Press Release

Embargoed Until Contact

May 15, 2014

Sue Ducat
Director of Communications
(301) 841-9962


Breakthrough Therapy Designation


The latest Health Policy Brief from Health Affairs and the Robert Wood Johnson Foundation describes a new expedited drug development pathway designed to speed up the Food and Drug Administration’s (FDA’s) premarketing approval process for drugs and devices that treat serious or life-threatening conditions. Created under a 2012 law, the Food and Drug Administration Safety and Innovation Act (FDASIA), a drug may be designated a “breakthrough therapy” if it shows far more promise over comparable treatments already on the market. At that point, the FDA will initiate a special rapid approval process. The pharmaceutical industry has responded positively to this law, and as of last month the FDA has received 178 requests for this designation. The law, whose full impact will not be known for several years, carries significant implications for approaches to clinical development, patient access to new drugs, and the drug regulations process itself.


Topics covered in this brief include:


  • What’s the background, and what's in the law? As the brief explains, the FDA already has other processes in place to speed up the review of promising drugs and biologics. What makes this approach unique is that it circumvents the traditional standard clinical testing, because to be designated as a breakthrough therapy, the drug has already shown extraordinary results or is for a disease with no known therapy. As the brief explains, accelerated approval is given if the manufacturer agrees to conduct postmarketing trials to confirm the drug’s clinical benefit. The law evolved from a 2011 conference and white paper, which provided the framework for breakthrough therapy designation.  
  • What’s the debate? As the brief explains, this “all hands on deck” approach to the drug approval process is very resource-intensive, at an agency which has been underfunded for some time and will receive no additional funding to cover breakthrough-related activities. The brief also notes that some policy experts are concerned about the public health implications of the expedited approval process, because the risks of a particular drug may not be immediately clear. A final point of debate is the uncertainty of insurance coverage for drugs potentially costing as much as $300,000 per patient annually, or whether patients will gain access to them. 
  • What’s next? While the full impact of the breakthrough therapy designation will not be clear for some time, the FDA is now working to address some of the concerns raised about the process. One provision of the 2012 reauthorization of the Prescription Drug User Fee Act of 1992 requires the FDA to strengthen postmarketing safety surveillance, thereby, addressing some of the concerns previously expressed about this new law. It is likely that the FDASIA will continue to evolve for some time in conjunction with new scientific discoveries.

About Health Affairs

Health Affairs is the leading journal at the intersection of health, health care, and policy. Published by Project HOPE, the peer-reviewed journal appears each month in print, with additional Web First papers published periodically and health policy briefs published twice monthly at Read daily perspectives on Health Affairs Blog. Download weekly Narrative Matters podcasts on iTunes.