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Califf: Decades Of Follow-Up Necessary For Gene Therapy

 
• By Sarah Karlin-Smith

FDA Commissioner Califf acknowledges that regulatory pathways for gene therapies are still being developed and that many factors outside of the modality impact agency requirements as he emphasized the need for long-term follow up due to the unknowns of gene modification.

editing DNA
Unknowns about gene therapy effects won’t preclude fast approvals but will demand lengthy follow up, Commissioner Califf said • Source: Shutterstock

Long periods of follow-up may be necessary for gene therapies, particularly if and when the US Food and Drug Administration is willing to grant a company an accelerated approval for such a treatment, FDA Commissioner Robert Califf said.

At the J.P. Morgan Healthcare Conference 10 January, Califf was asked for his assessment of what circumstances he envisions gene therapies being given accelerated approvals using surrogate endpoints and whether...

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