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

June 24, 2021

*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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