The US Food and Drug Administration (FDA) has been preoccupied with COVID-19 for nearly 2 years. But that hasn’t distracted the agency from ensuring the development of therapies for rare disease patients, said Acting FDA Commissioner Janet Woodcock, MD.

Dr. Woodcock spoke on October 19, 2021, the second day of the 2-day virtual National Organization for Rare Disorders (NORD) Breakthrough Summit 2021.

“The challenges involved with rare diseases are significant and unique,” she said. “To meet the needs of a community forced to operate under a different paradigm and spur development of new drugs, we have to do things differently, try new approaches, and use new and modern technologies to advance innovation.”

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Dr. Woodcock joined the FDA in 1986, only 3 years after congressional passage of the Orphan Drug Act (ODA).

Janet Woodcock, acting commissioner of the US Food and Drug Administration (FDA). Credit: Getty Images

“That groundbreaking law was designed specifically to provide incentives for the development of drugs for people with rare diseases,” said Dr. Woodcock, who last year received NORD’s Lifetime Achievement Award. “The ODA has actually changed the face of drug research. Since it was enacted, we’ve approved over 500 new drugs for the treatment, diagnosis, or prevention of more than 1000 orphan indications. And the trend continues upward.”

Last year, the FDA approved some 90 drugs to treat a wide range of rare conditions, including several that involve CAR-T cells, which use a patients’ own immune system to help fight cancer. In addition, during its most recent grant cycle, Dr. Woodcock said, the FDA awarded 11 new clinical trial research grants totaling $25 million.

“Of course, we have a long way to go,” she continued. “Despite significant advances, most rare diseases continue to lack an FDA-approved treatment, and there remain numerous obstacles for investment in research and development. One way we’re working to overcome these hurdles is by providing important funding for clinical trials and natural history studies to advance rare disease medical product development.”

More from the NORD summit: The Use of Data Science Could Accelerate Rare Disease Research

The 2016 passage of the 21st Century Cures Act created the regenerative medicine advanced therapy (RMAT) designation, which offers a new, expedited option for certain eligible biologic products. As of September 2021, the FDA’s Center for Biologics Evaluation and Research (CBER) had granted 64 RMAT requests, half for products that have orphan drug designations.

Dr. Woodcock also touched on gene therapy, which she said “offers great promise for a growing number of devastating rare diseases,” particularly those caused by single genetic mutations.

“The number of individuals usually diagnosed with a rare genetic disease is extremely small. Development of a gene therapy is likely to be avoided by many developers because it wouldn’t be considered economically feasible,” she said. “We recently issued final guidance on how we determine exclusivity and sameness related to gene therapy. The FDA is also working to encourage investment and innovation in this area.”

Three other FDA officials also participated in the session: Peter Marks, MD, PhD, director of CBER; Patrizia Cavazzoni, MD, director of the agency’s Center for Drug Evaluation and Research; and Jeffrey Shuren, MD, JD, director of its Center for Devices and Radiological Health.


FDA commissioner keynote and updates from FDA center directors. Presented at: NORD Breakthrough Summit 2021: October 19, 2021; Virtual.