By: Janet Woodcock, M.D.

In 2017, FDA’s Center for Drug Evaluation and Research (CDER) approved many new drugs never before marketed in the United States, known as “novel” drugs, to help improve people’s health.

Novel drugs often represent innovative therapies for advancing patient care. 2017 was no exception. We approved new treatments for patients with rare diseases such as Batten disease, Chagas disease, and hemophilia A with inhibitors. We also approved new cancer therapies, new antibiotics, and new therapies for patients with multiple sclerosis, Parkinson’s disease, tardive dyskinesia, Duchenne muscular dystrophy, and amyotrophic lateral sclerosis (often called Lou Gehrig’s disease), among many others.

For the past six years, we have summarized our novel drug approvals in an annual report. This year, we expanded the report beyond novel approvals to show a wide range of other drug therapy approvals that help improve health. For example, we approved many new uses for drugs already on the market, extending their benefits and expanding their reach into new populations, such as children. We have also approved new dosage forms for drugs already available. These are the kinds of actions, separate from approving a novel drug, that can also provide important medical value.

How valuable are these other approvals? Let’s look at an answer in terms of “firsts.” There are many. For instance, there was the first approval of a drug to treat liver cancer in almost a decade, and the first approval of a drug to treat sickle cell disease in almost 20 years. Other firsts for approvals in 2017 will benefit certain patients affected by a wide range of medical conditions, including:

• Giant cell arteritis, a dangerous condition that results in inflammation of blood vessels;
• Cytokine release syndrome – a condition related to a reaction caused by a treatment called chimeric antigen receptor (CAR) T cell therapy;
• Chronic graft versus host disease after a bone marrow transplant;
• A type of blood cancer called marginal zone lymphoma;
• Eosinophilic granulomatosis with polyangiitis, a rare autoimmune disease that causes vasculitis, an inflammation in the wall of blood vessels of the body; and,
• Erdheim-Chester Disease, a rare cancer of the blood.

Other firsts for CDER’s drug therapy approvals that are not novel drug approvals include the first:

• Biosimilars to treat certain cancers;
• Immediate-release opioid product with properties intended to deter abuse;
• Once-monthly injectable buprenorphine product to help patients struggling with opioid addiction;
• Cancer treatment based on a genetic feature of the cancer rather than the location of the body where the tumor originated;
• Treatment to help prevent recurrence of renal cell carcinoma (kidney cancer);
• Complete regimen to treat HIV-1 that contains only two drugs, neither a nucleoside reverse transcriptase inhibitor, which can be detrimental to a patient’s kidneys, bones, and heart;
• Drug approved in the United States with a sensor embedded in the pill that enables a patient to create an electronic record that the medication was taken; and,
• Short-acting “follow-on” insulin product to treat patients with diabetes.

Advancing Health through Innovation: New Drug Approvals and Other Drug Therapy Advances of 2017

Our report Advancing Health through Innovation: New Drug Approvals and Other Drug Therapy Advances of 2017 tells the story of these and other important drug therapy approvals that occurred last year. The report also shows the variety of regulatory methods we used to efficiently execute our review and approval of novel drugs, while always prioritizing safety over speed. For example, CDER used at least one “expedited” development and review method for 61 percent (28) of the 46 novel drug approvals of 2017.

In part, as a result of CDER’s efficient methods, 78 percent (36 of 46) of the novel drugs approved in 2017 were approved in the United States before any other country, and 100 percent were approved within their targeted user fee date for application review, as per the goals of the Prescription Drug User Fee Act.

The increasingly vital role patients play in drug development and approval is very important to new drug regulation. CDER collaborates with a wide range of patient advocates and patients to ensure that patients’ perspectives are considered when the agency reviews development plans and drug applications that meet previously unmet needs. We also work with stakeholders in manufacturing, scientific, and medical organizations across the globe to help advance the science needed to develop and evaluate new drug therapies. Our report highlights some of the many public-private partnerships and consortia that CDER leads or participates in to support innovation and improved public health.

Finally, the 2017 report describes CDER’s response to the devastation inflicted by the 2017 hurricane season, working with hospitals and pharmaceutical manufacturers to limit drug shortages and keep those affected safe.

We hope our new report provides a deeper understanding of the many ways CDER works to support innovation and access to medications for improved public health.

Janet Woodcock, M.D., is Director of FDA’s Center for Drug Evaluation and Research