Earlier quoted context omitted.
Major pharma companies are all constantly competing, and very often are duplicating work because they are not sharing major experimental results. The way it feels is that there's already "too many people" working in certain areas (e.g. in cancer), while almost no attention is paid to these rare diseases. I think that more people studying rare diseases would result in a net gain of lives saved; I don't think it's as z…
On the other hand, competition has given us what, 6 covid vaccines of varying effectiveness, and in record time. What if only the least effective one was developed, and took 18 months? The 1962 FDA effectiveness mandates have had the side effect of increasing drug development costs enormously, and that shuts down development of treatments for rare disorders.
The Kefauver Harris Amendment you refer to was immensely important towards the development of safe and efficacious drugs -- I do not see the connection between that act and rare disease therapeutic development. In fact, drugs that only offer marginal improvements in quality of life for rare genetic disease patients are often fast-tracked by the FDA. Requiring that a drug _works_ shouldn't inhibit drug development. Otherwise, we end up with tragedies like what happened with the use of thalidomide, which prompted this amendment in the first place.