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Rare Diseases: CBER Looks To ‘Lean Into’ Accelerated Approval, Align More With CDER

 
• By Sue Sutter

US FDA biologics center officials spoke about their efforts to increase collaboration and harmonization with the drugs center, and to internally involve more review disciplines in evaluating biomarker evidence, during a Reagan-Udall Foundation meeting that weighed potential use of accelerated approval for neuronopathic mucopolysaccharidoses disorders.

Walking in step
FDA's Peter Marks believes CBER and CDER will be walking in step more often going forward. • Source: Shutterstock

The US FDA Center for Biologics Evaluation and Research’s strategy to “lean in” to using biomarkers and accelerated approval for rare disease treatments includes more collaboration and harmonization with the agency’s drugs center, where possible, and taking a more multidisciplinary approach to the evidentiary evaluation of surrogate endpoints.

CBER officials spoke about the center’s approach to accelerated approval for gene therapies targeted at rare diseases at a 21 February meeting, hosted by the Reagan-Udall Foundation, on qualifying biomarkers...

Key Takeaways

  • CBER is committed to leveraging biomarkers and the accelerated approval pathway to bring gene therapies to market for rare diseases.

  • Collaboration between CBER and...

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