Rare Disease Focus: FDA’s Trio of Cell and Gene Therapy Draft Guidances Highlight Expedited Programs, Innovative Trial Designs, and Postapproval Evidence Generation

Lexology November 6, 2025
Ropes & Gray LLP

FDA’s Center for Biologics Evaluation and Research (“CBER”) recently issued a trio of new draft guidances to assist sponsors who are developing and conducting postapproval studies of cell and gene therapies (“CGTs”):

These draft guidances address commitments FDA made as part of the seventh authorization of the Prescription Drug User Fee Act (“PDUFA”), PDUFA VII: Fiscal Years 2023–2027, to promote greater transparency and increase efficiency in the development of CGTs. This Alert describes the key takeaways for industry from the new draft guidances, which largely reference and expand upon principles articulated in previous agency guidances and address their specific application to CGT products intended to treat rare diseases.

Notably, CBER published these draft guidances as rare disease companies, researchers, providers,...

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