Advances in Clinical Trial Design & Real World Evidence in Rare Disease

*Recommended for Young Investigators/Researchers

Addressing the Need for Innovative Clinical Trials to Advance Rare Disease Therapies

• Challenges in traditional double-blind randomized clinical trial design for rare disease studies
• Ethics and access to clinical trials in rare disease research

Basket & Umbrella Trials and Master Protocols: Applications for Rare Disease Research

• Developments in adaptive trial designs and regulatory flexibility

Statistical Implications of Trial Design on Measuring Efficacy & Endpoints