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by vikramkr 2301 days ago
It's also lead to unprecedented investment and development of drugs for rare diseases. Some 45% of drugs approved last year are for rare diseases.

The key with those is that high pricing let's those rare disease drugs be blockbusters - you can still make enough money in a disease with only a few thousand patients in the US if you're able to charge whatever you want.

We've seen less interest in drugs that can't be blockbusters because the costs have gone up, yes, but we also haven't had another thalidomide yet

2 comments

Avoiding another thalidomide is not the only objective in drug discovery. Arguably the pace of progress[1] in medicine has been made slower because of the extreme cost of regulation. How many thousands are dying because they will be treated by drugs that could have been but are not because of regulation. I don't know, and I doubt anyone does. Ultimately this is problem with a regulatory body who's incentives are tied (or more tied) to only one of two competing bad outcomes. Its really easy to never have another thalidomide, but we should aim higher than that.

[1] One example of this is dietary treatments for disorders. It is very difficult for these to become standard of practice, because no company will want to fork over the $$$ to do a clinical trial to prove that a specific dietary intervention improves outcomes. When after its all done people can just do the intervention on their own and not pay the company money. This biases treatment towards pills that can be prescribed and patented.

Earlier I said think of the thousands of lost lives, but it might truthfully be millions considering the damage caused by type II diabetes which pharmacological interventions are relatively impotent to treat.

> we also haven't had another thalidomide yet

I recall approval for some diet drugs was removed because they were causing fatal heart attacks. I recall recently some cholesterol drugs were causing unanticipated problems, too. Tylenol is now known for causing liver problems, even failure.