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Case Studies

Operationalise an EAP for a rare neurodegenerative condition (ALS)


  • High unmet need
  • History of challenges around early access to treatments

Customized Approach

  • Early engagement with patient advocacy community
  • Open, transparent communications


  • Collaboration with patient advocacy community on protocol, eligibility criteria and consent form
  • Community education and awareness on EAP process
  • Patient facing materials and FAQ’s incorporated into the design
  • >200 patients received early access to treatment