Case study

Comprehensive Patient Perspective

Examine what Really Matters to Patients​


A mid-sized pharmaceutical company was developing a new treatment for a neurodegenerative rare disease. 

Given the promising finding from the phase 1 study, the company decided to skip phase 2 and to start a phase 3 study with limited information.​

The planned COA (a PerfO measure) for the primary endpoint was not developed for clinical trial use, it was unclear how much change is considered clinically meaningful.

AESARA member was tasked to define the COA effect size to inform the Phase 3 trial sample size. ​


  • AESARA member developed a COA strategy to support the Phase 3 design and FDA engagement

  • Reviewed literature and conducted COA data analyses to understand the disease natural history and COA measurement property

  • Included exploratory endpoints and analysis plan in the Phase 3 to demonstrate minimal clinically important difference (MCID)

  • Conducted a qualitative study to understand the perceptions of meaningful change from patient, caregiver, and clinician’s view

Value & Access Consulting

  • Enabling Activity
    Comprehensive Patient Perspective
  • Region
  • Client


  • The COA strategy and tactics provided timely support for the design of Phase 3 study and FDA engagement. The phase 3 trial result was positive, and the treatment was approved by the FDA

  • The research collaboration with the patient advocacy groups strengthened the relationship with the disease community

  • The qualitative study is recognized as a classic example of how to support patient-focused drug development

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