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Natural History Studies: Launch With Care And Consider Alternative Evidence For Drug Approval, FDA’s Marks Says

 
• By Sue Sutter

A different evidence-generation approach may lead to a quicker approval than leveraging a natural history study, CBER Director Peter Marks says; EveryLife Foundation's Annie Kennedy says participation in natural history studies can empower patients who are not eligible for drug-specific clinical trials.

Rocket launch
Launching natural history studies requires careful consideration, CBER Director Peter Marks said. • Source: Shutterstock

Care needs to be taken in planning and launching long-term natural history studies if they are eyed to support drug approval decisions, with consideration given to potential alternative evidence that might help bring a new rare disease treatment across the regulatory finish line more quickly, the US Food and Drug Administration’s Peter Marks said.

Speaking at the World Orphan Drug Congress USA on 24 May, the Center for Biologics Evaluation and Research director was asked how to help patient groups design natural history studies...

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