Developing Standards for Breakthrough Therapy Designation in Oncology

• Published in: Clinical cancer research Vol. 19; no. 16; pp. 4297 - 4304
• Main Authors: HORNING, Sandra J, HABER, Daniel A, SELIG, Wendy K. D, IVY, S. Percy, ROBERTS, Samantha A, ALLEN, Jeff D, SIGAL, Ellen V, SAWYERS, Charles L
• Format: Journal Article
• Language: English
• Published: Philadelphia, PA American Association for Cancer Research 15.08.2013
• Subjects: Antineoplastic agents; Biological and medical sciences; Humans; Medical Oncology - standards; Medical sciences; Multiple tumors. Solid tumors. Tumors in childhood (general aspects); Neoplasms - therapy; Pharmacology. Drug treatments; Therapeutics - methods; Therapeutics - standards; Tumors; United States; United States Food and Drug Administration; United States; Cancerology; Treatment; Malignant tumor; Standards; Cancer
• ISSN: 1078-0432; 1557-3265
• DOI: 10.1158/1078-0432.ccr-13-0523

In July 2012, Congress passed the Food and Drug Administration Safety and Innovation Act (FDASIA). The Advancing Breakthrough Therapies for Patients Act was incorporated into a Title of FDASIA to expedite clinical development of new, potential "breakthrough" drugs or treatments that show dramatic responses in early-phase studies. Using this regulatory pathway, once a promising new drug candidate is designated as a "Breakthrough Therapy", the U.S. Food and Drug Administration (FDA) and sponsor would collaborate to determine the best path forward to abbreviate the traditional three-phase approach to drug development. The breakthrough legislation requires that an FDA guidance be drafted that details specific requirements of the bill to aid FDA in implementing requirements of the Act. In this article, we have proposed criteria to define a product as a Breakthrough Therapy, and discussed critical components of the development process that would require flexibility in order to enable expedited development of a Breakthrough Therapy.