New Medicines for Serious Diseases: The Case of the FDA’s Breakthrough Designation

Abstract

In the late 2000s, early-stage clinical trials for personalized drugs unveiled large beneficial treatment effects. These findings created an unexpected regulatory challenge: by removing genuine clinical uncertainty on the best treatment alternative, they made randomized controlled Phase 3 trials ethically contestable. This paper studies how the program enacted in response to this challenge — the FDA's Breakthrough Therapy designation---has shaped pharmaceutical R&D. Our results suggest that the program's 2012 creation ignited a large amount of new drug R&D targeting serious and life-threatening conditions. According to the program's history, these impacts may not have been fully premeditated. Measured by medium-term anticipated drug approvals, these indirect program impacts could be as large direct ones.