Breakthrough Therapy Designation

Abstract

Background:

A breakthrough therapy designation is intended to facilitate and expedite development and review of new drugs to address unmet medical need in the treatment of a serious condition.

Methods:

The Center for Drug Evaluation and Research (CDER) at Food and Drug Administration (FDA) analyzed 364 breakthrough therapy designation requests received from program inception on July 9, 2012, through June 30, 2016.

Results:

Of the 364 requests received during this time, CDER granted 133 (37%), denied 182 (50%), and the sponsor withdrew 49 (13%) before CDER made a decision.

Conclusion:

This analysis provides information on the distinguishing characteristics of the drugs seeking this designation and the decisional factors used by CDER to either grant or deny breakthrough therapy designation requests. This paper provides greater transparency into the CDER decision process, so the public can better understand how breakthrough therapy designations are determined.

Keywords

FDA expedited programs breakthrough therapy

Get full access to this article

View all access options for this article.

References

US Congress. Food and Drug Administration Safety and Innovation Act. 2012.

US Food and Drug Administration, Center for Biologics Evaluation and Research. Guidance for industry: expedited programs for serious conditions—drugs and biologics. Food and Drug Administration. Web site. https://www.fda.gov/downloads/Drugs/Guidances/UCM358301.pdf. Published 2014. Accessed November, 2016.

FDA. Guidance for industry and review staff: formal dispute resolution: appeals above the division level, 2015. Web site. http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm343101.pdf.

Orphan drugs. 21 CFR Part 316 2011.

US Food and Drug Administration, Center for Biologics Evaluation and Research, Center for Devices and Radiological Health. Guidance for industry: pharmacogenomic data submissions. Food and Drug Administration. Web site. https://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm079849.pdf. Published 2005. Accessed November, 2016.

Johnson

Ning

Farrell

Justice

Keegan

Pazdur

. Accelerated approval of oncology products: the Food and Drug Administration experience. J Natl Cancer Inst. 2011;103:636–644.

Concato

Shah

Horwitz

. Randomized, controlled trials, observational studies, and the hierarchy of research designs. N Engl J Med. 2000;342:1887–1892.

Woodcock

. Drug development in serious diseases: the new “breakthrough therapy” designation. Clin Pharmacol Ther. 2014;95:483–485.

FDA. Breakthrough therapy approvals. Web site. http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/DrugandBiologicApprovalReports/NDAandBLAApprovalReports/ucm373418.htm. Published 2016.