Spinning the ‘RARE Wheel’

Understand the trends in orphan drug approvals, clinical & commercial strategies
‘RARE’ is becoming the new common as these life-threatening diseases are affecting larger populations, with 6.5 to 10 cases in every 10,000 people as defined by the WHO. Last decade witnessed the researchers raring to spin the RARE wheel.

Numbers suggest that this area is a promising ‘mining opportunity’
But, to mine out a viable product, it is imperative to find answers to tough questions that some of the major pharmaceutical companies seem to be grappling with; most important being – “how to ensure optimal ROI from investments in orphan drugs while navigating through a complex maze of regulatory and pricing challenges?”

Key Takeaways

• Understanding the evolution of the regulatory benefits across different regions and their impact/benefits
• Five strategies for orphan drug development
• Top investment categories
• A rare cancer journey - to understand the market opportunities and pricing strategies
• Capitalizing on 6 spokes of the “Orphan Drug Development Wheel”
• Examples of market access engagements in the real world

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