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Thursday, October 15, 2026● Updated 6:00 AM ET
Medicines & FDA

FDA starts building a new path for rare disease treatments

Published

A healthcare professional wearing a protective mask and stethoscope

Only about 5% of rare conditions have an approved therapy. The agency wants clearer rules, earlier patient input and approaches suited to tiny trials, and it will hold a public meeting Oct. 28.

Source: HHS

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