*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

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