Pharmacists and specialty pharmacies in rare disease commercialisation

Rare diseases affect over 30 million people in Europe and pose significant challenges due to treatment complexity, high costs, and limited access. Pharmacists and specialty pharmacies play a critical role in addressing these issues by ensuring proper drug handling, supporting patient care, and aiding regulatory compliance. This paper highlights their contribution to improving access, treatment outcomes, and collaboration across the rare disease ecosystem.

In this guide we cover:

• Regulatory progress: The EU has developed robust frameworks like the Orphan Drug Regulation and European Reference Networks to foster innovation and improve rare disease treatment access.
• Pharmacist role: Pharmacists ensure the safe use, regulatory compliance, and personalised care of orphan drugs, contributing directly to patient safety and commercialisation success.
• Specialty pharmacy support: These pharmacies facilitate access through support programs, patient monitoring, and real-world data collection essential for rare disease management.
• Patient-centric approach: Collaboration with patient advocacy groups and personalised care models is central to improving treatment relevance, adherence, and outcomes.

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