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,
Key Takeaways
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CBER is committed to leveraging biomarkers and the accelerated approval pathway to bring gene therapies to market for rare diseases.
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Collaboration between CBER and CDER on rare disease issues is increasing, and there should be an explanation when there are differences in advice across the centers, Peter Marks said