A Review of CDER’s Novel Drug Approvals for 2016

By: John Jenkins, M.D.

This past year was another successful year for the new drugs program in FDA’s Center for Drug Evaluation and Research (CDER).

John JenkinsCDER reviewed and approved 22 novel drugs, most of which have the potential to add significant clinical value to the care of thousands of patients with serious and life-threatening diseases.

Among the novel drugs approved in 2016 were the first treatment for patients with spinal muscular atrophy, the first drug approved to treat Duchenne muscular dystrophy, a new drug to treat hallucinations and delusions in people with Parkinson’s disease, another to treat patients with a rare chronic liver disease known as primary biliary cirrhosis, and two new treatments for patients with hepatitis C. There were also new oncology drugs to treat patients with ovarian cancer, bladder cancer, soft tissue sarcoma, and chronic lymphocytic leukemia — as well as two new diagnostic agents for detecting certain forms of cancer.

Nearly three out of four of these novel products – or 73 percent – benefitted from at least one of FDA’s programs to expedite drug development and review (i.e., Fast Track designation, Breakthrough Therapy designation, priority review designation, accelerated approval).

CDER’s review team also met the goal dates specified by the Prescription Drug User Fee Act (PDUFA) for 95 percent of 2016’s novel drug approvals. We also approved 95 percent of the novel products on the “first cycle,” meaning additional information was not requested that would delay approval and lead to another review cycle. Moreover, 86 percent of the novel drug approvals were approved in the U.S. before they were approved by any other regulatory authorities. The upshot of these efficiencies is that CDER is reviewing drugs as quickly as possible while continuing to uphold FDA’s traditionally high approval standards.

The total number of novel drugs approved in 2016 is lower than the 45 novel drugs approved the year before and below the average of 29 drug approvals per year on average over the last 10 years. There are several reasons for this. While the number of novel new drug applications received for review in 2015 was similar to our most recent 10-year average of 35 applications per year, the natural fluctuation of the timing of application submissions and their PDUFA goal dates, meant there was a smaller pool of novel drug applications to target action on than in recent years. 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 issued 14 CR letters for novel drugs in 2016, higher than in recent years.

Each application for a new drug must meet the statutory and regulatory standards for approval by demonstrating that the new drug is safe and effective for its intended use, and that the quality of the manufacturing of the product is high, before it can be approved. FDA reviews each application on its own merits. While we report on groupings of applications submitted and approved each year, given the expected variation in the quality of the data contained in any particular application it is not surprising that the ratio of approvals to CR letters tends to fluctuate from year-to-year. In examining the deficiencies cited in the CR letters issued to novel drugs in 2016 it is notable that the primary deficiency for several of the applications was failure to comply with FDA’s current Good Manufacturing Practice (cGMPs) regulations. These regulations are designed to control the quality of manufacturing procedures for drugs in order to ensure that patients receive drug products of a consistently high quality, which is an important component of the safety and efficacy of a drug. The number of CR letters that cited failure to comply with cGMP regulations was unusually high for a single year. By comparison, only four of the 47 novel drug applications for which a CR was issued from 2010 through 2015, included 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.

Our annual Novel Drugs summary provides more details about CDER’s novel drug approvals for calendar year 2016.

On a personal note, I will retire from FDA on January 7, 2017, after nearly 25 years of service at FDA and nearly 32 years of service in the Federal government. It has been a great honor and privilege to serve with the dedicated public servants at FDA whose hard work, under often challenging circumstances, serves to promote and protect the public health of Americans and patients around the world. During my time at FDA we have seen the erasure of the “drug lag” of the 1980’s where drugs were approved in other countries years before they were approved in the United States. Today more than two-thirds of novel drugs are approved first by the FDA, providing patients in the U.S. with earlier access to new drugs that have the potential to significantly improve their quality of life, and in some cases to extend their lives. This remarkable change has been accomplished without compromising FDA’s standards for approval; in fact, we have also significantly strengthened and modernized our pre-market and post-market drug safety programs at the same time as we dramatically improved the efficiency of our new drugs review program. I am proud to have been a part of helping to make FDA the “gold standard” for drug regulatory agencies around the world. While I am leaving FDA, FDA will not leave me; its principles and high standards will help to guide me in my future work.

There are many new challenges and exciting opportunities for CDER in 2017 and beyond; I am confident that the highly professional and dedicated staff in the new drugs program will meet those challenges and have the experience and vision to translate the exciting and rapid advances in science into new safe and effective treatments for patients in need.

John Jenkins, M.D., is Director of the Office of New Drugs in FDA’s Center for Drug Evaluation and Research

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