In rare disease, every Medical Affairs action counts—and Medical Affairs groups are increasingly seeking to prove their value. This report empowers Medical Affairs leaders with data-driven insights and practical frameworks to connect what they do with the outcomes that matter most.
Through extensive benchmarking and analysis, this research reveals how top-performing organizations are redefining Medical Affairs as a strategic engine of real-world impact. By linking leading indicators—like engagement quality, evidence generation, and MSL insights—with lagging indicators such as treatment uptake, clinical guideline inclusion, and access milestones, these teams are demonstrating measurable influence on patient care and organizational success.
As rare disease portfolios expand, so does the need for clear proof of value. Traditional activity metrics are no longer enough. Medical leaders must now show how their strategies directly drive treatment adoption, shape clinical practice, and accelerate access.
Developed by Best Practices, LLC, this report explores how innovative organizations are advancing measurement maturity through strategic indicator mapping, integration of real-world data, and smarter outcome linkage. Yet it also exposes a critical challenge—the ongoing gap between actions and outcomes. Closing that gap is the next frontier. It’s how Medical Affairs will secure its role as a central driver of evidence-based impact, cross-functional alignment, and sustained organizational value.