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

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.

The FDA’s January 2019 draft guidance on rare disease drug development emphasizes the role of natural history data. "The natural history of rare diseases is often poorly understood, and...

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