Panel: Tackling trial design challenges in advanced therapies

Designing clinical trials for advanced therapies requires adapting to small and heterogeneous patient populations while maintaining scientific rigor. This panel examines approaches to leverage digital tools, real-world data, and proactive regulatory collaboration to enhance trial efficiency and translational validity. 

Key Takeaways: 

• Adapt trial design and recruitment strategies for small, rare, or underserved populations 

• Integrate real-world data and digital endpoints to improve efficiency and monitoring 

• Engage regulators early to streamline first-in-human trial approvals 

• Apply innovative approaches to maintain statistical power and predictive validity 

• Collaborate across stakeholders to ensure operational readiness and patient access