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Orphan Drug Pricing Heading To Negotiating Table in US?

 
• By Cathy Kelly

The Institute for Clinical and Economic Review is launching an initiative to adapt its value assessment approach to orphan and ultra-orphan drugs, including a possible consideration of what should be a fair return on investment for manufacturers.

Handshake of businessmen

US payers are seeking tools for managing orphan drug costs in a way that more explicitly ties pricing to value. This is relatively uncharted territory in the US, where the prices set by manufacturers for drugs targeting few patients have generally been considered non-negotiable.

Cost pressures on payers are growing, driven by a significant acceleration in orphan drug approvals, as well as increasing prices (see box).

Orphan Drugs: A Burgeoning Market
  • 30 million Americans are affected by one of 7,000 rare diseases
  • Worldwide sales of orphan drugs expected to exceed $200bn by 2022
  • Average annual cost for orphan...

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