Support multiple clinical development objectives, including in rare disease or subtypes
Characterize disease
Establish
endpoints
Engage stakeholders
Consider
external comparators
• Understand current standard
of care or treatment options
• Estimate disease prevalence
• Identify biomarkers
• Understand disease
progression
• Establish endpoints
or develop surrogate
endpoints
• Develop Clinical
Outcome Assessments
(COAs)
• Engage early with
patients for the trial
• Build relationships
with sites and KOLs
• Plan for future use
as an external
comparator arm
