US FDA advances rare disease drug development

The US Food and Drug Administration (FDA) has introduced the Rare Disease Evidence Principles (RDEP) to provide greater speed and predictability in the review of therapies intended to treat rare diseases with very small patient populations with significant unmet medical need and that are driven by a known genetic defect.

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Through the RDEP process, sponsors will receive clearer guidance on the types of evidence that can be used to demonstrate substantial evidence of effectiveness. The RDEP developed and implemented jointly by the C