Drug Breakthroughs, with Some Reservations

Published on: 
BioPharm International, BioPharm International-01-01-2015, Volume 28, Issue 1

New designations lead to faster drug approvals, but there is more work to be done.


As 2014 drew to a close, FDA reported a number of milestones in drug approvals. Through early December, the agency had approved 35 new molecular and biologic entities, up from 27 in 2013; this total was the second highest number of approvals in a single year in the past decade. Biologics, with 10 approvals, represented nearly one-third of all approvals (1). 

Patients with rare diseases benefited from a record number of novel drugs entering the market in 2014. The 15 drugs approved were the most in a single year since the Orphan Drug Act was enacted in 1983. 

FDA Commissioner Margaret Hamburg noted in the FDA blog (2) that 15 of the approvals were first-in-class drugs, an indicator of their potentially strong clinical impact.  

Hamburg noted that three new approvals were for antibacterial drugs--Dalvance (Durata Therapeutics), Sivextro (Cubist Pharmaceuticals), and Orbactiv (The Medicines Company)--to treat skin infections, specifically acute bacterial skin and skin structure infections. From 2004 to 2013, only five new systemic antibacterial drugs were approved, FDA reports. 

FDA also issued a report card of its own performance. Hamburg cited FDA’s expedited development and review programs--fast track, priority review, accelerated approval, and breakthrough therapy designation--as being instrumental in moving drugs more quickly to market. More than half of the drugs (57%) were approved under priority review; 37% received fast-track designation. “Early and repeated communications with sponsors have also been helpful in speeding these products to market,” she wrote in the blog. 

Hamburg noted that 34 of the 35 drugs approved as of Dec. 3, 2014 were approved before or on their Prescription Drug User Fee Act review goal date; 23 of the 35 drugs were available to patients in the United States before they were available to patients in Europe. Another indication of performance improvements, noted John Jenkins, director of the Office of New Drugs (OND), Center for Drug Evaluation and Research (CDER), is that more submissions are moving through the review process in just one review cycle (1).  


The review and approval process still has room for improvement. The pace of requests for breakthrough designation have remained steady; however, of the 211 breakthrough therapy requests, 49% were denied; 6% were pending; and 15% were withdrawn. FDA plans to issue further guidance to clarify subjective statutory criteria and basic standards needed for breakthrough requests.  

Jenkins said that manufacturing development and scale up must be accelerated, and noted that several approvals, while expedited or on time, were delayed due to the need to address manufacturing issues. 

New Year, New OPQ
CDER’s Office of Pharmaceutical Quality (OPQ) goes live in January 2015, as a single unit dedicated to product quality. FDA expects the OPQ to better align all drug quality functions at CDER, including review, inspection, and research for domestic and foreign drug manufacturing sites.  This “super-office” will bring all non-enforcement-related drug quality work into one office, creating “one quality voice and improving our oversight of quality throughout the lifecycle of a drug product,” as described on www.fda.gov. Janet Woodcock, director of CDER, will serve as acting director.  

1.  J. Jenkins, CDER New Drug Review: 2014 Update, presented at FDS/CMS Summit (Washington, DC, Dec. 11, 2014).

2.  Margaret A. Hamburg, 2014 Drug Approvals: Speeding Novel Drugs to the Patients Who Need Them, http://blogs.fda.gov, accessed Dec. 22, 2014.