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Agency specifies several approaches to augment safety assessment in a new draft guidance, which also encourages participation by patients and their advocates in the drug development process.

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including gathering robust natural history data and using a concurrent comparator arm, the US FDA advises in a new draft guidance.

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Payers and health technology assessment bodies in the Netherlands, Germany and Italy are either unwilling to use real-world data in assessments or cannot due to their existing frameworks, say representatives from Gilead Sciences and Autolus Therapeutics.

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