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

 
• By Brenda Sandburg

Agency specifies several approaches to augment safety assessment in a new draft guidance, which also encourages participation by patients and their advocates in the drug development process.

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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including gathering robust natural history data and using a concurrent comparator arm, the US FDA advises in a new draft guidance.

The draft guidance, "Rare Diseases: Common Issues in Drug Development," revises and replaces a draft by the same name issued in August 2015

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