Access Strategies for Rare Disease Treatment
Time: 11:00 am
day: Pre-Conference Day Blue Track - Midday
Details:
- Exploring initiatives that provide drugs to children with rare diseases, focusing on specialized approaches and strategies
- Highlighting the steps and regulatory considerations involved in establishing managed access programs for rare conditions
- Discussing potential loopholes and challenges in providing very early access to drugs for super rare diseases without robust clinical data
- Suggesting practical measures that regulatory agencies can implement to facilitate drug development and access for patients with rare conditions
- Engaging with patient communities groups
