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Industry Trends, Successes, and Failures in Orphan and Rare Disease Therapeutics

Track:
Rare/Orphan Diseases

Day & Time:
June 17, 10:30AM - 11:30AM (Pacific Standard Time)

Session Number:
249

Room Number:
32AB

Level:
Intermediate

Type:
Forum

Title:
Industry Trends, Successes, and Failures in Orphan and Rare Disease Therapeutics

Chair(s):
Christine Blazynski, PhD
Chief Science Officer and Senior Vice President
Citeline, United States

Description:
This forum will overview orphan and rare disease drug development from the patient and affected community perspective to the industry's response over the past 30 years until today. The activist community will be contrasted with how industry has approached the needs of the few.

Learning Objective(s):
Evaluate industry's successes and failures in developing drugs that address rare and orphan diseases; Discuss failed and/or abandoned efforts in this therapeutic area; Report on current development pipeline and high interest versus low interest diseases.

Presentation(s) & Speaker(s):
When Your Company Adopts an Orphan Drug
Robin L. Winter-Sperry, MD
President and Chief Executive Officer
Scientific Advantage LLC, United States

Industry Trends, Successes and Failures in Orphan and Rare Disease Therapeutics
Christine Blazynski, PhD
Chief Science Officer and Senior Vice President
Citeline, United States