Growth in orphan drug development presents operational hurdles

A recently completed analysis from the Tufts Center for the Study of Drug Development (CSDD) finds that rare disease drugs now account for nearly one-third of all therapies in active development.

Orphan drugs are defined in the USA as those which treat a medical condition affecting 200,000 or fewer people. In Europe, the standard is fewer than five people per 10,000.

The authors find that the share of new drug approvals represented by orphan drugs has doubled, from 29% in 2010 to 58% in 2018.