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

 
• By Derrick Gingery

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

Existing US Food and Drug Administration programs intended to foster innovative and streamlined drug development may be the answer for the ultra-rare disease sector instead of new incentives, said Kerry Jo Lee, associate director for rare diseases in the Center for Drug Evaluation and Research’s Office of New Drugs.

Key Takeaways

  • Kerry Jo Lee said existing programs targeting innovative trial designs, modeling and simulation can be applied to rare and ultra-rare disease drug development.

  • Lee also said sponsors who work with FDA officials come to understand the tools that are available to help in rare disease drug development

Advocates have called for creation of an ultra-rare disease designation that would better tailor incentives for sponsors developing drugs in diseases affecting

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