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