Innovation in New Drug Approvals of 2019 Advances Patient Care – FDA.gov

By: Janet Woodcock, M.D., Director, Center for Drug Evaluation and Research

Happy New Year! The beginning of a new year is always a rewarding time for the many dedicated staff at the FDA's Center for Drug Evaluation and Research (CDER) who work on approving new drug therapies. We look back over the past year and see how the work we did came together to help patients in need one of the main reasons why many of us, including me, have chosen a career in regulatory science and public health.

In 2019, we approved a wide variety of new drugs never before marketed in the United States, known as "novel" drugs, along with a range of new approvals containing active ingredients already on the market put to new and innovative uses. Many will have a positive, and even life-saving impact, on countless patients' lives.

New drug therapies for patients suffering from rare diseases are often among the most important approvals. Patients with rare diseases frequently have few or no drugs available to treat their condition and for them, approvals of so-called orphan drugs can mean new hope for an enhanced quality of life, and in some cases, survival.

In 2019, 21 of CDERs 48 novel drug approvals, or 44%, were orphan drugs. Like other recent years, 2019 shows a significant increase in CDERs orphan drug approvals compared to earlier years. The graph below shows that CDER approved more than twice as many novel orphan drugs from 2012-2019 than in the previous 8-year period from 2004-2011 142 vs. 63, a 125% increase.

In addition to CDERs approval of new therapies to treat patients with rare diseases, we approved many other important new therapies in 2019. These include:

Of note, we also approved ten new biosimilars, which can further help to create competition, increase patient access, and potentially reduce the cost of important biological drug therapies.

It is also gratifying to take a look back at 2019 and appreciate the efficiencies in our review of these newly approved therapies. The decisions we made on these approvals were generally completed by or before their goal dates as defined by Congressionally-mandated user fee programs a high-priority commitment for the FDA. Most were approved in the United States before any other country in the world.

Over the years, as the number of orphan drugs, targeted therapies (sometimes called personalized medicines), cancer treatments, and other medical advances being developed and reviewed has increased, so too has the need to tailor drug development programs and review to these particular therapies, as well as the use of FDAs expedited drug development and review programs to help potentially bring a wide range of new drugs to patients in need. In the past four years, from 2016 through 2019, of the 175 novel drugs CDER approved, 116 or 66%, used at least one expedited development program. By comparison, in the four years from 2012 through 2015, of the 152 novel drugs CDER approved, 89 or 59%, used at least one expedited development program.

As the use of tools intended to expedite the development and review of needed medications increases, our high standards for safety and efficacy have remained unchanged.

More details about CDERs new drug therapy approvals including many specific examples of notable new approvals for the year are available in our annual New Drug Therapy Approvals report.

I am proud to lead a team of dedicated professionals working to bring new therapies to patients as quickly as possible, while at the same time ensuring that approval for each and every one of these advances is based on our same high standards for science, safety and efficacy. As 2020 begins, we look forward to another strong year of safely advancing health care for the American public.

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Innovation in New Drug Approvals of 2019 Advances Patient Care - FDA.gov