By: Patrizia Cavazzoni, M.D., Director, Center for Drug Evaluation and Research
The U.S. Food and Drug Administration’s Center for Drug Evaluation and Research (CDER) approved a wide variety of safe and effective new drug therapies in 2021 despite challenges brought on by the ongoing COVID-19 pandemic. These new approvals, spanning a wide range of diseases and conditions, aim to help many patients live better and potentially longer lives.
Innovation Across Medical Conditions
In 2021, we approved 50 new drugs never before approved or marketed in the U.S., known as “novel” drugs. We also approved drugs in new capacities, such as for new uses and patient populations.
The 2021 approvals target diseases and conditions such as:
- Heart, blood, kidney, and endocrine diseases, including treatments for diabetes, chronic kidney disease, and chronic weight management.
- Autoimmune, inflammatory and lung diseases, such as inflammatory bowel disease and cystic fibrosis.
- Neurological conditions, such as Alzheimer’s disease, opioid overdose, and migraine.
- Infectious diseases, including HIV-1, smallpox, and cytomegalovirus infection.
- Different types of cancers, such as lung cancer, basal cell carcinoma, kidney cancer, breast cancer and stomach cancer.
New Drugs for Patients with Rare Diseases
Patients with rare diseases are often in critical need of new therapies, as these individuals often have few or no existing treatment options. In encouraging news, last year more than half (26 of 50, or 52%) of our novel drug approvals were for rare diseases, including:
- Achondroplasia (the most common form of dwarfism); polycythemia vera (a blood disease); Duchenne muscular dystrophy (a disorder leading to progressive muscle degeneration); generalized myasthenia gravis (a chronic neuromuscular disease that causes weakness in skeletal muscles); and Pompe disease (a disease characterized by an enzyme deficiency).
- Rare cancers, such as cholangiocarcinoma (cancers starting in the bile duct); tumors associated with von Hippel-Lindau disease (an inherited disorder characterized by tumors and cysts); light chain amyloidosis (a cancer associated with abnormal protein buildup); perivascular epithelioid cell tumor (a group of rare cancers); and ovarian cancer.
Efficiencies in Bringing Therapies to Patients
Our 2021 approvals demonstrate efficiencies in our review process, such as:
- Meeting User Fee Goals: In 2021, CDER met its Prescription Drug User Fee Act goal dates for 49 of the 50 novel drugs approved (98%).
- First Cycle Approvals: In 2021, CDER approved 43 of the 50 novel approvals (86%) on the first cycle. This contrasts with CDER not approving the drug at first and possibly asking the applicant for more information.
- Approvals Before Other Countries: 38 of the 50 novel drugs approved in 2021 (76%) were approved in the U.S. before being approved in another country.
- Expedited Programs for Serious Conditions: the FDA has four programs to facilitate and expedite development and review of drugs for serious or life-threatening conditions: Fast Track designation, Breakthrough Therapy designation, Priority Review designation, and Accelerated Approval. In 2021, 37 of the 50 of CDER’s novel drug approvals (74%) used one or more of these expedited programs, which helped bring new therapies to the market sooner than expected.
The First Interchangeable Biosimilars
CDER approved the first two interchangeable biosimilar products in 2021, which are biological products that may be substituted without the intervention of a prescriber. More biosimilar and interchangeable biosimilar products on the market means greater competition that may lead to increased access to therapies and lower costs for patients.
The 2021 New Drug Therapy Approvals Report
More details about CDER’s drug approvals for 2021 — including specific examples of notable approvals — are available in our annual New Drug Therapy Approvals report. I want to also mention that the FDA’s Center for Biologics Evaluation and Research (CBER) approves important therapies as well. In 2021, CBER approved the first COVID-19 vaccine, an important tool in our continued fight against the pandemic. For more information about CBER actions, visit CBER’s web page for 2021 Biological Approvals.
I enjoy working with so many talented individuals in CDER who are determined to bring new drug therapies to patients as quickly as possible, while also ensuring that every approval is based on our consistently high standards for science, safety, and effectiveness. As 2022 begins, our center looks forward to continuing this important work in the name of public health.