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Baby Steps To Real-World Evidence Of Efficacy: External Controls Gain Popularity In Rare Disease Trials

 
• By Bridget Silverman

The Pink Sheet takes a closer look at recent clinical announcements showing how rare disease therapy sponsors are already increasingly reliant on natural history studies to guide drug development.

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Increasing familiarity with the possibilities and constraints of natural history data can only help industry and regulators alike.

While the US FDA grapples with its emerging regulatory framework for real-world evidence, a small wave of recent clinical announcements illustrates how rare disease therapy sponsors are already increasingly reliant on natural history studies to guide drug development.

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