Agenda Archive

Registration and refreshments

Understanding the patient advocacy ecosystem and its growing influence on clinical research
Leveraging patient advocacy organizations to enhance study design and patient engagement
Learn best practice for building effective partnerships with advocacy groups to support more robust and patient-centered trials

Designing development strategies that support earlier intervention and improve long-term outcomes
Gaining practical insights into how to optimize trial design, patient recruitment, and the use of real-world endpoints
Addressing the complexities of small, heterogeneous pediatric populations to enhance study feasibility and success
Engaging regulators effectively and plan for how pediatric medicines are evaluated differently—especially for cell and gene therapies and earlier treatment approaches

The real-world barriers families face when participating in rare disease trials
Practical ways sponsors and trial designers can reduce burden in protocol design
The role of patient advocacy organizations in helping improve recruitment and retention
Lessons learned from the Angelman syndrome community as multiple clinical trials have launched simultaneously

Tips and tricks on how to shorten development timelines and reach first-in-human studies faster
Identifying opportunities to compress timelines without compromising quality
Collaborating with CROs effectively to drive operational efficiency and aid faster study start-up