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Gene Therapy: Years After Accelerated Approval, Will US FDA Still Be Asking ‘Does It Work?’

 
• By Sue Sutter

Retiring Office of Tissues and Advanced Therapies’ Director Wilson Bryan expects the expedited pathway will be used more frequently moving forward, but worries that ambiguous confirmatory trial results many years down the line will still leave efficacy questions unanswered.

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The FDA's Wilson Bryan worries that answers to gene therapy efficacy questions will not emerge with the passage of time. • Source: Shutterstock

Accelerated approval is likely to be used more extensively in the future for gene therapies that treat very rare diseases, but the slowly progressive nature of some conditions means there may be many years of uncertainty about the clinical benefits provided by the one-time treatments, the head of the US FDA’s Office of Tissues and Advanced Therapies said.

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