Trends in pharmaceutical targeting of clinical indications: 1930–2013

•Indications targeted by NMEs have been dynamic, especially since the 1980s.•Infectious diseases are the most common indications but this recently plummeted.•Orphan diseases now account for more than one-third of all new indications.

An analysis of FDA-approved new molecular entities (NMEs) reveals trends in therapeutic applications. Four groupings (infectious diseases, cardiovascular diseases, autoimmune/inflammatory diseases and cancer) capture more than 60% of NMEs. Infectious diseases are the most targeted indications. Near the turn of the new millennium, the rate of new approvals for infectious diseases decreased. The absolute and relative number of NMEs targeting psychiatric, neurological and pain/itch indications also declined. By contrast, NMEs targeting cancer have risen in the past two decades as have NMEs targeting orphan indications. These results suggest the drug development community has largely been responsive to public health and market needs. However, finite resources might indicate emphasis on some unmet needs could come at the cost of others.