White Paper: Navigating the Landscape of Compassionate Use

This white paper summarizes insights on compassionate use developed at Siren Interactive’s workshop,

Critical Issues in Patient Advocacy Engagement & Collaboration: How Do We Find Common Ground?, at

the World Orphan Drug Congress in April 2014. Participants included representatives from the industry

in commercialization and patient advocacy roles, rare disease patient advocacy group (PAG) leaders, and

patient opinion leaders (POLs). 

Download now

Special thanks are due to our moderator Wendy White, founder and CEO

of Siren Interactive, and panelists:

• Barbara Wuebbels, vice president of patient advocacy and medical affairs
• at Audentes Therapeutics
• Pat Furlong, founding president and CEO of Parent Project Muscular Dystrophy
• Ben Lenail, co-founder and business strategy officer of ALD Connect
• Julie Raskin, executive director of Congenital Hyperinsulinism International
• Carrie Burke, director of alliance development at Shire Pharmaceuticals
• Nicole Boice, founder and president of Global Genes/Rare Project