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Regulatory Focus™ > News Articles > FDA Approvals Strong in 2013, but Far From Highs of 2012

FDA Approvals Strong in 2013, but Far From Highs of 2012

Posted 16 January 2014 | By Alexander Gaffney, RAC

A new report published by the Center for Drug Evaluation and Research (CDER) shows that while approvals of new molecular entities (NMEs) dipped in 2013 relative to 2012, trends appeared to have held steady compared to the turbulence and uncertainties of the last decade.

The report, Novel New Drugs: 2013 Summary, observes that FDA approved 27 new drugs in 2013, down from 39 in 2012 and about even with the 30 approved in 2011. The 2013 total is also higher than the number of approvals in any year between 2005 (20) and 2010 (21).

But FDA can only approve a NME-essentially a drug that has never before been approved for an indication-for which it has received applications. When assessed as an approval percentage of applications received, 2013 was a decidedly more average year for drug approvals.

Consider the following chart:


Applications Received

Applications Approved

Approval Percentage





































By this analysis, 2013 was the fourth-best year for drug approvals by percentage since 2005, and was almost identical to 2008 and 2009 when it received and approved nearly the same number of NMEs.

Other Achievements

But buried within those numbers are other notable achievements. For example, a third (33%) of all NMEs approved in 2013 were for orphan or rare disease indications, defined as diseases affecting fewer than 200,000 patients in the US.

"This is significant because patients with rare diseases often have few or no drug treatment options," the report explains. Out of all drugs approved, a third were also first-in-class drugs, meaning they exhibited new and unique mechanisms of action that had never before been tried against any disease or treatment indication. To FDA, this was a strong indication that the drug applications it is receiving are highly innovative. Comparable rates for prior years were not available.

FDA also used many of its expedited review pathways to review drugs this year. Ten drugs (37%) were reviewed using the fast track designation, three (11%) were given breakthrough product designation, 10 (37%) were given priority review designation, and two (7%) were given accelerated approvals. Thirteen drugs were given more than one designation, FDA said.

"Each of these designations helps expedite the speed of the development and/or approval process and is designed to help bring important medications to the market as quickly as possible," the report explained.

Predictable Approvals

But just as eventual approval of a drug is important to its sponsor, so too is the predictability of the timing of that approval. In this regard, FDA said it was especially successful, as it managed to approve all 27 drugs under their agreed-upon Prescription Drug User Fee Act (PDUFA) goal dates in 2013. Twenty-four of the 27 drugs were also approved during their first review cycle, meaning no complete response letters (CRLs) were issued. Those letters typically request more data or raise significant questions about the quality of an application, and can sometimes take years to address.

By another measure, FDA also had a rather successful year: First-in-world approvals. Seventy-four percent of NMEs approved in 2013 were approved in the US before anywhere else in the world-a point of pride for US regulators.

"The report speaks volumes on the work that you all do directly or indirectly to support our goal to ensure that our reviews remain efficient and of high-quality," said John Jenkins, director of CDER's Office of New Drugs (OND), in a letter to FDA staff. "It also reflects CDER's willingness to exercise regulatory flexibility and creative approaches to help industry meet our standards-without lowering them-so that we may continue to provide medicines to patients in need."

That last point-that it's not all about speed, but rather safety and efficacy-was emphasized in the report as well. "In all cases, while striving for efficiency of review and approval of applications for new drugs, CDER does not compromise its standards for demonstration of effectiveness and safety in the process," FDA concluded.

Novel New Drugs: 2013 Summary

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