Rare Diseases: US FDA Gives More Guidance On Premarket Safety Evaluation, Historical Controls

Agency specifies several approaches to augment safety assessment in a new draft guidance, which also encourages participation by patients and their advocates in the drug development process.

Rare Disease ribbon

Sponsors developing drugs for rare diseases should pursue several strategies to assess safety, including gathering robust natural history data and using a concurrent comparator arm, the US FDA advises in a new draft guidance.

The draft guidance, "Rare Diseases: Common Issues in Drug Development," revises and replaces a draft by the same name issued in August 2015. (Also see "As Orphan Drugs...

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