New reports from the U.S. Food and Drug Administration (FDA) show a total of 55 novel new medicines were approved in 2019. Among these, 48 were approved by the Center for Drug Evaluation and Research (CDER) and 7 were approved by the Center for Biologics Evaluation and Research (CBER). While medicine approval numbers vary each year, 2019 saw exciting advancements in drug development. These strong approval figures demonstrate biopharmaceutical companies’ unwavering commitment to developing innovative treatments for patients, offering hope for improved health outcomes. 2019 also highlights continued success of the Prescription Drug User Fee Act (PDUFA) program that helps ensure timely and effective review of new drug applications (NDA). FDA’s CDER noted they “met or exceeded almost every PDUFA goal date for application review agreed to with the pharmaceutical industry and approved by Congress.”
CDER approved a variety of novel medicines that have never been approved or marketed in the U.S. that are advancing patient care as they treat previously unmet needs and diseases. Twenty of the 48 novel medicines (42%) approved in 2019 were considered first-in-class, utilizing previously unavailable approaches to treat illnesses. These first-in-class treatments approved in 2019 include medicines for bladder cancer, postpartum depression and other devastating illnesses where there is significant unmet patient need.
CBER approvals notably contributed to advances in cell and gene therapy, one of the most cutting-edge areas of research which use genetic material to treat the underlying cause of disease. This past year’s approval of a gene therapy for pediatric patients under 2 years of age with spinal muscular atrophy caused by a specific genetic mutation demonstrates the progress made in an area not long ago considered science fiction. Today, there are 6 approved cell and gene therapies in the U.S. which are fundamentally changing the trajectory of many serious and life-threatening conditions and transforming the lives of patients.
Progress for Rare Disease
Last year of CDER’s approvals, 21, were for patients with rare diseases, which are defined as diseases that affect fewer than 200,000 people in the U.S. These approvals included treatments for conditions such as sickle cell disease, cystic fibrosis, Duchenne muscular dystrophy and more. It’s worth noting that while drug development is already challenging, developing a treatment for a rare disease is even more so, with a Tufts Center for the Study of Drug Development finding it takes 2.3 years, or 18%, longer to develop an orphan drug compared with medicines to treat more common conditions.
2019 also marked a critical year for biosimilar approvals. CDER approved 10 new biosimilars, and we now have 26 approved as of December 2019. The U.S. market is evolving rapidly, and the benefits will continue to grow over time as more products are introduced. As of January 2020, there are 13 biosimilars on the market in the U.S., with 13 additional FDA approved products due to come to market over the next several years. Already biosimilars have increased competition, leading to lower prices and savings for patients
Improved Patient Access
Although regulatory processes differ greatly between the U.S. and other countries, 33 of the 48 novel therapies approved in 2019 (69%) were approved by the FDA before receiving approval in any other country, solidifying our nation’s role as the global leader in biopharmaceutical innovation. Additionally, 43 of the 48 novel medicines (90%) were approved on the “first cycle” of review, meaning that these candidate therapies were not delayed by re-submission.
As we look ahead to 2020, CDER’s 2019 drug approvals serve as a reminder of the remarkable progress of the biopharmaceutical research pipeline. Learn more about innovation from America’s biopharmaceutical companies here.
Andrew Powaleny is Senior Director of Public Affairs at PhRMA and leads the organization's scientific communications. Before joining PhRMA in 2015, he worked in public affairs for a small firm in Washington, DC and served as Deputy Press Secretary for the House Committee on Energy and Commerce. Andrew came to Washington, D.C. via Connecticut with a degree from Eastern Connecticut State University where he majored in public policy and government. Andrew is active as a runner and volunteer with the DC Front Runners; most recently serving on its Board of Directors for three years as co-race director. He is also a member of the NLGJA: The Association of LGBTQ Journalists and mentors students through his alumni association with The Fund for American Studies. Andrew is passionate about scientific innovation, especially for mental illness, and his heroes are the men and women of America’s biopharmaceutical research companies.