> there's just about 200k people diagnosed with some form of hemophilia of which only a fraction has the funds or the insurance to obtain that kind of treatment in the first place (both the current and the new one)
This was actually the crux of the discussion where I learned about the $50k figure. There are a lot of hemophiliacs in countries that spend, on average, just a few hundred dollars per capita each year on health care. Even if Hemgenix went royalty-free right away and no one ever turned a dollar of profit on it, gene-therapy cures are still inaccessible to the bulk of people the World Federation of Hemophilia represents.
$3.5 million would be like landing on the moon, and $0.05 million would be like a few orbits at ISS height, but they're both astronomical. They're equally out of reach for most people with severe hemophilia.
That doesn't account for the R&D effort which has to be recouped during the runtime of the patent. Depending on whom you ask, that can reach into billions of dollars [1], and there's just about 200k people diagnosed with some form of hemophilia of which only a fraction has the funds or the insurance to obtain that kind of treatment in the first place (both the current and the new one), so these few patients have to account for the R&D cost of the medication, the R&D cost of failed candidate substances, the R&D for ongoing other medication and profits. For "orphan diseases", these economies are a serious problem.
Pharmaceutical development is incredibly expensive; to make it worse a lot of governments have cut back drastically on fundamental R&D grants for universities and so private companies with their profit interests stepped in.
[1] https://msf-access.medium.com/how-much-do-clinical-trials-co...
[2] https://www.pfizer.com/disease-and-conditions/hemophilia