I hope one-day things would turn around. If the regulators asked pharmaceutical companies to study for these drugs on the side so much could change. Probably less than 1% of their R&D budget would be enough to move things.
Absolutely. A lot of funding gets poured into dead end alzheimers research or similar that will never work. A fraction of that redirected to rare diseases that get zero funding will do wonders. It is a shame that academia and research are so burdened with graft and politics.