Existing Innovative Trial Design, Endpoint Tools Could Be Help Ultra-Rare Sponsors Need

Amid a push for new incentives, the US FDA’s Kerry Jo Lee, associate director for rare diseases in the Center for Drug Evaluation and Research’s Office of New Drugs, said in an interview with the Pink Sheet that the tools ultra-rare sponsors need already are available.

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Kerry Jo Lee said tools like model-informed drug development meeting program could be applied to ultra-rare disease drug development. • Source: Shutterstock

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