And with only 18 FDA decisions on new drugs expected in 2017, according to BioPharma Catalyst, the number of FDA approvals may continue to decline to a level the industry has not seen since 2007, when 18 new molecular entities (NMEs) and new biologic license applications (BLAs) were approved.
But as John Jenkins, Director of FDA’s Office of New Drugs, who retired from FDA on 7 January 2017, wrote on 04 JAN 2017 (and explained in November 2016), the lower number may not be a clear signal that industry innovation is stalling. Jenkins wrote, “…For example, CDER approved five novel drugs in 2015 that had PDUFA goal dates in 2016. These early approvals benefited patients by making the drugs available sooner, but also decreased the total of novel drugs approved in 2016. Another factor was the number of Complete Responses (CR), which describe deficiencies in the application, precluding approval, with advice on what the sponsor needs to do for FDA to support resubmission of the application. CDER (the FDA Center for Drug Evaluation and Research) issued 14 CR letters for novel drugs in 2016, higher than in recent years…”
Jenkins also notes that the primary deficiency in several of the applications was the failure to comply with FDA’s current Good Manufacturing Practice (cGMPs) regulation, whereas by comparison, only four (4) of the 47 new drug applications issued a CR from 2010 through 2015 included a failure to comply with cGMPs as the primary deficiency. “2016 may serve as a reminder to sponsors that all of their manufacturing facilities must be in compliance with cGMP regulations if they wish to ensure approval of their application. Failure of manufacturing facilities to pass FDA inspection can unnecessarily delay patient access to novel new drugs,” Jenkins added.
Of the new drugs approved in 2016, the majority (like in previous years) benefited from at least one of FDA’s programs to speed up a drug’s development or expedite a review.
- Eight of 22 approvals (36%) in 2016 benefitted from a fast track designation (meaning they had the potential to address unmet needs);
- seven (32%) were considered breakthrough therapies (meaning that preliminary clinical evidence demonstrated substantial improvement over other therapies);
- 15 (68%) received a priority review designation (meaning a six-month review period instead of the standard 10 months); and
- six (27%) received accelerated approval (meaning early approval for a serious or life-threatening illness with benefit over existing therapies).
Those numbers are similar to what occurred in 2015, when 14 of the 45 approvals (31%) came via the fast track designation, while 10 (22%) were so-called breakthrough therapies, 24 (53%) were priority review drugs and six of the drugs (13%) were approved under the accelerated approval program.
And for those still claiming that FDA does not approve drugs faster than other countries: 19 of the 22 novel drugs approved in 2016 (86%) were approved in the US before any other country, which is similar to what occurred in 2015, when 29 of 45 approvals (64%) occurred first in the US.
REFERENCE: RAPS Regulatory Focus; 04 JAN 2017; Zachary Brennan