From innovation to access: securing the last mile in rare disease

Why do approved rare disease therapies still struggle to reach European patients?

Bringing a rare disease therapy to market is only one of the first challenges after approval. Across Europe’s more than 27 healthcare systems, biotech companies face complex regulatory pathways, diverse HTA requirements and operational barriers that delay patient access, even after approval.

This white paper explores the potential barriers between regulatory approval and real-word patient access; and the strategic solutions proven to work.

Download to discover:

• Operational bottlenecks slowing therapy delivery across Europe
• Why clinical evidence fails to secure reimbursement and what payers actually need
• How innovative trial designs and stakeholder engagement accelerate access
• The role of real-world evidence in proving therapy value to payers
• Practical frameworks for building commercial strategies across 27+ markets

Download your free copy and close the gap between innovation and access.