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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.

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