alt Hacker News> It's too expensive to prove that a drug works
I'm not sure that is necessary a bad reason. You need to factor in a lot of concerns to determine what "too expensive" means.
But if you are going to spend billions of dollars to develop a drug that only treats about 2 people a year it is likely too expensive even if it is 100% effective. That money would be better spent on treatments that have wider applicability.
Of course this is not simple to measure. Costs aren't known upfront and the research may end up proving invaluable to more widely applicable treatments.
So it is a judgment call and not necessarily a bad reason.
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.