Targeting rare diseases
Is working on genetic mutations that affect only a few in every thousand people risky?
There are a number of ways to set up a drug company’s portfolio. At the broadest level, generic companies stay in business by selling lots of low-margin drugs in a competitive marketplace (well, that’s how it’s supposed to work, anyway). The research-driven companies accept far lower chances of success in the hope that they can charge proportionately more for their patented compounds – at least until that patent expires or is overturned. But different firms target different diseases, and with different kinds of drugs.