Conjoining Six Spokes of ‘Orphan Wheel’

‘Rare’ is turning into the new common as these rare, life-threatening diseases are affecting larger populations – 6.5 to 10 cases in every 10,000 people, as defined by the WHO.
The last 10 years witnessed researchers raring to spin the ‘Rare Wheel’, dealing with successes and failures alike.
It is critical to understand and identify what it takes to spin this wheel in a positive direction, how much to prepare before the wheels of development are set in motion, how to correct the course after turning, and when to accelerate. These small, yet measured, decisions could make a huge difference in transforming the rare story.

Key Takeaways

• What has been done so far?
• Which are the 6 spokes of the orphan drug development wheel?
• What are the key considerations for keeping the ‘Rare Wheel’ turning?

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