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NewsFDAMonday, February 23, 2026 · February 23, 2026

FDA Launches Framework for Accelerating Development of Individualized Therapies for Ultra-Rare Diseases

WHY IT MATTERS

FDA announcement relevant to rare disease patients and caregivers.

The U.S. Food and Drug Administration today issued draft guidance for sponsors seeking approval for targeted individualized therapies by generating substantial evidence of effectiveness and safety when randomized controlled trials are not feasible due to small patient populations.

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Read the original at FDA
FDA DesignationOrphan Drug

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