Meeting the Needs of Patients with Rare Diseases


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Product Description

The National Institutes of Health Office of Rare Diseases (ORD) lists more than 6,000 rare diseases affecting an estimated 25 million people in the United States.
The goals of ORD are to stimulate and coordinate research on rare diseases and to support research to respond to the needs of patients who have any one of these rare diseases.
“More than 280 products have been approved since 1983 under the Orphan Drug Act (ODA),” says Marlene Haffner, M.D., MPH, director of the Office of Orphan Products Development (OOPD). “In the aggregate, those products have treated more than 14 million people in the United States. So the incentives provided by the ODA are working well. The downsides are that we do not know if the products developed for a particular rare disease will help everyone who has that disease and we are challenged to find all the patients and give them access to treatments.”
In the last decade alone, 160 new medicines to treat orphan-designated diseases have been approved by the Food and Drug Administration, according to a new report by the Pharmaceutical Research and Manufacturers of America (PhRMA), National Organization for Rare Disorders (NORD), and the Genetic Alliance (GA)…

Global Orphan Drug Legislation
Recently Approved Orphan Drugs
Orphan Drug Approvals in Europe Lag

Experts on this Topic
Marlene Haffner, M.D., MPh. Director of the Office of Orphan Products Development (OOPD), U.S. Food and Drug Administration; Rockville, Md.; the FDA is responsible for protecting the public health by assuring the safety, efficacy, and security of human and veterinary drugs, biologics, devices, and products that emit radiation. For more information, visit
Emil D. Kakkis, M.D., Ph.D. Chief Medical Officer, BioMarin Pharmaceutical Inc., Novato, Calif.; BioMarin develops and commercializes biopharmaceuticals for serious diseases and medical conditions. For more information, visit
Gregg Lapointe. Chief Operating Officer, Sigma-Tau Pharmaceuticals Inc., Gaithersburg, Md.; Sigma-Tau is dedicated to creating novel therapies for the unmet needs of patients with rare diseases. For more information, visit
Peter Norman, Ph.D. Norman Consulting, Burnham, United Kingdom; Peter Norman is an independent consultant. For more information, e-mail
Joshua D. Schein, Ph.D. CEO and Director, Lev Pharmaceuticals Inc., New York; Lev Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing therapeutic products for the treatment of inflammatory diseases. For more information, visit