Breakthrough Therapy Designation

Abstract

Breakthrough therapy designation is a US Food and Drug Administration (FDA) program to expedite the development and review of urgently needed investigational products (including biologics) for serious or life-threatening conditions. To receive a breakthrough therapy designation grant, the preliminary clinical evidence must suggest substantial improvement over available therapy on a clinically significant endpoint. 1 Drugs so designated receive the following benefits: 2 • All Fast Track designation features • Intensive guidance on an efficient drug development program, beginning as early as Phase 1 • Organizational commitment involving senior FDA managers The breakthrough therapy designation has significantly shortened drug development time. A comparison of development time for recent oncology drugs reveals that the time from submission of an investigational new drug application (IND) to submission of a new drug application (NDA) or biologics license application (BLA) for non-breakthrough designated drugs is median 7.4 years, compared to only 5.2 years for breakthrough designated drugs. This is an average savings of over two years for breakthrough...