10 October 2019 - The US FDA is taking notice of a unique and recent phenomenon where physicians and specialists can create a tailored treatment to help or even cure an individual patient with a rare genetic mutation that could prove fatal.

In an editorial published Tuesday in the New England Journal of Medicine, Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research, and Peter Marks, director of the agency’s Center for Biologics Evaluation and Research, explain how these new technologies can “permit the delineation of pathways for truly individualised drug development," but currently this type of "drug discovery and development is most advanced for antisense oligonucleotides, other types of treatments, including individualised cell and gene therapies, are following closely behind.”

An FDA spokesperson told Focus the agency expects to publish a draft guidance on individualised therapies regulation soon.

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