The FDA is Developing, Approving, and Expediting Exciting New Therapies


Janet Woodcock, Director of the FDA’s Center for Drug Evaluation and Research (CDER) recently gave an interview to the publication FDA Voices about the agency’s approval in 2019 of a wide range of drugs.

A total of forty-eight novel drugs were approved in 2019 of which forty-four percent were orphan drugs. The number of orphan drug approvals has increased consistently every year.

Dr. Woodcock emphasized that drug therapies for rare diseases are one of the agency’s top priorities. She acknowledged that patients who have a rare disease are usually unable to find treatment. Therefore the “orphan” drugs that were approved last year can bring better quality of life for these patients; in some cases, it may mean their survival.

About Orphan Drugs

 Orphan Drug status is granted to biologics (large complex molecules) and drugs through the Orphan Drug Designation program.

Orphan drugs are administered for the prevention, diagnosis, or treatment of a rare condition or disease. To qualify as “rare” the condition would be one that affects less than two hundred thousand people in the United States or which conforms to the provisions (cost recovery) of the Act.

A Fast Paced Environment

Most definitions in the industry are constantly changing in order to keep pace with new products being brought to market.

A few new therapies approved by the FDA’s Center for Drug Evaluation and Research (CDER) in 2019 were for rare diseases such as cystic fibrosis, erythropoietic protoporphyria, systemic sclerosis-associated interstitial lung disease, Duchenne muscular dystrophy, and neuromyelitis optica spectrum disorder.

Emphasis on Efficiency

Dr. Woodcock was pleased to discuss the Agency’s efficiency in its review of new therapies. She stated that the decisions CDER made on granting approvals were mostly completed on or before their due dates.

Dr. Woodcock said a high priority for the FDA is meeting deadlines in accordance with user fee programs mandated by Congress. It is noteworthy that most of the novel drugs were approved in the U.S. last year before being approved in other countries.

New therapies have been approved for autoimmune conditions, infectious disease, tumors, blood disorders, and cancer. Endocrinology and neurological disorders can be added to the list.

The need for cancer drugs has grown rapidly. At this point, it amounts to over one-third of CDER’s drug approvals.

About Biosimilars

Dr. Woodcock gives special attention in the interview to the ten biosimilars that were also approved. A biosimilar functions in tandem with a biological product that has been tested and approved.

Biological products are either produced from or contain fragments of, living organisms. There is usually very little difference with respect to purity, potency, and safety.

Biosimilars offer patients additional treatment options. They increase the number of lifesaving drugs available to patients as well as lowering costs by promoting competition.

About First Cycles

The period between CDER’s acceptance of a new drug application and its decision about whether or not to approve the drug is called the First Cycle. The approval is followed by a complete response letter to the applicant.

Ninety percent of the forty-eight applications were approved by CDER during the first cycle in 2019. This is an improvement over eighty-four percent approvals from 2011 through 2018.

CDER is proud of its record and brings forward its commitment to work with applicants as they design studies and submit application data.

Expedited Programs

 CDER has four programs designed to facilitate the development of new drugs for life-threatening diseases, namely priority review and breakthrough designations. The other two programs are fast track and accelerated approval.

Sixty percent of CDER’s forty-eight new drug approvals took advantage of these programs in 2019. These programs cut the approval time by months or even years.

Dr. Woodcock refers to the number of targeted or personalized therapies and cancer treatments that are being developed. This has created a need to design programs specifically for these new therapies and encourage the use of the FDA’s expedited review programs.

Details of CDER’s novel drug approvals can be accessed in its New Drug Therapy Approvals report.

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia four years ago. He was treated with a methylating agent While he was being treated with a hypomethylating agent, Rose researched investigational drugs being developed to treat relapsed/refractory AML.

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