Panel Discussion: Clinical Development Processes for Rare Disorders: Moving Towards a Patient-Driven Approach

Time: 1:30 pm
day: Day One


  • The unique challenges faced in attaining treatments by patients with rare disorders
  • Where patient perspectives can be conducive to success in the clinical trial endpoint design of therapies for rare disorders
  • Good and bad clinical development stories from trials taking different approaches to patient advocacy input
  • The case behind biotech and pharma incorporating patient perspective early in their drug development processes