Therapeutic Value Assessments of Novel Medicines in the US and Europe, 2018-2019

Among 67 novel drugs approved by both the FDA and EMA in 2018-2019, only 32% were assessed as having high added therapeutic value by German and French health technology assessment agencies, with none of the 8 accelerated approval drugs achieving a high rating. These findings are consistent with earlier research and support the need for greater scrutiny of which drugs qualify for expedited regulatory programs and rare disease designations, given that so few of these products demonstrate substantial clinical improvement over existing therapies.