Throughout 2020, the U.S. Food and Drug Administration’s Center for Drug Evaluation and Research (CDER) was challenged to respond 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’d like to share information on the new drug therapy approval successes we achieved during 2020 despite the unprecedented challenges caused by the pandemic.

Innovation Across Many Different Medical Conditions

In 2020, 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. These new approvals include:

  • Advances to treat infectious disease, including the first FDA-approved medication in the U.S. for the treatment of patients with COVID-19 (hospitalized adults and adolescents). Among others, we also approved new treatments for HIV-1, chronic hepatitis C, hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia, and flu prevention;
  • Neurological advances such as those to treat certain patients with Parkinson’s disease, migraine, and multiple sclerosis;
  • Heart, blood, kidney, and endocrine disease advances which include new treatments for certain patients with, heart failure, osteomalacia, and growth hormone deficiency;
  • Innovative new therapies for certain patients with autoimmune conditions including those with polyarticular juvenile idiopathic arthritis, and ulcerative colitis, and;
  • Many new therapies for a wide array of cancers, particularly lung cancers, for which we approved twelve notable new treatments, two of which were also approved to treat patients with certain types of thyroid cancer. We also approved four new notable therapies to treat patients with various forms of breast cancer. Other advances include new therapies for patients with other forms of cancer urothelial cancer, colorectal cancer prostate cancer, and bladder cancer.
Patrizia Cavazzoni M.D.
Patrizia Cavazzoni M.D.

Some CDER approvals will help detect and diagnose disease: a diagnostic to help clinicians assess patients being evaluated for Alzheimer’s disease, a new diagnostic for finding tumors called somatostatin receptor positive neuroendocrine tumors, and a new diagnostic for 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. 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, increased survival. In 2020, more than half (31 of 53, or 58%), of all of our novel approvals were for drugs to treat patients with rare diseases. Among many others, these diseases include:

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

Efficiencies in Getting New Therapies to Patients as Quickly as Possible

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

  • Meeting User Fee Goals: In 2020, CDER met its Prescription Drug User Fee Act (PDUFA) goal dates for 100% of the novel drugs approved (53 of 53);
  • First Cycle Approvals: In 2020, CDER approved 49 of our 53 novel approvals (92%) on the first cycle. A first cycle is the time from when CDER accepts an application for a new drug until we make the first decision to approve or not approve the application. From 2011 through 2019, CDER approved 357 novel drugs, of which 304 (85%) were approved on the first cycle. Our consistently high first cycle approval rate for new drugs reflects CDER’s commitment to working closely with applicants as they design their studies and build their marketing applications;
  • Approvals Before Other Countries: Although regulatory processes differ widely between the FDA and those of regulatory agencies in other countries, 40 of the 53 novel drugs approved in 2020 (75%) were approved in the United States before receiving approval in any other country;
  • Expedited Programs for Serious Conditions: CDER houses four programs intended to facilitate and expedite development and review of new drugs to significantly advance treatment or address unmet medical need for a serious or life-threatening condition: fast track, breakthrough designation, priority review designation, and accelerated approval. In 2020, 68% of CDER’s novel drug approvals (36 of 53) used one or more of these expedited programs, bringing new therapies to patients months, or even years, sooner than expected.

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 CDER’s new drug therapy approvals for 2020 — including many specific examples of notable new approvals for the year — are available in our annual New Drug Therapy Approvals report.

I am honored to work with so many in CDER dedicated to bringing 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 consistently high standards for science, safety, and efficacy. As 2021 begins, we look forward to continuing to serve the American public.