Successful Phase 2 clinical trial results for a rare disease product meant there was a need to develop a robust strategy and a concrete tactical plan for real-world evidence generation to fill gaps in understanding and to complement available clinical data. To develop this strategy and plan, a detailed understanding of stakeholder beliefs and behaviours was needed, as well as a comprehensive oversight of existing real-world evidence and the remaining evidence gaps.

Our approach to developing the real-world evidence strategy included:

  • Desktop research: market/competitor analyses and literature review to identify key themes and gaps in real-world evidence in the disease area
  • Stakeholder research: qualitative interviews with key internal and external stakeholder advisors (payors and clinicians) to gain input into local, regional, national, and global gaps and needs and emerging research and opinion that should be addressed through the real-world evidence strategy/plan

This research allowed us to create a tactical plan that outlined:

  • Real-world evidence gaps and key challenges, by market
  • Real-world evidence needs and potential research approaches
  • Available real-world data sources

Stakeholder interview insights, together with a literature review and gap analysis, identified key real-world evidence needs and revealed potential strategies to fill gaps in understanding. The process of validating the draft strategy and plan via internal stakeholder workshops created strong internal buy-in and support for the plan and increased awareness of the value of real-world evidence within the broader cross-functional team.