Comment by A_D_E_P_T

So as for #1: Payers were reportedly okay with paying for aducanumab, at something like $30k/year, which was approved by the FDA despite effectively zero efficacy in clinical trials.

And, besides, I think it stands to reason that if time-to-market and drug development costs are reduced, prices will inevitably drop, and there'll be more competition in the market.

Right now, small firms are effectively priced out of the pharmaceutical market. The typical business model is to develop something to preclinical/Phase I and pray for a buyer or "partner" -- a larger firm that specializes in regulatory compliance and has pockets deep enough for continued development.

As for #2:

What makes you think you need a placebo control? And have you considered that in certain otherwise-fatal diseases, a placebo control might be grossly unethical in and of itself?

There's no way to eliminate confounders, anyway. Even in clinical trials, patients aren't kept in bubbles. (Some of them might even drink grapefruit juice!)

The problem with "proving effectiveness" is that it's inordinately difficult (sometimes actually impossible) and the costs are borne by patients and society. "Golden Age" drugs like penicillin and corticosteroids, among many others, didn't need efficacy trials. Drugs don't really need them today. With enough data derived from patient populations, we can do much better -- faster, too, and more ethically.