Research Advances for Rare Diseases and Orphan Products

Track:
Rare/Orphan Diseases

Day & Time:
June 25, 8:00AM - 9:30AM

Session Number:
222

Room Number:
257AB

Level:
Intermediate

Type:
Session

Title:
Research Advances for Rare Diseases and Orphan Products

Chair(s):
Peter L. Saltonstall
President and CEO
National Organization For Rare Disorders (NORD), United States

Description:
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(s):
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.

Presentation(s) & Speaker(s):
The Direction of Rare Disease Research
Marshall Summar, MD
Chief, Genetics and Metabolism
Children's National Medical Center, United States

Place of Value to Patients in Drug Development for Rare Diseases
Russell Teagarden
Senior Vice President, Medical and Scientific Affairs
National Organization For Rare Disorders (NORD), United States