Evidence-based value and access strategies

Case Study: Endpoint Testing & Utilization

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For a large pharma company, Compass assessed an in-house orphan asset and provided evidence-based guidance for the clinical development plan

Client requested evidence-based guidance of the target product profile from both KOLs and payers to advance the asset in product development

Key Objective:
Identify drivers of value for a novel, first in class treatment of an orphan disease, to assist in guiding the commercial development and clinical teams

In-depth interviews with 30 KOLs and 25 payers in 8 countries including US, EU3, Brazil, Japan, South Korea and Taiwan


  • Screening, diagnosis and treatment of this rare disease in 8 markets highlighting similarities and differences
  • Estimates of prevalence and distribution of patients by disease severity
  • Identification of top endpoints that would be required for registration and use of a new therapy including minimal levels of improvement and time period to demonstrate effect needed
  • Comprehensive product assessment by KOLs and payers and expected utilization
  • Price recommendation by market
  • Findings were used by the client to plan the Phase III deliverable
Sample Deliverable

Utilization of the minimal and optimal profiles was assessed by disease severity