There are more than 3000 diseases classified as “orphan diseases” and 300 approved medicines for the treatment of these diseases.

While regulatory approvals for orphan drugs are at an all time high there are still significant challenges in bringing innovative medicines to patients. A sense of urgency to develop effective treatments for orphan diseases is exemplified by the actions of passionate patient advocacy groups demanding changes in the drug development and regulatory processes to approve innovative treatments for orphan diseases.

Come join us to hear our panel discuss challenges in the development of preclinical models for orphan diseases and address such questions as:

• How are new technologies being leveraged to facilitate target identification and led optimization?
• What are the risks associated with these new paths towards clinical development?
• What are the challenges from a regulatory and clinical design perspective as we bring new technologies into clinical development?
• Are there ways to derisk innovative treatments more quickly in the orphan disease space ?

In Part II we will carry the discussion forward from proof of concept studies to market approval as we explore the many challenges and solutions found on the development side of rare disease research.