Read Now: The Intersection of Rare Disease and Advanced Therapies

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The Intersection of Rare Disease and Advanced Therapies and What it Means for Clinical Development

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Learn best practices and lessons learned from experts in advanced therapy and rare disease clinical trials as well as:

  • Stakeholder interest in advanced therapies
  • Added challenges of clinical development of advanced therapies for rare diseases
  • Setting up for success in advanced-therapy trials for rare diseases
    • Commitment to collaboration
    • Relevant and simple endpoints
    • Integrate patients into all trial functions
    • Partner with regulators early
    • Plan for site variability and long follow up
    • Adjust for COVID-19