Roundtable: Rare & Ultra-Rare Diseases: Designing Programs for Maximum Patient Impact

• How are organisations currently navigating corporate pressures while maintaining ethical and equitable access in rare and ultra-rare disease programs?
• What are the key challenges sponsors face when deciding between single-patient access and cohort programs in today’s regulatory environment?
• What practical program design or operational approaches have been most effective in ensuring meaningful patient access in rare and ultra-rare diseases?

Speakers:

Michele Rhee

Vice President, Patient Experience, Patient Advocacy & Early Access

Savara Pharmaceuticals