Breakthrough Therapy Designation: CDER Analysis of Requests 4 Years Into the Program

Therapeutic Innovation & Regulatory Science
Ryan ConradAmy Bertha

Abstract

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. 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. 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. 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.

References

Jun 22, 2000·The New England Journal of Medicine·J ConcatoR I Horwitz
Jul 12, 2005·Regulatory Toxicology and Pharmacology : RTP·Steven D Brynes
Mar 23, 2011·Journal of the National Cancer Institute·John R JohnsonRichard Pazdur
Apr 22, 2014·Clinical Pharmacology and Therapeutics·J Woodcock

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Citations

Oct 11, 2019·Therapeutic Innovation & Regulatory Science·Cameron M KiefferAndrew S Robertson

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Software Mentioned

CDER

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