As the year comes to a close, Patrick Frey, senior advisor to the director of the Office of New Drugs (OND) at the Center for Drug Evaluation and Research (CDER), looked back at FDA's performance over the final year of the fifth iteration of the Prescription Drug User Fee Act (PDUFA V) and what's to come in PDUFA VI.

Speaking at the FDA/CMS Summit in Washington, D.C. on Tuesday, Frey said that before PDUFA, and during the first four PDUFA programs, FDA was approving about 80% of all applications for new molecular entities (NMEs).

While that figure rose during PDUFA V to about 85%, the biggest difference from earlier programs according to Frey, is that FDA was able to reach decisions on more of those submissions earlier in the process.

Frey said that in part this was due to FDA's "focus on getting complete applications during PDUFA V, focusing on the fact that a complete action gets a complete review."

Where PDUFA V built in additional time for FDA to review NMEs and specified "touch points" during reviews for FDA and sponsor communications, Frey said that PDUFA VI gives FDA and sponsors more flexibility to tailor those communication.

"The program as written in PDUFA V was pretty prescriptive and held us to the letter for certain things, so there wasn't too much flexibility for us … so we added that in in PDUFA VI," he said.

For instance, in PDUFA VI sponsors and FDA can agree to a formal communication plan that features monthly teleconferences instead of the standard mid-cycle communication and late-cycle meeting.

Frey also said PDUFA VI formalizes some of the informal practices FDA adopted during PDUFA V for expedited reviews.

"Where we plan to act at least one month or more before the goal date, generally for priority review applications because there is a real public health need to get the product out there, we didn't want the parameters of the program slowing us down," Frey said.

CDER Performance and Workload in 2017

So far in 2017, CDER has approved 41 NMEs (29 new drug applications (NDAs) and 12 novel biologics license applications (BLAs)), nearly double the number it approved in 2016 (15 NDAs and 7 novel BLAs) but just shy of an all-time high in 2015.

While first cycle approvals fell in terms of percentage in FY2017 at 71%, that figure is still higher than in all years prior to PDUFA V.

Of those 40 NMEs, 17 (42%) were orphan drugs, following a trend observed over recent years.

According to Frey, CDER met its PDUFA goal date for all 40 products approved so far in 2017, 70% of which were given a priority review. CDER also approved a record number of drugs granted breakthrough therapy designations (17) and fast track designations (18) in a single year.

And Frey said that FDA set a record for the number of approved orphan indications in a single year, with 56 approvals across all NDAs, BLAs and efficacy supplements so far in 2017. The previous record was set in 2014, with 49 approved orphan indications.

As for CDER's workload in 2017 and beyond, Frey said the center saw an increase in the number of submissions in FY2017 compared the five year average over PDUFA V, with 48 NME NDAs and original BLAs, a 14% increase; 106 non-NME NDAs, a 28% increase; and 231 efficacy supplements, a 43% increase. Frey noted that by the end of June 2017, there were more than 7,000 active investigational new drug applications for drugs and biologics.

Frey also said that the US continues to lead the world in first time NME approvals, with about two-thirds of the NMEs being approved first in the US.

"We still see about two-thirds of the NMEs launched on the world market occur here in the US," he said, with the EU coming in at a distant second with about 15% of first launches.

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