CDER Approved Many Innovative Drugs in 2014

By: John Jenkins, M.D.

Each year, FDA’s Center for Drug Evaluation and Research (CDER) will typically approve more than 100 new medications. A portion of those are novel new drugs, medications that have not previously been approved by FDA and are often among the most innovative products serving previously unmet medical needs or otherwise significantly helping to advance patient care and public health.

John JenkinsThis year, the news media has been concentrating on the number of novel new drugs – either new molecular entities or new therapeutic biologics – approved by CDER in 2014. And that’s understandable because we approved 41 novel drugs this year, the most in nearly 20 years. But instead of looking at the approval tally, we prefer to focus on the significant benefits that many of these drugs bring to patients and the steps that CDER took to get these products to market in a timely manner while maintaining FDA’s standards for safety, effectiveness, and quality.

Many of the 41 new drugs have the potential to add significant clinical value to the care of thousands of patients with serious or life-threatening diseases. They include eight new drugs for treating patients with various types of cancer, four new drugs to treat type-2 diabetes, four new antibiotics to treat serious infections, and two new products to treat patients with hepatitis C.

Moreover, consider these facts:

  • Seventeen (41%) of the 41 novel new drugs were approved to treat rare diseases that affect 200,000 or fewer Americans. This is the highest yearly total of such drugs ever — surpassing the previous high of 13 from 2012. These approvals are particularly significant because patients with rare diseases often have few or no drugs available to treat their conditions.
  • Seventeen (41%) of the 41 novel new drugs are identified by CDER as “First-in-Class,” one indicator of a drug’s degree of innovation. The total for First-In-Class approvals in 2014 approaches the highest yearly total of 20 reported in 2012.

To expedite the development and review of these products, CDER used a number of regulatory programs, including Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval.

  • Fast Track and Breakthrough Therapy designations are designed to speed the development of promising new drugs intended to treat serious conditions with unmet medical needs. Almost half – 19 or 46% of the 41 novel new drugs approved in 2014 — were designated as Fast Track, Breakthrough, or both.
  • Twenty-five (61%) of the 41 novel new drugs were designated for Priority Review. These are drugs in which CDER sees potential for providing a significant advance in medical care, and sets their review target to within six instead of the standard 10 months.
  • Six (20%) of the 41 novel new drugs were approved under FDA’s Accelerated Approval program, which allows early approval of a drug for a serious or life-threatening illness that offers a benefit over current treatments. Accelerated Approval is based on a “surrogate endpoint” or an intermediate clinical endpoint that is thought to be “reasonably likely to predict clinical benefit.” Additional clinical trials are required after approval to confirm the predicted clinical benefit. A surrogate endpoint is a marker of drug effect (e.g., an effect on a lab value or tumor size) that does not directly represent an improvement in how a patient feels or functions, but is expected to predict such a benefit.

Here are a few other ways we assess our contributions to last year’s approvals:

  • Under the Prescription Drug User Fee Act (PDUFA), sponsors pay fees when they submit a product application. This money is used to provide FDA with additional resources to meet performance goals, such as a goal date for completing its review of the application. In 2014, CDER acted on or before the PDUFA goal date for 40 (98%) of the 41 novel new drugs approved.
  • CDER approved more than three-quarters — 32 (78%) — of the 41 novel new drugs on the “first cycle” of review, meaning without requests for additional information that would delay approval and lead to another cycle of review.
  • Nearly two-thirds of the novel new drugs – 26 (63%) — were approved in the U.S. before approval in another country.

It’s been another strong year for approval of novel new drugs for patients in need. We are proud of our role in helping to safely and efficiently bring important new medications to the American public.

Our Novel New Drug Summary for 2014 provides more details. A current list of CDER’s 2014 novel new drug approvals is available on our Web site.

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

FDA: Reflection on 2011 and Looking Forward to 2012

By: Margaret A. Hamburg, M.D.

The beginning of a New Year is a wonderful time to reflect on the past year and to think about what lies ahead. While 2011 had its share of challenges for us at FDA, it was also a time of significant accomplishment. We are excited about the many opportunities that 2012 will bring to the Agency.

2011 began with the most sweeping reform of our food laws in more than 70 years. FSMA promises an even safer food supply than we enjoy today and a wonderful opportunity to help promote public health.  We also continued the hard work of implementing the Family Smoking Prevention and Tobacco Control Act.

Margaret Hamburg On the medical product front, there were a multitude of outstanding activities and advances.  Importantly, we renewed our focus on the importance of innovation, in our own work and in the products we are tasked to review.  This was also a remarkable year for innovative drug approvals.  During Fiscal Year 2011, the FDA approved 35 novel drugs, targeting diseases such as late-stage lung cancer, metastatic breast cancer, and hepatitis C.

We also worked to continually strengthen the science that guides our policies, including many important collaborations with key partners and stakeholders.  Even with all of our ongoing work, when crises came in 2011, we acted swiftly to protect the American people.  We also continued to strengthen our capacity to address one of the greatest challenges in this new century – our increasingly globalized world.  “Pathway to Global Product Safety and Quality”

In 2012, FDA will meet new and complex challenges and continue our work in implementing FSMA and the Tobacco Act, promoting advancements and innovation in medical products, and broadening our strategic globalization and regulatory science initiatives. We will be working closely with Congress and stakeholders to support the reauthorization of user fees for pharmaceutical products (PDUFA), and medical devices (MDUFA), as well as to authorize new user fees for generic drugs (GDUFA) and biosimilars.

I am confident, thanks to the dedication of the employees of the FDA, that we will continue to make invaluable contributions to the lives of you, the American people.

I wish you and your family a healthy and happy 2012.

Margaret Hamburg, M.D., is Commissioner of the U. S. Food and Drug Administration.