The FDA has approved 35 novel new drugs so far this year, up from 27 products last year, with officials touting 2014 as the best year ever for rare disease treatment approvals.

The figure includes most of the 35 new molecular entity applications for NDAs and BLAs submitted so far this year to CDER. However, CDER admitted that the bar for approval under the breakthrough therapy program remains confusing.

Of the total approvals, 15 were for rare diseases, the most in any year. The figure surpasses the previous record of 13 rare disease approvals in 2012, said Commissioner Margaret Hamburg.

She also touted that CDER met its PDUFA deadlines for all but one of the approved products.
CDER also approved 75 percent of its products on the first review cycle.

The use of expedited development tools, such as fast track, priority review, accelerated approval and breakthrough therapy, played an increasing role in the approvals. More than half (57 percent) of the new drugs this year were cleared through one of those pathways.

However, the numbers did show a recurring problem with industry confusion over the breakthrough therapy designation. Since the pathway was created in 2012, the agency has received 211 applications, but designated only 63 breakthrough therapy products. Of those products, CDER has approved 13 and CBER only one.

The problem is the criteria for approval under this pathway remains subjective, requiring judgment by the FDA, according to John Jenkins, director of CDER’s Office of New Drugs, in a presentation on the 2014 results. Another problem is the number of requests exceeded expectations, and the FDA didn’t receive new resources to handle the pathway when it was approved as part of the 2012 FDA Safety and Innovation Act.

FDA and Brookings Institute will hold a public workshop next April on the breakthrough therapy designation process.

Despite the high rate of denials, Jenkins said the pace of requests for breakthrough therapy designations remain steady.

To read CDER’s presentation on 2014 approvals, visit www.fdanews.com/12-11-14-2014Approvals.pdf. — Robert King

Originally appeared in Drug Industry Daily, the pharmaceutical industry’s number one source for regulatory news and information. Click here for more information.