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A Message from the Commissioner
Scientists have identified nearly 7,000 rare diseases ranging from rare cancers to metabolic disease and in the United States alone, more than 30 million people—or 1 out of every 10 Americans—have at least one rare disease. Tragically, more than half of these people are children.
The United States observes the last day of February as Rare Disease Day to raise awareness among the public and decision-makers about rare diseases and their impact on patients’ lives. Here at FDA, the observance has been expanded to include a full week of activities to reflect on what progress we have made—and what more needs to be done.
Thirty-five years ago, there were few drugs and fewer devices to treat these rare diseases. To spur drug development, Congress enacted the Orphan Drug Act in 1983, which provided financial incentives and other inducements. This was followed in 1990 with legislation establishing a rare disease path to market for medical devices. Since the original Act was passed, we’ve made great progress. Over the last 35 years, drugs and biologics have been developed and approved for 650 rare disease indications, and 72 devices have received approval for orphan indication under the humanitarian device program. In 2017, FDA had its most approvals yet for rare disease indications.
Despite our successes, there are still no treatments for the vast proportion of rare diseases or conditions. FDA is committed to do what we can to stimulate the development of more products by improving the consistency and efficiency of our reviews, streamlining our processes and supporting rare disease research. We have developed a series of new videos and other materials that explain the scope of what FDA is doing to encourage the development of medical products for rare diseases. I invite you to take a look at them.
—Scott Gottlieb, M.D., Commissioner of Food and Drugs
FDA is announcing the availability of the following materials and/or activities.
- FDA Voice Blog: “FDA Marks the 11th Rare Disease Day” by Scott Gottlieb, M.D.
- New pilot program: FDA is launching a new pilot for orphan designation requests, including a new fillable form for sponsors.
- Public workshop: The FDA is announcing this public workshop to help the agency prepare for the changing landscape of orphan drug development posed by targeted therapies and molecularly defined diseases.
- Memorandum of Understanding: This new MOU defines the framework for collaboration between FDA and the National Organization for Rare Disorders (NORD).
- 2018 needs assessment report: FDA and the NIH National Center for Advancing Translational Sciences/Office of Rare Diseases Research conducted this needs assessment to better understand unmet medical device needs for rare diseases, among other goals. The report is available on the FDA’s website.
- Data: Drug Development for Very Rare Diseases
Learn about a recently approved new therapy that helps treat a rare childhood cancer from Peter Marks, M.D., Director of FDA’s Center for Biologics Evaluation and Research.
Janet Woodcock, M.D., Director of FDA’s Center for Drug Evaluation and Research, gives her perspective on new therapies, along with a Batten disease advocate and other FDA staff.
FDA has approved 72 devices for rare diseases under the Humanitarian Use Device program, notes Jeffrey Shuren, M.D., J.D., Director of FDA's Center for Devices and Radiological Health. Agency experts describe three of them.
Debra Lewis, O.D., M.B.A.
Acting Director, OOPD
Andrea C. Furia-Helms, M.P.H.
Acting Director, Patient Affairs Staff, OMPT
RADM Denise Hinton
Acting Chief Scientist
Kathleen L. Miller, Ph.D.
John Swann, Ph.D.
These patient and stakeholder videos reflect the views of those in the videos and should not be construed to represent FDA's views or policies.
FDA will join the National Institutes of Health (NIH) to celebrate Rare Disease Day with a one-day program at NIH Masur Auditorium on March 1, 2018. The FDA's Office of Orphan Products Development will present FDA rare disease highlights and its grants research collaboration with NIH for rare diseases. For more information about this event, see Rare Disease Day at NIH 2018.
Previous Rare Disease Day Commemorations