Situation: A Phase 3 study in a rare disease

The Success:

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.