A Matter of Life and Death

The mission of the FDA is to protect human health. To fulfill that mission in the realm of pharmaceutical research and development, it is typically quite important to have placebo-controlled trials. Such trials, in which some patients blindly receive false, inactive treatments in place of the experimental drug, are the gold standard in demonstrating the effectiveness and safety of a given therapy, and they’re ideal for nonfatal diseases that affect large patient populations.

Rare diseases — defined as those that affect fewer than 200,000 patients in the United States — are different. A significant number of rare disorders are considered ultrarare and affect fewer than 1,000 patients, typically children, and are fatal. Most of these illnesses you have probably never heard of like Niemann-Pick Type C (NPC) disease. In studies of treatments for these diseases, the placebo control is an almost impossible standard. And yet it holds.