FDA Guidance–Rare Diseases: Natural History Studies for Drug Development

Mar 27, 2019 | FDA, Natural History, News, Rare Diseases, Regulations, Regulatory Affairs, Regulatory Findings

Rare Disease

The Food and Drug Administration (FDA) has released a draft guidance titled “Rare Diseases: Natural History Studies for Drug Development” with the intention to inform both the design and execution of natural history studies to support the development of safe and effective rare disease treatments. Significant progress aside, few rare diseases have approved therapies. A great unmet need now moves the FDA into action. Follow the link to read the actual guidance.


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