is prescribed as an antidepressant in large doses, one of the most potent H1 histamine antagonists known. The H1 system in our bodies promotes wakefulness. In very low doses, doxepin acts against the H1 to promote sleep. To avoid the upcharges of low dose doxepin, I am prescribed the high dose version, which I have to break the capsules to administer about 5 to 10 mg. placed in an empty gelatin capsule (it's bitter). It really works well, however you are fairly tired and useless the next day.

Obviously you use the neighboring country’s population, or an ethnic minority, or prisoners, or orphans, or…

These folks gave an interesting talk on producing pharmaceuticals at defcon a couple years ago.

IIRC it was more about production methods than developing new treatments.

https://fourthievesvinegar.org/

This made me think of the "Institute for one world health" . It came out as a non-profit pharmaceutical company in the mid 2000's. Victoria Hale was the founder-it got her a MacArthur fellowship. It is focuses(focused?) on global health and populations underserved by for-profit models. I think they successfully developed a treatment for leishmaniasis. it's an adorable model and should be pushed but as usual it seems like the philantropy money is limiting.