White paper


Managed Access: Creating Pathways to Treatment for Rare Disease Patients

Rare diseases present unique challenges, often leaving patients and their families searching for answers and options. Did you know that over 90% of rare diseases lack approved treatments? For those facing life-threatening conditions, waiting years for a drug to become available isn’t an option.

So, what’s the solution?

Enter Managed Access Programs (MAPs)—a lifeline for patients to access investigational treatments before regulatory approval. But MAPs are more than just a compassionate solution; they’re a transformative step in rare disease innovation.

Our latest white paper, “Managed Access: Creating Pathways to Treatment for Rare Disease Patients,” explores:

  • How MAPs provide early access to groundbreaking treatments.
  • Their role in advancing orphan drug development and accelerating commercialization.
  • The potential of real-world data to reshape regulatory and clinical pathways.
  • Practical steps to implement an effective MAP strategy for your treatment.