Pharma companies are basing pricing for these one-time-in-a-life-dose drugs on supply and demand principles. There will never be high demand for these drugs because the conditions are so rare. And only needing to be dosed once for a complete lifetime cure means that there is no recurrent payment happening the way you would have with a drug that needed to be dosed repeatedly over a lifetime.
You’ll hear all the usual excuses about “muh R&D costs 😭😭” but the truth is they’re pricing it this way because they can. Because somewhere in the bible of capitalism, this is the way things work.
(R&D costs are just an excuse for greed: https://www.treatmentactiongroup.org/resources/tagline/tagline-fall-2018/pharma-lies-people-die-myth-busting-fact-sheet-on-medicine-development-and-pricing/)
I just wanna say that I appreciate the hell out of you.