Why drug companies say no

7:35 AM ET Tue, 5 Aug 2014

Disappointed patients often paint pharmaceutical companies as evil, heartless corporations when they turn down compassionate use requests. But drug makers are in a tough spot. They need to balance the long-term goal of getting therapies to market for the broader population against helping one patient. Issues include supply, the possibility of a bad outcome influencing the long-term approval outlook and the risk of patients defaulting to compassionate use requests over participating in a clinical trial, where they may get a placebo. There are also ethical dilemmas in saying yes to one patient while saying no to the next. One Biogen exec calls the process "agonizing."