The Not So Rare Challenge that Faces Rare Disease Development: Demonstrate Value

Track:
Rare/Orphan Diseases

Day & Time:
June 27, 9:00AM - 10:30AM

Session Number:
416

Room Number:
154

Level:
Basic

Type:
Session

Title:
The Not So Rare Challenge that Faces Rare Disease Development: Demonstrate Value

Chair(s):
Charles A. Stevens, JD,MBA
Vice President and General Manager
PAREXEL Consulting, United States

Description:
Increased development of orphan products has changed how payer regulators view rare diseases. Rare disorders can now have several products FDA indicated for use; this requires that developers demonstrate the value of these rare disease products.

Learning Objective(s):
Explain how rare disease and orphan products are changing payer regulator views on how to manage these products; Discuss how patient subpopulations, even within rare disorders, can impact overall product value and access; Describe the importance that budget impact models have even in rare disorders.

Presentation(s) & Speaker(s):
Panelist
Paul T. Kim, JD
Partner
Foley Hoag LLP, United States

Panelist
Jim Long
Co-Founder and Principal
BioSolutia, United States