The words

By: Patrizia Cavazzoni, MD, Acting Director, Center for Drug Evaluation and Research

Throughout 2020, the U.S. Food and Drug Administration’s Center for Drug Evaluation and Research (CDER) was challenged responding to the COVID-19 pandemic while still working to bring safe and effective new drug therapies for a range of other diseases and conditions to patients in need. Today I want to share information about the successes with the approval of new drug treatments that we have achieved in 2020, despite the unprecedented challenges caused by the pandemic.

Innovation in many different medical conditions

In 2020, we approved a wide variety of new drugs that had never been marketed in the United States before, known as “new” drugs, along with a series of new approvals that include active ingredients already on the market for new ones. and innovative applications. Many will have a positive and even life-saving impact on the lives of countless patients. These new approvals include:

  • Advances in the treatment of infectious diseases, including the first FDA-approved drug in the US for the treatment of patients with COVID-19 (hospitalized adults and adolescents). We also approved new treatments for HIV-1, chronic hepatitis C, hospital-acquired bacterial pneumonia and ventilator-related bacterial pneumonia, and influenza prevention;
  • Neurological advancements such as those for the treatment of certain patients with Parkinson’s disease, migraine and multiple sclerosis;
  • Progression of heart, blood, kidney, and endocrine diseases, including new treatments for certain patients with heart failure, osteomalacia, and growth hormone deficiency;
  • Innovative new therapies for certain patients with autoimmune diseases, including those with polyarticular juvenile idiopathic arthritis and ulcerative colitis, and;
  • Many new therapies for a wide variety of cancers, especially lung cancer, for which we have approved twelve remarkable new treatments, two of which are also approved for the treatment of patients with certain types of thyroid cancer. We have also approved four new noteworthy therapies to treat patients with different forms of breast cancer. Other advancements include new therapies for patients with other cancers, urothelial cancer, colorectal cancer, prostate cancer and bladder cancer.
Patrizia Cavazzoni, MD
Patrizia Cavazzoni, MD

Some CDER approvals will help detect and diagnose disease: a diagnosis to help clinicians assess patients being evaluated for Alzheimer’s disease, a new diagnosis for finding tumors that have somatostatin receptor-positive neuroendocrine tumors and a new diagnosis for the detection and localization of prostate cancer.

New drug therapies for patients with rare diseases

New drug therapies for patients with rare diseases are often among the most important approvals. Rare disease patients often have little or no drugs available to treat their condition – and for them, approvals of so-called “orphan drugs” can mean new hope for improved quality of life and, in some cases, greater survival. In 2020, more than half (31 out of 53, or 58%) of all our new approvals involved drugs for the treatment of patients with rare diseases. These illnesses include:

  • Spinal muscle atrophy, Duchenne muscular dystrophy, tuberous sclerosis complex, Dravet syndrome, neuromyelitis optic spectrum disorder, lupus nephritis, Still’s disease, hypereosinophilic syndrome, pro-opio melanocortin deficiency, Cushing’s disease, thyroid eye disease, Ebola and severe malaria;
  • Rare cancers, including multiple myeloma, high-risk refractory or recurrent neuroblastoma, gastrointestinal stromal tumor, epithelioid sarcoma, metastatic cholangiocarcinoma, and mesothelioma.

Efficiency in getting new therapies to patients as quickly as possible

Our 2020 approvals reflect many efficiencies in our review process. The decisions we made about these approvals were generally completed on or before the target dates defined for the user compensation program set by Congress. Most were approved in the United States before any other country in the world.

  • Meet the goals of the user fee: In 2020, CDER met target dates of the Prescription Drug User Fee Act (PDUFA) for 100% of approved new drugs (53 of 53);
  • First cycle approvals: In 2020, CDER approved 49 of our 53 new approvals (92%) for the first cycle. A first cycle is the time from when CDER accepts an application for a new drug until we make the initial decision whether or not to approve the application. From 2011 to 2019, CDER approved 357 new drugs, of which 304 (85%) were approved during the first cycle. Our consistently high first cycle approval rate for new drugs reflects CDER’s commitment to working closely with applicants to design their studies and build their marketing applications;
  • Approvals for other countries: While regulatory processes differ widely between the FDA and regulatory agencies in other countries, 40 of the 53 new drugs approved in 2020 (75%) were approved in the United States before receiving approval in another country;
  • Accelerated programs for severe conditions: CDER houses four programs intended to facilitate and accelerate the development and revision of new medicines to significantly accelerate treatment or to address an unmet medical need for a serious or life-threatening condition: fast track, breakthrough designation, priority assessment and accelerated approval. In 2020, 68% of CDER approvals for new drugs (36 out of 53) used one or more of these accelerated programs, bringing patients to new therapies months or even years earlier than expected.

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

More details on CDER’s new drug therapy approvals for 2020 – including many specific examples of notable new approvals for the year – are available in our annual report. New Approval Report for Drug Therapy.

I am honored to work with so many in CDER who are committed to bringing new therapies to patients as quickly as possible, while at the same time ensuring that the approval for each of these advancements is based on our consistently high standards of science , safety, and efficacy. As 2021 begins, we look forward to continuing to serve the American public.