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Research Advances for Rare Diseases and Orphan Products
Session Chair(s)
Peter L. Saltonstall
President and CEO
National Organization For Rare Disorders (NORD), United States
Research into new orphan drugs has grown substantially in recent years. In 2012, a third of the drugs approved by the FDA had orphan designation. This session will examine the current research efforts and discuss the role of the patient community, the FDA, the NIH and the drug industry in research priorities and challenges. Included will be the role of patient registries and natural histories. This session is hosted by the National Organization for Rare Disorders (NORD).
Learning Objective : Discuss current and future research priorities for orphan drugs; Describe the challenges facing the development of new orphan drugs in the evolving health care environment.
Speaker(s)
The Direction of Rare Disease Research
Marshall Lynn Summar, MD
Children's National Medical Center, United States
Division Chief, Genetics and Metabolism; Director, Rare Disease Institute
Place of Value to Patients in Drug Development for Rare Diseases
J. Russell Teagarden, PhD, RPh
Independent Advisor, United States
Senior Health Care Practioner, Researcher and Writer
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