Completing Enrollment Early for a Phase 3 Rare Disease Study
Situation: A Phase 3 study in a rare disease
On a recent Phase 3 study for a rare disease, we randomized all 120 patients 4 weeks ahead of schedule. How were we able to do this?
- Created a critical path map showing all tasks needed to reach the next milestone (e.g., site activation, FPFV)
- Shared this map with all team members, sites, and vendors
- Collaborated closely with a patient advocacy group for the rare disease in question
- Maintained frequent and close communication with all stakeholders
This study included 37 sites across 5 countries. As a result, a treatment that has the potential to fulfill an unmet medical need for a serious rare disease is one step closer to being available to patients.