36 new orphan drugs approved by FDA in 2014

According to the website RDR Rare Disease Report, “a remarkable 36 orphan drugs were approved for new indications by the U.S. Food and Drug Administration (FDA) in 2014.” The indications addressed by the new therapies include (among others) rare cancers as well as gaucher disease and idiopathic pulmonary fibrosis. This development shows that rare diseases are gaining importance, also for the pharmaceutical industry. The need of innovative statistical methods for these trials is thus immanent.