Case study
Comprehensive Patient Perspective
Examine what Really Matters to Patients
Situation
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.
Action
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
HEOR Consulting
- Enabling Activity
Comprehensive Patient Perspective
- Region
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- Client
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Impact
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